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Clinical Trials - Gene Therapy Trial Browser

The Gene Therapy Trial Browser represents a unique publicly accessible, free database for the benefit of users seeking information on gene therapy development. The information within integrates various sources, including clinicaltrials.gov, publications, sponsor press releases, patent applications, and more to give a comprehensive overview of the gene therapy clinical trial landscape.

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Indication

AADC Deficiency (3)

Achromatopsia (3)

Adrenoleukodystrophy (1)

Adrenomyeloneuropathy (1)

Alpha 1-Antitrypsin Deficiency (3)

Alpha-Thalassemia (1)

Alzheimer's Disease (1)

Alzheimer's Disease | Mild Cognitive Impairment (1)

Amyotrophic Lateral Sclerosis (1)

Arrhythmogenic Right Ventricular Cardiomyopathy (3)

Autosomal Recessive Congenital Ichthyoses, TGM1-deficient (1)

Batten Disease (Late Infantile Neuronal Ceroid Lipofuscinosis) (2)

Beta-Thalassemia Major (1)

Beta-Thalassemia, transfusion-dependent (5)

Bietti Crystalline Dystrophy (2)

Canavan Disease (2)

Choroideremia (1)

Chronic Granulomatous Disease, X-linked (2)

Chronic Granulomatous Disease, p47phox (2)

Congenital Adrenal Hyperplasia (1)

Congenital Hearing Loss (3)

Congestive Heart Failure (1)

Cystic Fibrosis (1)

Cystic Fibrosis, Modulator-Ineligible/Intolerant (1)

Cystinosis (1)

DMD-Associated Dilated Cardiomyopathy (1)

Danon Disease (1)

Diabetic Macular Edema (2)

Dravet Syndrome, SCN1A-Associated (1)

Drug Resistant Epilepsy (1)

Duchenne Muscular Dystrophy (4)

Dystrophic Epidermolysis Bullosa (2)

Fabry Disease (3)

Fabry Disease With or Without Cardiac Involvement (1)

Familial Hypercholesterolemia, Heterozygous (2)

Familial Hypercholesterolemia, Refractory (1)

Fanconi Anemia, subtype A (1)

Friedreich Ataxia (1)

Friedreich's Ataxia, with Cardiomyopathy (1)

Frontotemporal Dementia (3)

GM1 Gangliosidosis (2)

Gaucher Disease, Type 1 (2)

Gaucher Disease, Type 2 (1)

Geographic Atrophy (2)

Giant Axonal Neuropathy (1)

Glutaric Acidemia Type I (1)

Glycogen Storage Disease Type 2 (Late-Onset Pompe Disease) (4)

Glycogen Storage Disease Type Ia (1)

HIV-1 Infection, subgroup B (1)

Heart Failure with Preserved Ejection Fraction (1)

Heart Failure with Reduced Ejection Fraction (1)

Hemoglobin H disease, Constant Spring type (1)

Hemophilia A (9)

Hemophilia B (4)

Hereditary Angioedema (2)

Hereditary Pulmonary Alveolar Proteinosis (1)

Herpes Simplex Virus Type I Stromal Keratitis (1)

Huntington's Disease, Early Manifest (2)

Hypertrophic Cardiomyopathy (1)

Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome (IPEX) (1)

Inherited Retinal Disease Associated With RPE65 Mutations (1)

Inherited Retinal Dystrophy Associated With RPE65 Mutations (1)

Krabbe Disease (1)

Leber Congenital Amaurosis - Type 1 (1)

Leber Congenital Amaurosis - Type 5 (1)

Leber Congenital Amaurosis-Type 2 (2)

Leber Hereditary Optic Neuropathy (mt-ND1) (1)

Leber Hereditary Optic Neuropathy (mt-ND4) (2)

Leukocyte Adhesion Defect - Type I (1)

Limb-Girdle Muscular Dystrophy Type 2B/R2 (1)

Limb-Girdle Muscular Dystrophy, Type 2C/R5 (1)

Limb-Girdle Muscular Dystrophy, Type 2D/R3 (1)

Limb-Girdle Muscular Dystrophy, Type 2E/R4 (1)

Limb-Girdle Muscular Dystrophy, Type 2I/R9 (2)

Mucopolysaccharidosis Type I (Hurler Syndrome) (1)

Mucopolysaccharidosis Type II (Hunter Syndrome) (1)

Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome) (2)

Mucopolysaccharidosis Type IV (1)

Multiple System Atrophy, Parkinsonian Subtype (1)

Myotubular Myopathy, X-Linked (1)

NGLY1 Deficiency (1)

Neovascular (Wet) Age-Related Macular Degeneration (12)

Ornithine Transcarbamylase Deficiency (1)

Ornithine Transcarbamylase Deficiency, Late Onset (1)

Ornithine Transcarbamylase Deficiency, Neonatal Onset (1)

Osteoarthritis, Knee (3)

Parkinson's Disease (3)

Phenylketonuria (2)

Primary Hyperoxaluria, Type I (1)

Pyruvate Kinase Deficiency (1)

Radiation-induced Xerostomia (1)

Refractory Angina (1)

Refractory Hypercholesterolemia (1)

Retinitis Pigmentosa (8)

Retinitis Pigmentosa (1)

Retinitis Pigmentosa, Non-Syndromic (1)

Retinitis Pigmentosa, X-Linked (4)

Retinoschisis, X-linked (4)

Rett Syndrome (2)

Severe Combined Immunodeficiency, ADA-deficient (1)

Severe Combined Immunodeficiency, DCLRE1C Deficient (2)

Severe Combined Immunodeficiency, X-linked (2)

Sickle Cell Disease (8)

Spastic Paraplegia Type 50 (SPG50) (1)

Stargardt Disease (2)

Stargardt Disease (Juvenile Macular Degeneration) (2)

Temporal Lobe Epilepsy (1)

Transthyretin Amyloidosis with Cardiomyopathy/Polyneuropathy (1)

Transthyretin Amyloidosis, with Polyneuropathy/Cardiomyopathy (1)

Wilson's Disease (1)

Wiskott-Aldrich Syndrome (1)

Status

Active not recruiting (52)

Completed (12)

Not yet recruiting (18)

Recruiting (121)

Terminated (1)

Phases

Early phase1 (15)

Phase1 (155)

Phase2 (138)

Phase3 (19)

Standard Ages

Adult (175)

Child (93)

Older adult (140)

Therapy Type

Gene editing (26)

Gene transfer (178)

Therapy Route

Ex-vivo (39)

In-vivo (165)

Drug Product Type

Autologous cells (37)

MRNA, LNP (10)

Viral vector (157)

Delivery System

Electroporation (5)

Lipid encapsulation (11)

Viral transduction (187)

Sponsor Class

Industry (152)

NIH (3)

Other (48)

Other gov (1)

Sponsor

4D Molecular Therapeutics (6)

ASC Therapeutics (1)

AbbVie (2)

Abeona Therapeutics, Inc (1)

Adrenas Therapeutics Inc (1)

Adverum Biotechnologies, Inc. (1)

Akouos, Inc. (1)

Ascidian Therapeutics, Inc (1)

Asklepios Biopharmaceutical, Inc. (4)

Aspa Therapeutics (1)

Assistance Publique - Hôpitaux de Paris (2)

Astellas Gene Therapies (3)

Atamyo Therapeutics (2)

Atsena Therapeutics Inc. (2)

AviadoBio Ltd (1)

Bacchetta, Rosa, MD (1)

Bayer (1)

Be Biopharma (1)

Beacon Therapeutics (1)

Beam Therapeutics Inc. (2)

Benjamin Greenberg (1)

Brain Neurotherapy Bio, Inc. (1)

Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio) (1)

Chengdu Origen Biotechnology Co., Ltd. (2)

Children's Hospital Medical Center, Cincinnati (1)

Children's Hospital of Fudan University (1)

Children's Hospital of Philadelphia (1)

CorrectSequence Therapeutics Co., Ltd (1)

David Williams (2)

Donald B. Kohn, M.D. (1)

Editas Medicine, Inc. (2)

Elpida Therapeutics SPC (1)

Encoded Therapeutics (1)

Essen Biotech (1)

Excision BioTherapeutics (1)

Exegenesis Bio (1)

Expression Therapeutics, LLC (1)

First Affiliated Hospital of Guangxi Medical University (2)

Fondazione Telethon (2)

Forge Biologics, Inc (1)

Freeline Therapeutics (1)

Frontera Therapeutics (4)

GenSight Biologics (2)

Genascence Corporation (1)

Grace Science, LLC (1)

Great Ormond Street Hospital for Children NHS Foundation Trust (1)

Huashan Hospital (1)

HuidaGene Therapeutics Co., Ltd. (2)

ICM Co. Ltd. (1)

InnoVec Biotherapeutics Inc. (1)

Innostellar Biotherapeutics Co.,Ltd (2)

Institut National de la Santé Et de la Recherche Médicale, France (1)

Institute of Hematology & Blood Diseases Hospital, China (2)

Intellia Therapeutics (4)

Janssen Research & Development, LLC (2)

Kamau Therapeutics (1)

Kanglin Biotechnology (Hangzhou) Co., Ltd. (1)

Krystal Biotech, Inc. (3)

Krzysztof Bankiewicz (1)

Lexeo Therapeutics (3)

Mark Tuszynski (1)

Mark Walters, MD (1)

Medical College of Wisconsin (1)

MeiraGTx UK II Ltd (3)

MeiraGTx, LLC (2)

Myrtelle Inc. (1)

NGGT INC. (1)

Nanoscope Therapeutics Inc. (2)

National Human Genome Research Institute (NHGRI) (1)

National Institute of Allergy and Infectious Diseases (NIAID) (2)

Neuracle Genetics, Inc (1)

Neurogene Inc. (2)

Neurophth Therapeutics Inc (2)

Ocugen (3)

Opus Genetics, Inc (1)

Orchard Therapeutics (1)

PTC Therapeutics (1)

Pacira Pharmaceuticals, Inc (1)

Passage Bio, Inc. (2)

Peking Union Medical College Hospital (1)

Peking University Third Hospital (1)

Pfizer (1)

Prevail Therapeutics (2)

Prime Medicine, Inc. (1)

REGENXBIO Inc. (2)

Ray Therapeutics, Inc. (1)

Regeneron Pharmaceuticals (1)

RenJi Hospital (1)

Rocket Pharmaceuticals Inc. (5)

STZ eyetrial (2)

Sangamo Therapeutics (1)

Sanofi (1)

Sardocor Corp. (3)

Sarepta Therapeutics, Inc. (3)

Sensorion (1)

Shanghai BDgene Co., Ltd. (2)

Shanghai Belief-Delivery BioMed Co., Ltd (2)

Shanghai Jiao Tong University School of Medicine (1)

Shanghai Refreshgene Technology Co., Ltd. (1)

Shanghai Vitalgen BioPharma Co., Ltd. (4)

Shenzhen Second People's Hospital (1)

Skyline Therapeutics (US) Inc. (1)

Solid Biosciences Inc. (1)

SparingVision (1)

Spark Therapeutics, Inc. (1)

Suzhou Municipal Hospital (1)

SwanBio Therapeutics, Inc. (1)

Taysha Gene Therapies, Inc. (2)

Tenaya Therapeutics (2)

The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army (1)

Ultragenyx Pharmaceutical Inc (4)

UniQure Biopharma B.V. (3)

University College, London (3)

University of California, Los Angeles (2)

University of California, San Diego (1)

University of California, San Francisco (1)

University of Florida (1)

University of Manchester (1)

VegaVect, Inc. (1)

Verve Therapeutics, Inc. (3)

ViGeneron GmbH (1)

West China Hospital (3)

Xiamen Ophthalmology Center Affiliated to Xiamen University (1)

Xinhua Hospital, Shanghai Jiao Tong University School of Medicine (1)

XyloCor Therapeutics, Inc. (1)

YolTech Therapeutics Co., Ltd (1)

eyeDNA Therapeutics (1)

iECURE, Inc. (1)

uniQure France SAS (1)

Vector Type

AAV (16)

AAV A101 (1)

AAV C102 (1)

AAV R100 (1)

AAV SNY001 (1)

AAV-SLB101 (1)

AAV.7m8 (2)

AAV.SPR (1)

AAV1 (4)

AAV2 (18)

AAV2.5 (1)

AAV2.7m8 (4)

AAV2/5 (4)

AAV2/6 (2)

AAV2/8 (8)

AAV2/9 (1)

AAV2i8 (1)

AAV2tYF (1)

AAV5 (7)

AAV5.2 (1)

AAV6 (2)

AAV8 (21)

AAV843 (1)

AAV9 (29)

AAVOlig001 (1)

AAVS3 (1)

AAVhu37 (1)

AAVhu68 (1)

AAVrh.74 (1)

AAVrh10 (6)

AAVrh74 (4)

Ad5 (2)

Anc80L65 (1)

Chim.hCD18-LV (1)

HIV (1)

HSV-1 (3)

LDLR (3)

LDLR + GalNAc (2)

LK03 (2)

LNP (6)

LV (24)

LZRSE (1)

PGK-coPRK-WPRE (1)

RV (1)

VSV-G (4)

dual AAV1 (1)

none (4)

scAAV9 (1)

Editor Type

ABE (2)

ABE8 (1)

ABE8.8m (1)

ABE8e-SpRY (1)

ARCUS (1)

ASCas12a (1)

ASCas12a mRNA (1)

Cas9 (1)

Cas9 RNP (1)

Cas9 mRNA (8)

SpCas9 mRNA (1)

SpRY-CBE (1)

YolCas12 (1)

base editor (1)

eTAM (1)

hfCas13Y (1)

none (178)

prime editor (1)

transformer BE RNP (1)

Target Tissue or Cell

Airway epithelial cells (1)

B cells (1)

Bipolar cells (2)

CD34+ cells (34)

CD4+ T cells (1)

Cardiomyocyte (2)

Cone cells (3)

Entorhinal cortex & hippocampus (1)

GABAergic inhibitory interneurons (1)

Hair cell (2)

Hepatocyte (7)

Keratinocytes (2)

Liver (4)

Lung (1)

Megakaryocytes (1)

Muscle cells (1)

Oligodendrocytes (1)

Photoreceptors (1)

Putamen (1)

Retinal ganglion (2)

Striatum (2)

Substantia nigra & ventral tegmental area (1)

Target Gene or Variant

(HBA2):c.427T>C (p.Ter143Gln) (1)

ABCA4 (1)

ABCA4 pre-mRNA (1)

ABCD1 (2)

ABD-VEGFR (1)

ADA (1)

ANGPTL3 (1)

AP4M1 (1)

APOE2 (1)

AQP1 (1)

ARSB (1)

ASPA (2)

ATP7B (1)

Aflibercept (2)

Anti-VEGF (5)

BCL11A (2)

BDNF (1)

CD59 (1)

CFTR (2)

CHM (1)

CLN5 (1)

CNGA1 (1)

CNGA3 (2)

CNGB3 (1)

COL7A1 (2)

CSF2RA (1)

CTNS (1)

CYBB (1)

CYBB c.676 C>T (1)

CYP21A2 (1)

CYP46A1 (1)

CYP4V2 (2)

ChR-tdT (1)

ChR2 (1)

Codon optimized ChR-3M (1)

Codon optimized aflibercept (2)

DCLRE1C (2)

DDC (4)

DMD (1)

DYSF (1)

F8 (9)

F9 (4)

FANCA (1)

FKRP (2)

FOXP3 (1)

FXN (2)

G6PC (1)

GAA (4)

GAD1/2 (1)

GALC (1)

GAN (1)

GBA1 (3)

GCDH (1)

GDNF (2)

GLA (4)

GLB1 (2)

GRN (3)

GUCY2D (1)

HAO1 (1)

HBA (1)

HBB (9)

HBG1/HBG2 (3)

HIV genes (1)

HSV genes (1)

IDS (1)

IDUA (1)

IL1RA (1)

IL1RN (1)

IL2RG (2)

ITGB2 (1)

KCNA1 (1)

KLKB1 (2)

LAMP2B (1)

LCA5 (1)

LDLR (1)

MCO (2)

MECP2 (1)

MFSD8 (1)

MTM1 (1)

MYBPC3 (1)

MiGRIK2 (1)

MiHTT (1)

MiSOD1 (1)

MiVEGFC (1)

Micro-dystrophin (3)

MiniMECP2 (1)

NCF1 (1)

NCF1 (c.75_76delGT) (1)

ND1G3460A (1)

ND4G11778A (2)

NGLY1 (1)

NKX3-2 (1)

NR2E3 (1)

NXNL1 (1)

OTC (3)

OTOF (3)

PAH (2)

PCSK9 (2)

PDE6A (1)

PDE6B (1)

PKLR (1)

PKP2 (2)

PKP2A (1)

PPP1R1A (1)

RHO (1)

RORA (2)

RPE65 (4)

RPGR (3)

RPGRORF15 (1)

RS1 (4)

SCN1A (1)

SERCA2a (3)

SERPINA1 (2)

SERPINA1 (p.Glu366Lys) (1)

SGCA (1)

SGCB (1)

SGCG (1)

SGSH (2)

TGM1 (1)

TTR (2)

VEGF (1)

VEGF-trap (1)

VEGFA mRNA (1)

VEGFR1/R2 fusion protein (1)

WAS (1)

Route of Administration

Broncoscopy (1)

CED (2)

Inhalational (3)

Intraarterial (4)

Intraarticular (3)

Intracerebroventricular (7)

Intracisterna magna (5)

Intracochlear (3)

Intracranial (1)

Intramuscular (1)

Intramyocardial (1)

Intraocular (1)

Intraparenchymal (9)

Intrastromal (1)

Intrathalamic (1)

Intrathecal (7)

Intravenous (97)

Intravitreal (21)

Isolated limb infusion (1)

Parotid (1)

Skin graft (2)

Subretinal (30)

Suprachoroidal (1)

Topical (1)

Mechanism of Action

Exon skipping/splice editor (3)

Functional gene replacement (127)

Gene excision (2)

Gene inactivation (10)

Genetic delivery of therapeutic protein (13)

MiRNA knockdown of mutant/aberrant gene (3)

Mutation correction (7)

Overexpression of protective allele/gene (39)

Locations

Argentina (2)

Australia (10)

Belgium (3)

Brazil (7)

Bulgaria (1)

Canada (19)

China (41)

Czechia (1)

Denmark (5)

France (20)

Germany (16)

Greece (1)

Hungary (1)

Israel (4)

Italy (14)

Japan (3)

Korea (1)

Netherlands (9)

New Zealand (5)

Poland (3)

Portugal (4)

Puerto Rico (3)

Republic of (1)

Saudi Arabia (1)

Singapore (1)

Spain (14)

Sweden (3)

Switzerland (2)

Taiwan (5)

Turkey (5)

United Kingdom (41)

United States (137)

Showing all 204 results ...

Trial ID	Indication	Compound Name	Sponsor	Funder Type	Target Gene/Variant	Therapy Type	Therapy Route	Mechanism of Action	Route of Administration	Drug Product Type	Target Tissue/Cell	Delivery System	Vector Type	Editor Type	Dose 1	Dose 2	Dose 3	Dose 4	Dose 5	Current Stage	Status	Submit Date	Completion Date	Last_Update	Eligibility Age	Enrollment Count	No. of Trial Sites	Locations	Has US IND	Patents	Recent Updates	Resources/Links
Trial ID	Indication	Compound Name	Sponsor	Funder Type	Target Gene/Variant	Therapy Type	Therapy Route	Mechanism of Action	Route of Administration	Drug Product Type	Target Tissue/Cell	Delivery System	Vector Type	Editor Type	Dose 1	Dose 2	Dose 3	Dose 4	Dose 5	Current Stage	Status	Submit Date	Completion Date	Last_Update	Eligibility Age	Enrollment Count	No. of Trial Sites	Locations	Has US IND	Patents	Recent Updates	Resources/Links
NCT04483440	Choroideremia	4D-110	4D Molecular Therapeutics	Industry	CHM	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV R100		3 x 10^11 vg/eye	1 x 10^12 vg/eye				Phase1	Active not recruiting	2020-07-20	2027-06	2024-05-09	>= 18 Years	13	2	United States	True			News and Press Releases : https://ir.4dmoleculartherapeutics.com/news-releases/news-release-details/4d-molecular-therapeutics-reports-full-year-2021-financial
NCT04517149	Retinitis Pigmentosa, X-Linked	4D-125	4D Molecular Therapeutics	Industry	RPGR	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV		3 x 10^11 vg/eye	1 x 10^12 vg/eye				Phase2, Phase1	Active not recruiting	2020-08-14	2029-05	2024-01-12	>= 12 Years	21	7	United States	True	US11613766B2; US20220290181A1	FDA granted Fast Track designation 1/10/22	News and Press Releases : https://ir.4dmoleculartherapeutics.com/news-releases/news-release-details/4d-molecular-therapeutics-announces-fda-fast-track-designation Clinical Publications : https://ir.4dmoleculartherapeutics.com/news-releases/news-release-details/4d-molecular-therapeutics-presents-interim-results-ongoing-4d
NCT05248230	Cystic Fibrosis, Modulator-Ineligible/Intolerant	4D-710 (A101-CMV173-coCFTRΔR)	4D Molecular Therapeutics	Industry	CFTR	Gene transfer	In-vivo	Functional gene replacement	Inhalational	Viral vector	Airway epithelial cells	Viral transduction	AAV A101		2.5 x 10^14 vg	5 x 10^14 vg	1x10^15 vg	2 x 10^15 vg (discontinued due to high transduction to interstitium)	MTD: 1 x 10^15 vg	Phase2, Phase1	Recruiting	2022-02-10	2030-01	2024-10-30	>= 18 Years	40	16	United States	True	US11499166B2; WO2023172873A2	Phase III Study Initiation planned H2 2025	News and Press Releases : https://ir.4dmoleculartherapeutics.com/news-releases/news-release-details/4dmt-announces-update-regulatory-interactions-and-development Clinical Publications : https://ir.4dmoleculartherapeutics.com/news-releases/news-release-details/4dmt-presents-positive-interim-data-phase-12-aerow-clinical-0 https://ir.4dmoleculartherapeutics.com/4DMT-Corporate-Presentation_2024 https://4dmoleculartherapeutics.com/wp-content/uploads/Presentation_ECFS-2024_Taylor-Cousar_AEROW-4D-710-CFTR-Expression_Final.pdf
NCT05930561	Diabetic Macular Edema	4D-150	4D Molecular Therapeutics	Industry	MiVEGFC	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Intravitreal	Viral vector		Viral transduction	AAV		Dose range: 5 x 10^9 -3 x 10^10 vg/eye	Planned Phase III dose: 3x10^10 vg/eye				Phase2	Recruiting	2023-06-26	2028-07-01	2023-11-13	>= 18 Years	72	15	Puerto Rico, United States	True		Interim Phase 2 analysis: Q4 2024	News and Press Releases : https://ir.4dmoleculartherapeutics.com/4DMT-Corporate-Presentation_2024 Clinical Publications : https://ir.4dmoleculartherapeutics.com/static-files/a324e2da-8ad5-474b-b894-24edd77ffdcb
NCT04519749	Fabry Disease With or Without Cardiac Involvement	4D-310	4D Molecular Therapeutics	Industry	GLA	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector	Cardiomyocyte	Viral transduction	AAV C102		1 x 10^13 vg/kg					Phase2, Phase1	Active not recruiting	2020-08-14	2030-06	2024-04-08	>= 18 Years	18	4	United States	True	WO2021222094A1		News and Press Releases : https://ir.4dmoleculartherapeutics.com/4DMT-Corporate-Presentation_2024 Clinical Publications : https://4dmoleculartherapeutics.com/wp-content/uploads/Thomas_02.09.2024_4D-310fabrydisease_FINAL_06Feb2024.pdf
NCT05197270	Neovascular (Wet) Age-Related Macular Degeneration	4D-150	4D Molecular Therapeutics	Industry	Anti-VEGF	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Intravitreal	Viral vector		Viral transduction	AAV		1 x 10^10 vg/eye	3 x 10^10 vg/eye	Planned Phase III dose: 3x10^10 vg/eye			Phase2, Phase1	Recruiting	2022-01-05	2026-11	2024-11-05	>= 50 Years	215	24	Puerto Rico, United States	True	US11766489B2; US11613766B2; US20240226336A9	Phase III trial to begin Q1 2025	News and Press Releases : https://ir.4dmoleculartherapeutics.com/4DMT-Corporate-Presentation_2024 Clinical Publications : https://ir.4dmoleculartherapeutics.com/static-files/a324e2da-8ad5-474b-b894-24edd77ffdcb https://4dmt.gcs-web.com/static-files/eb8b1b3c-971d-4f8d-8961-1e33063b6414
NCT04676048	Hemophilia A	ASC618 (HCB promotor)	ASC Therapeutics	Industry	F8	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV2/8		Undisclosed dose 1					Phase2, Phase1	Recruiting	2020-12-15	2026-12	2023-02-01	>= 18 Years	12	1	United States	True		First patient dosed January 2024	Preclinical Publications : PMID: 29552578 PMID: 26015976 https://www.asctherapeutics.com/wp-content/uploads/2021/10/ASGCT-2020-poster_final.pdf News and Press Releases : https://www.asctherapeutics.com/the-news-post/
NCT02556736	Retinitis Pigmentosa	RST-001 (AGN-151597)	AbbVie	Industry	ChR2	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravitreal	Viral vector	Retinal ganglion	Viral transduction	AAV2.7m8		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Phase2, Phase1	Completed	2015-09-21	2024-10-21	2024-11-08	>= 18 Years	14	4	United States				Protocol : https://cdn.clinicaltrials.gov/large-docs/36/NCT02556736/Prot_000.pdf https://cdn.clinicaltrials.gov/large-docs/36/NCT02556736/SAP_001.pdf
NCT04704921	Neovascular (Wet) Age-Related Macular Degeneration	ABBV-RGX-314	AbbVie	Industry	Anti-VEGF	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Subretinal	Viral vector		Viral transduction	AAV8		2.5 x 10^11 GC/eye	5.0 x 10^11 GC/eye	1.0 x 10^12 GC/eye			Phase3, Phase2	Recruiting	2021-01-08	2026-05	2024-04-26	50 Years - 89 Years	300	80	United States	True	US20230057519A1; US20200093939A1;	Pivotal trial initiation expected H1 2025; BLA/EMA submissions H1 2026	Preclinical Publications : PMID: 31408444 PMID: 29292162 News and Press Releases : https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-reports-first-quarter-2024-financial-results-and Clinical Publications : PMID: 38554726 https://www.regenxbio.com/getattachment/9463317c-3806-4434-aa79-898162f2665a/Barakat_03Nov23_ABBV-RGX-314-SCS-ALTITUDE-AAO-2023-FINAL_02Nov23.pdf?lang=en-US&ext=.pdf https://www.regenxbio.com/getattachment/09eea7b5-fa2a-4474-90e2-19d8670a6f6f/ABBV-RGX-314-SCSwAMD-AAVIATE_Pitcher_RHE2024_FINAL_approved-(1).pdf?lang=en-US&ext=.pdf
NCT05725018	Dystrophic Epidermolysis Bullosa	Prademagene zamikeracel (EB-101)	Abeona Therapeutics, Inc	Industry	COL7A1	Gene transfer	Ex-vivo	Functional gene replacement	Skin graft	Autologous cells	Keratinocytes	Viral transduction	LZRSE		Transduced 35cm^2 keratinocyte sheets, up to 6 wound sites					Phase3	Recruiting	2023-01-23	2025-06-30	2024-06-27	>= 12 Months	12	2	United States	True	US20240067926A1; US20230045590A1	BLA resubmission October 2024, new PDUFA date pending	Grant : R01 AR055914 Preclinical Publications : PMID: 25703736 PMID: 20497034 News and Press Releases : https://investors.abeonatherapeutics.com/press-releases/detail/291/abeona-therapeutics-completes-pz-cel-biologics-license https://investors.abeonatherapeutics.com/press-releases/detail/276/abeona-therapeutics-provides-regulatory-update-on-pz-cel Clinical Publications : PMID: 27802546 PMID: 36253825 PMID: 31578311
NCT04783181	Congenital Adrenal Hyperplasia	AAV5 BBP-631 (AAV5-CYP21A2)	Adrenas Therapeutics Inc	Industry	CYP21A2	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV5		1.5 x 10^13 vg/kg	3.0 x 10^13 vg/kg	6.0 x 10^13 vg/kg			Phase2, Phase1	Active not recruiting	2021-03-01	2029-02	2024-09-26	>= 18 Years	8	5	United States	True		Bridge Bio is cutting this program, lack of sufficient efficacy	Preclinical Publications : PMC7209390 https://adrenastx.com/wp-content/uploads/ASGCT-2021-Adrenas-14May21-Final.pdf News and Press Releases : https://bridgebio.com/news/bridgebio-pharma-reports-topline-results-from-phase-1-2-trial-of-investigational-gene-therapy-for-congenital-adrenal-hyperplasia-cah/ https://adrenastx.com/wp-content/uploads/CAH-Letter-to-Community-Q1-2023-Update-FINAL.pdf Protocol : https://adrenastx.com/wp-content/uploads/FINAL_POSTER_ENDO2021-03MAR2021.pdf
NCT05536973	Neovascular (Wet) Age-Related Macular Degeneration	Ixoberogene soroparvovec (ADVM-022)	Adverum Biotechnologies, Inc.	Industry	Codon optimized aflibercept	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Intravitreal	Viral vector		Viral transduction	AAV.7m8		6 x 10^10 vg/eye (selected as pivotal dose)	2 x 10^11 vg/eye				Phase2	Active not recruiting	2022-09-08	2028-11	2024-01-08	>= 50 Years	69	39	United States, United Kingdom, France	True	US20220265740A1; US20230322911A1	H1 2025: Initiation of Phase III trial	Grant : https://investors.adverum.com/news/news-details/2024/Adverum-Biotechnologies-to-Host-Webcast-to-Review-Clinical-Data-from-the-26-Week-Interim-Analysis-of-the-Ongoing-LUNA-Phase-2-Trial-in-Wet-AMD-Being-Presented-at-ASRS-Annual-Meeting/default.aspx Preclinical Publications : PMID: 30528929 News and Press Releases : https://investors.adverum.com/news/news-details/2024/Adverum-Biotechnologies-Reports-Second-Quarter-2024-Financial-Results-and-Provides-Program-and-Corporate-Highlights/default.aspx Clinical Publications : PMID: 38152412 https://adverum.com/wp-content/uploads/2022/03/AAO-2019-ADVM-022-OPTIC-Phase-1-Data.pdf https://adverum.com/wp-content/uploads/2024/07/Ixo-vec-IVT-Gene-Therapy-for-nAMD-First-Time-26-Week-IA-Results-from-the-Ph2-LUNA-Study-Jul24.pdf
NCT05821959	Congenital Hearing Loss	AAVAnc80-hOTOF	Akouos, Inc.	Industry	OTOF	Gene transfer	In-vivo	Functional gene replacement	Intracochlear	Viral vector	Hair cell	Viral transduction	Anc80L65		4.1 x 10^11 vg/cochlea	8.1 x 10^11 vg/cochlea				Phase2, Phase1	Recruiting	2023-02-08	2028-10	2024-10-30		14	5	United States, Taiwan, United Kingdom	True	US20210017235A1; US11104885B2; US20240011039A1; EP4063510A1		Preclinical Publications : PMID:36327973 PMID:28367981 https://akouos.com/wp-content/uploads/2023/02/Preclinical-Development-of-a-Genetic-Medicine-for-Otoferlin-Gene-mediated-Hearing-Loss-AK-OTOF.pdf News and Press Releases : https://investor.lilly.com/news-releases/news-release-details/positive-phase-12-clinical-trial-data-investigational-gene
NCT06467344	Stargardt Disease	ACDN-01	Ascidian Therapeutics, Inc	Industry	ABCA4 pre-mRNA	Gene editing	In-vivo	Exon skipping/splice editor	Subretinal	Viral vector		Viral transduction	AAV		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Phase2, Phase1	Recruiting	2024-05-31	2030-12-01	2024-10-15	>= 18 Years	13	10	United States	True	US20240091381A1; WO2020214990A1	Rare Pediatric Disease designation granted 4/25/24	Preclinical Publications : ASGCT 2024 Abstract #706 PMID: 38769406 News and Press Releases : https://ascidian-tx.com/wp-content/uploads/2024/04/FINAL-ACDN-01-ASGCT-2024-Curtain-Raiser_4.25.24.pdf Clinical Publications : ASGCT 2023, Abstract #351
NCT05230459	Limb-Girdle Muscular Dystrophy, Type 2I/R9	AB-1003 (LION-101)	Asklepios Biopharmaceutical, Inc.	Industry	FKRP	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV9		Undisclosed dose 1	Undisclosed dose 2				Phase2, Phase1	Recruiting	2022-01-27	2028-12	2024-04-03	18 Years - 65 Years	10	6	United States	True	US20200061092A1; US20190008881A1	First patient dosed (8/3/23)	Grant : R01 NS082536 Preclinical Publications : PMID: 37919902 PMID: 31296900 PMID: 28480302 PMID: 23817215 News and Press Releases : https://www.askbio.com/askbio-announces-first-patient-dosed-in-phase-1-phase-2-lgmd2i-r9/
NCT03533673	Glycogen Storage Disease Type 2 (Late-Onset Pompe Disease)	AAV2/8-LSPhGAA (ACTUS-101)	Asklepios Biopharmaceutical, Inc.	Industry	GAA	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector	Hepatocyte	Viral transduction	AAV2/8		1.6 x 10^12 vg/kg	Undisclosed dose 2	Undisclosed dose 3			Phase2, Phase1	Active not recruiting	2018-04-13	2026-03	2023-06-28	>= 18 Years	7	1	United States	True	US20180371440A1; US20220389399A1; US20230210884A1;	First patient dosed 1/22/19	Grant : R01 AR065873 U01 AR071693 Preclinical Publications : PMID: 35102744 PMID: 35081735 PMID: 31909086 PMID: 29187643 PMID: 26015962 PMID: 23514839 News and Press Releases : https://www.askbio.com/first-patient-dosed-with-gene-therapy-in-phase-1-2-study-of-actus-101-in-patients-with-pompe-disease/ Clinical Publications : PMID: 37701327 PMID: 36805083
NCT05598333	Congestive Heart Failure	AB-1002 (BNP116.sc-CMV.I1c)	Asklepios Biopharmaceutical, Inc.	Industry	PPP1R1A	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraarterial	Viral vector	Cardiomyocyte	Viral transduction	AAV2i8		3.25 x 10^13 vg	6.5 x 10^13 vg				Phase2	Recruiting	2022-10-25	2030-12-31	2024-10-10	>= 18 Years	150	28	United States	True	US20230340528A1; AU2021320244A1	Received Fast Track designation 4/18/24	Grant : P50 HL112324 P20 HL100396 R01 HL119046 R01 AR064369 P01 HL112761 R01 HL131404 R01 HL092130 Preclinical Publications : PMID: 23271792 PMID: 25023328 PMID: 28958332 PMID: 21775667 PMID: 29350681 News and Press Releases : https://www.askbio.com/askbio-receives-fda-fast-track-designation-for-ab-1002-investigational-gene-therapy-program-in-congestive-heart-failure/ Clinical Publications : https://www.escardio.org/static-file/Escardio/Advocacy/Initiatives/Cardiovascular%20Round%20Table/Documents/New%20Pharmacotherapy%2031Jan%20-1Feb%202024%20PPT/06%20Roger%20Hajjar%20ESC_PPT_The%20Revolution%20in%20Pharmacotherapy_2024%20HAJJAR.pdf
NCT06285643	Parkinson's Disease	AB-1005 (AAV2-GDNF)	Asklepios Biopharmaceutical, Inc.	Industry	GDNF	Gene transfer	In-vivo	Overexpression of protective allele/gene	CED	Viral vector		Viral transduction	AAV2		Up to 1.8mL (3.3 x 10^12 vg/ml/gadoteridol 2mM co-infusion)					Phase2	Recruiting	2024-02-14	2027-11-30	2024-10-26	45 Years - 75 Years	87	18	United States	True	US20180140672A1; US20180344199A1; WO2023183594A2	Received FDA Fast Track designation	Grant : ZIA NS003051 F32 NS064692 U54 NS045309 NIH/NHLBI K08 HL 146991 and NIH/NHLBI K08 HL 140078 CLIN2-11661 Preclinical Publications : PMID: 33131343 PMID: 29785638 PMID: 21343917 PMID: 21102559 PMID: 22132130 PMID: 20631185 PMID: 19671001 PMID: 19254173 PMID: 19203243 News and Press Releases : https://www.askbio.com/askbio-presents-18-month-phase-ib-trial-results-of-ab-1005-gene-therapy-for-patients-with-parkinsons-disease/ https://www.askbio.com/askbio-receives-fda-fast-track-and-mhra-innovation-passport-designations-for-ab-1005-investigational-gdnf-gene-therapy-for-parkinsons-disease/ https://www.askbio.com/askbio-welcomes-brain-neurotherapy-bio-to-expand-clinical-pipeline-for-neurodegenerative-diseases/ Clinical Publications : PMID: 38718138 PMID: 35957524 PMID: 31145831 https://index.mirasmart.com/AAN2024/SearchResults.php?Author_Institution=&Program_Number=&Topic=&Session_Name=&Author=Bankiewicz%2C+Krystof&Title= Protocol : https://www.nejm.org/doi/suppl/10.1056/NEJMoa2104205/suppl_file/nejmoa2104205_protocol.pdf
NCT04998396	Canavan Disease	AAV9 BBP-812 (AAV9-ASPA)	Aspa Therapeutics	Industry	ASPA	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV9		Undisclosed low dose	Undisclosed high dose	Undisclosed expansion dose			Phase2, Phase1	Recruiting	2021-08-05	2030-10-08	2024-10-22	<= 30 Months	26	4	United States	True			Grant : P01 AI100263 R01 NS076991 R01NS076991 Preclinical Publications : PMID: 23817205 PMID: 27039844 PMID:28194442 News and Press Releases : https://aspatx.com/#news Clinical Publications : PMID: 37601414
NCT03964792	Sickle Cell Disease	DREPAGLOBE	Assistance Publique - Hôpitaux de Paris	Other	HBB	Gene transfer	Ex-vivo	Overexpression of protective allele/gene	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells (range: 6 - 8.1 x 10^6 cells/kg)					Phase2, Phase1	Active not recruiting	2019-05-14	2024-01	2024-01-09	12 Years - 20 Years	6	1	France	False	US20200190536A1	Uses the same βAS3-globin as NCT02247843; vector performed poorly	Preclinical Publications : PMID: 30140714 Clinical Publications : https://ashpublications.org/blood/article/138/Supplement%201/1854/480806/Clinical-Results-of-the-Drepaglobe-Trial-for
NCT05071222	Severe Combined Immunodeficiency, DCLRE1C Deficient	EF1a-hArtemis LV (modified EF1a promotor) ARTEGENE	Assistance Publique - Hôpitaux de Paris	Other	DCLRE1C	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells					Phase2, Phase1	Recruiting	2021-08-27	2041-07-19	2023-11-27	<= 47 Months	5	1	France	False			Preclinical Publications : PMID: 31720302
NCT03199469	Myotubular Myopathy, X-Linked	Resamirigene bilparvovec (AT132), rAAV8-des-hMTM1	Astellas Gene Therapies	Industry	MTM1	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV8		1.3 x 10^14 vg/kg	3.5 x 10^14 vg/kg				Phase3, Phase2	Active not recruiting	2017-06-21	2030-03-31	2024-10-29	<= 5 Years	27	6	Canada, United States, France, Germany	True	US20220411819A1; WO2023196853A1; WO2022251208A1	On clinical hold since 2021	Grant : R21 AR064503 R01 AR044345 R01 HL115001 P50 NS040828 Preclinical Publications : PMID: 37490339 PMID: 33076971 PMID: 28370029 PMID: 28237839 PMID: 24861988 PMID: 24452262 News and Press Releases : https://www.astellas.com/en/news/25731 Clinical Publications : PMID: 38086156 PMID: 37977713 PMID: 37673065 PMID: 34641930 PMID: 34366366
NCT06483802	Friedreich Ataxia	ASP2016	Astellas Gene Therapies	Industry	FXN	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV8		Undisclosed dose escalation					Phase1	Not yet recruiting	2024-06-26	2031-01-31	2024-10-31	18 Years - 40 Years	14	5	United States	True	WO2023044424A1; AR127081A1; EP4401756A1
NCT04174105	Glycogen Storage Disease Type 2 (Late-Onset Pompe Disease)	Zocaglusagene nuzaparvovec (AT845)	Astellas Gene Therapies	Industry	GAA	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV8		3 x 10^13 vg/kg	6 x 10^13 vg/kg	1 x 10^14 vg/kg			Phase2, Phase1	Recruiting	2019-11-13	2029-11-30	2024-08-19	18 Years - 80 Years	18	4	United States, United Kingdom	True	US20210162073A1; US20220411819A1; US20220387562A1; WO2023150688A8		Preclinical Publications : PMID: 34850579 News and Press Releases : https://www.astellas.com/en/news/27331
NCT05224505	Limb-Girdle Muscular Dystrophy, Type 2I/R9	ATA-100 (GNT0006)	Atamyo Therapeutics	Industry	FKRP	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV2/9		9.0 x 10^12 vg/kg	2.7 x 10^13 vg/kg				Phase2, Phase1	Recruiting	2021-12-15	2030-10	2024-07-31	16 Years - 99 Years	39	3	Denmark, United Kingdom, France	False	US20230321277A1; EP4031673A1; EP4198047A1	FDA granted Fast Track designation 6/24/24	Preclinical Publications : PMID: 28334834 News and Press Releases : https://atamyo.com/wp-content/uploads/PR-June-24-2024-Atamyo-Therapeutics-Gets-FTD-for-ATA-100.pdf
NCT05973630	Limb-Girdle Muscular Dystrophy, Type 2C/R5	ATA-200	Atamyo Therapeutics	Industry	SGCG	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV2/8		1.0 x 10^14 vg/kg	3.0 x 10^14 vg/kg				Phase2, Phase1	Not yet recruiting	2023-07-24	2031-12-31	2024-09-19	6 Years - 13 Years	6	3	United States, Italy, France	False	EP4198134A1		Preclinical Publications : PMID: 31194043 News and Press Releases : https://atamyo.com/press-releases/the-dion-foundation-and-atamyo-therapeutics-announce-a-partnership-to-expand-into-the-us-atamyos-clinical-trial-of-ata-200-gene-therapy-to-treat-limb-girdle-muscular-dystrophy-type-2c-r5/
NCT05878860	Retinoschisis, X-linked	ATSN-201 (AAV.SPR-hGRK1-hRS1syn)	Atsena Therapeutics Inc.	Industry	RS1	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV.SPR		1.5 x 10^10 vg/eye	5.0 x 10^10 vg/eye	1.7 x 10^11 vg/eye			Phase2, Phase1	Recruiting	2023-05-12	2029-10	2024-07-30	>= 6 Years	21	4	United States	True		FDA granted Rare Pediatric Disease Designation	Preclinical Publications : https://atsenatx.com/wp-content/uploads/2023/12/ESGCT2023_INDXLRS-1.pdf News and Press Releases : Atsena Therapeutics – Atsena Therapeutics Receives Rare Pediatric Disease Designation from the U.S. FDA for ATSN-201 Gene Therapy to Treat X-linked Retinoschisis (atsenatx.com) Clinical Publications : https://www.atsenatx.com/wp-content/uploads/2024/07/Atsena_FFBInnovationsSummit_2024.pdf
NCT03920007	Leber Congenital Amaurosis - Type 1	ATSN-101 (AAV5-hGRK1-hGUCY2D)	Atsena Therapeutics Inc.	Industry	GUCY2D	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2/5		1.0 x 10^10 vg/eye (300ul)	3.0 x 10^10 vg/eye (300ul)	1.0 x 10^11 vg/eye (300ul); expansion dose			Phase2, Phase1	Active not recruiting	2019-04-10	2027-05-19	2024-02-20	>= 6 Years	15	2	United States	True		FDA granted Rare Pediatric Disease Designation, ODD, RMAT	Grant : R01 EY024280 News and Press Releases : https://atsenatx.com/press-release/atsena-therapeutics-receives-rare-pediatric-disease-designation-from-fda-for-atsn-101-gene-therapy-for-gucy2d-associated-leber-congenital-amaurosis/ Clinical Publications : PMID: 33997691 https://www.atsenatx.com/wp-content/uploads/2024/02/MaculaSociety2024Feb.pdf PMID: 39244273
NCT06064890	Frontotemporal Dementia	AAV.PGRN (AVB-101)	AviadoBio Ltd	Industry	GRN	Gene transfer	In-vivo	Functional gene replacement	Intrathalamic	Viral vector		Viral transduction	AAV9		Undisclosed low dose	Undisclosed high dose				Phase2, Phase1	Recruiting	2023-09-12	2030-10-31	2024-07-01	30 Years - 75 Years	9	10	Netherlands, United States, Poland, Spain	False		Partnership with Astellas for clinical development of this product announced	Preclinical Publications : https://aviadobio.com/wp-content/uploads/2024/07/AAIC-2024_AVB-101_FINAL_DIGITAL.pdf https://aviadobio.com/wp-content/uploads/2023/10/ESGCT2023.pdf https://aviadobio.com/wp-content/uploads/2023/10/ESGCT-2022.pdf News and Press Releases : https://aviadobio.com/astellas-and-aviadobio-announce-exclusive-option-and-license-agreement-for-gene-therapy-avb-101-targeting-frontotemporal-dementia-and-other-indications/ https://aviadobio.com/first-patient-treated-in-aspire-ftd-clinical-trial/
NCT05241444	Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome (IPEX)	CD4^LVFOXP3	Bacchetta, Rosa, MD	Other	FOXP3	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD4+ T cells	Viral transduction	LV		1.0 x 10^6 CD4+ cells/kg	3.0 x 10^6 CD4+ cells/kg	1.0 x 10^7 CD4+ cells/kg			Phase1	Recruiting	2022-01-12	2037-02	2024-07-08	4 Months - 35 Years	30	1	United States	True	US20220273712A1; EP3672617A4; US20230183804A1		Grant : R01 FD007540 CLIN2-13259 CLIN1-11591 Preclinical Publications : PMID: 38027059 PMID: 33304583 PMID: 24337481 News and Press Releases : https://primaryimmune.org/resources/news-articles/boy-ipex-treated-gene-therapy-stanford-university
NCT03588299	Hemophilia A	Peboctocogene camaparvovec (DTX201), liver-specific TTR promotor	Bayer	Industry	F8	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAVhu37		0.5 x 10^13 GC/kg	1 x 10^13 GC/kg	2 x 10^13 GC/kg	4 x 10^13 GC/kg		Phase2, Phase1	Active not recruiting	2018-07-06	2026-11-30	2024-10-17	>= 18 Years	11	13	Netherlands, United States, United Kingdom, Bulgaria, France, Germany	True			Preclinical Publications : PMID: 29890905 PMID: 28056565 News and Press Releases : https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-progress-across-broad-gene-therapy Clinical Publications : PMID: 36111754 https://ash.confex.com/ash/2020/webprogram/Paper139803.html https://ashpublications.org/blood/article/136/Supplement%201/44/471180/First-in-Human-Gene-Therapy-Study-of-AAVhu37
NCT06611436	Hemophilia B	BE-101 (Padua variant)	Be Biopharma	Industry	F9	Gene editing	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	B cells	Viral transduction	AAV6	Cas9 RNP	Undisclosed dose 1	Undisclosed dose 2	Undisclosed dose 3			Phase2, Phase1	Recruiting	2024-09-18	2027-05	2024-10-29	>= 18 Years	24	2	United States	True			Preclinical Publications : https://be.bio/wp-content/uploads/2023/12/R-Morgan-ASH-final_09Dec23.pdf
NCT04850118	Retinitis Pigmentosa, X-Linked	Laruparetigene zovaparvovec (AGTC-501)	Beacon Therapeutics	Industry	RPGR	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2tYF		6.8 x 10^11 vg/eye					Phase3, Phase2	Recruiting	2021-04-05	2029-10	2024-10-08	12 Years - 50 Years	75	13	United States, United Kingdom, Australia	True		First patient dosed in registrational trial 6/12/24	Grant : R01 EY017549 Preclinical Publications : PMID: 32414297 PMID: 31910043 PMID: 30280954 News and Press Releases : https://www.beacontx.com/news-and-events/beacon-therapeutics-treats-first-patient-in-vista-registrational-trial-for-agtc-501/ Clinical Publications : PMID: 32414297 PMID: 31910043 PMID: 30280954 https://www.beacontx.com/wp-content/uploads/2024/02/2024-02-07-Macula-Society-SKYLINE-M12-safety-efficacy-Mark-Pennesi-FINAL_website-1.pdf https://www.beacontx.com/wp-content/uploads/2024/09/2024-09-12-EURETINA-2024-HORIZON-36M-data-Paul-Yang-FINAL_website.pdf
NCT05456880	Sickle Cell Disease	BEAM-101	Beam Therapeutics Inc.	Industry	HBG1/HBG2	Gene editing	Ex-vivo	Mutation correction	Intravenous	Autologous cells	CD34+ cells	Electroporation	none	ABE8	Transduced CD34+ cells					Phase2, Phase1	Recruiting	2022-06-23	2027-02-01	2024-06-26	18 Years - 35 Years	15	15	United States	True	US20200399626A1; US11142760B2; US20230080198A1		Preclinical Publications : https://beamtx.com/media/esfjwtee/240522-eha-poster-beam-101-mfg-final-1.pdf https://beamtx.com/media/kmkew24k/202111-ash-applied-base-editing-to-treat-beta-hemoglobinopathies-vfinal.pdf https://beamtx.com/media/pgbjcxjv/2022_0627_faseb_v3_final.pdf News and Press Releases : https://investors.beamtx.com/static-files/a2f39903-04a5-4d95-8270-6ca6d5c47630 https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-reports-pipeline-updates-and-second-quarter-0
NCT06389877	Alpha 1-Antitrypsin Deficiency	BEAM-302	Beam Therapeutics Inc.	Industry	SERPINA1 (p.Glu366Lys)	Gene editing	In-vivo	Mutation correction	Intravenous	MRNA, LNP	Liver	Lipid encapsulation	LNP	ABE	Undisclosed low dose	Undisclosed intermediate dose	Undisclosed high dose			Phase2, Phase1	Recruiting	2024-04-22	2027-08	2024-10-31	18 Years - 70 Years	106	3	New Zealand, United Kingdom	True	US20200399626A1; US20210371858A1; US20230080198A1	First patient dosed 6/2024, initial clinical data expected 2025	Preclinical Publications : https://beamtx.com/media/1dljik4k/alpha-1-national-conference_june2024_final_presentation.pdf https://beamtx.com/media/deipu0tx/beam_esgct23-57.pdf https://beamtx.com/media/5jej4yvz/beam302_aatd-liver2023.pdf News and Press Releases : https://investors.beamtx.com/news-releases/news-release-details/beam-therapeutics-reports-pipeline-updates-and-second-quarter-0
NCT04737460	Batten Disease (Late Infantile Neuronal Ceroid Lipofuscinosis)	AAV9/CLN7	Benjamin Greenberg	Other	MFSD8	Gene transfer	In-vivo	Functional gene replacement	Intrathecal	Viral vector		Viral transduction	AAV9		5 x 10^14 vg	1 x 10^15 vg				Phase1	Active not recruiting	2021-01-15	2029-02-01	2023-11-28	1 Year - 18 Years	4	1	United States	True	WO2020033833A1; US11753655B2		Preclinical Publications : PMID: 35025759
NCT04680065	Multiple System Atrophy, Parkinsonian Subtype	AB-1005 (AAV2-GDNF)	Brain Neurotherapy Bio, Inc.	Industry	GDNF	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraparenchymal	Viral vector		Viral transduction	AAV2		Undisclosed dose 1					Phase1	Recruiting	2020-12-15	2028-12	2024-10-02	35 Years - 75 Years	9	6	United States	True	US20180140672A1; US20180344199A1; WO2023183594A2	First patient randomized 11/17/23	News and Press Releases : https://www.askbio.com/askbio-announces-first-patient-randomized-in-phase-1-trial-of-ab-1005-aav2-gdnf-gene-therapy-for-multiple-system-atrophy-parkinsonian-type-msa-p/
NCT05541627	Huntington's Disease, Early Manifest	AB-1001 (AAVrh10.CAG.hCYP46A1)	Brainvectis, a subsidiary of Asklepios BioPharmaceutical, Inc. (AskBio)	Industry	CYP46A1	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraparenchymal	Viral vector		Viral transduction	AAVrh10		4 x 10^8 vg/uL	1.1 x 10^9 vg/uL				Phase2, Phase1	Active not recruiting	2022-09-13	2029-12-31	2024-08-06	18 Years - 65 Years	5	1	France	False	WO2012049314A1		Preclinical Publications : PMID: 31286142 PMID: 26912634 News and Press Releases : https://www.askbio.com/brainvectis-a-subsidiary-of-askbio-receives-clearance-to-conduct-phase-i-ii-clinical-trial-in-france-for-its-novel-gene-therapy-for-early-stage-huntingtons-disease/ Clinical Publications : https://ehdn.org/ehdn2022-day3/ PMID: 37483021
NCT06458595	Neovascular (Wet) Age-Related Macular Degeneration	KH658	Chengdu Origen Biotechnology Co., Ltd.	Industry	Anti-VEGF	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Suprachoroidal	Viral vector		Viral transduction	AAV		Undisclosed dose 1					Phase2, Phase1	Not yet recruiting	2024-06-09	2026-12-28	2024-06-13	50 Years - 85 Years	44	1	China	False	WO2023236964A1		News and Press Releases : https://news.futunn.com/en/post/41568297/kanghong-pharmaceutical-002773-sz-clinical-trial-of-kh658-ophthalmic-injection?level=1&data_ticket=1725909982909790
NCT05657301	Neovascular (Wet) Age-Related Macular Degeneration	KH631	Chengdu Origen Biotechnology Co., Ltd.	Industry	VEGFR1/R2 fusion protein	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Intravitreal	Viral vector		Viral transduction	AAV8		Undisclosed dose 1	Undisclosed dose 2	Undisclosed dose 3	Undisclosed dose 4	Undisclosed dose 5	Phase1	Recruiting	2022-12-10	2027-09	2024-02-26	50 Years - 85 Years	25	7	United States	True	WO2022051537A1	1st patient dosed 11/20/23	Preclinical Publications : PMID: 37752703 News and Press Releases : https://www.biospace.com/chengdu-origen-and-vanotech-announce-first-patient-dosed-in-van-2201-phase-1-trial-of-gene-therapy-for-wet-age-related-macular-degeneration
NCT05761899	Hereditary Pulmonary Alveolar Proteinosis	Gene-Corrected Macrophages (SIN-LV/EFS/CSF2RA)	Children's Hospital Medical Center, Cincinnati	Other	CSF2RA	Gene transfer	Ex-vivo	Functional gene replacement	Broncoscopy	Viral vector	CD34+ cells	Viral transduction	LV		11.1 x 10^6 cells/kg ideal body weight					Phase2, Phase1	Recruiting	2023-02-28	2038-10-01	2024-02-28	>= 18 Years	3	1	United States	True	US20220315900A1; US20200199623A1	Licensed to Altius therapeutics	Grant : R01 HL118342 R01 HL136721 Preclinical Publications : PMID: 38596536 PMID: 31326401 PMID: 28854814 Protocol : PMID: 38722740
NCT06207552	Fabry Disease	BBM-F101	Children's Hospital of Fudan University	Other	GLA	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV		Undisclosed dose					Early phase1	Recruiting	2023-12-18	2029-06	2024-07-09	7 Years - 18 Years	6	1	China	False	WO2022222869A1
NCT06364774	Beta-Thalassemia, transfusion-dependent	CHOP-ALS20 (βA-T87Q-globin)	Children's Hospital of Philadelphia	Other	HBB	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells					Phase2, Phase1	Recruiting	2024-03-26	2027-12-31	2024-10-02	18 Years - 35 Years	12	1	United States	True			Preclinical Publications : PMID: 33515514 News and Press Releases : https://www.chop.edu/centers-programs/sickle-cell-and-red-cell-disorders-curative-therapy-center-cured
NCT06291961	Beta-Thalassemia Major	CS-101	CorrectSequence Therapeutics Co., Ltd	Industry	BCL11A	Gene editing	Ex-vivo	Overexpression of protective allele/gene	Intravenous	Autologous cells	CD34+ cells	Electroporation	none	transformer BE RNP	Transduced CD34+ cells					Phase1	Recruiting	2024-02-23	2025-07	2024-07-01	12 Years - 35 Years	8	3	China	False	US11384353B2; WO2020156575	Information on tBE system, IIT study given at ASGCT 2024	Preclinical Publications : PMID: 33972728 PMID: 37989316 News and Press Releases : https://www.correctsequence.com/news/shownews.php?id=412&lang=en Protocol : https://protocolexchange.researchsquare.com/article/pex-1433/v1
NCT05353647	Sickle Cell Disease	Autologous CD34+ HSC cells transduced with the lentiviral vector containing BCL11a-targeted shRNA	David Williams	Other	BCL11A	Gene transfer	Ex-vivo	Overexpression of protective allele/gene	Intravenous	Autologous cells	CD34+ cells	Viral transduction	VSV-G		Transduced CD34+ cells					Phase2	Recruiting	2022-04-21	2026-05	2024-09-19	13 Years - 40 Years	25	9	United States	True			Grant : CLIN2SCD-12031 R01 HL137848 Preclinical Publications : PMID: 26080908 PMID: 27599293 Clinical Publications : PMID: 33283990 Protocol : https://www.nejm.org/doi/suppl/10.1056/NEJMoa2029392/suppl_file/nejmoa2029392_protocol.pdf
NCT03311503	Severe Combined Immunodeficiency, X-linked	G2SCID lentiviral vector transduced CD34+ cells (rHIV_IL2RGcoG2SCID)	David Williams	Other	IL2RG	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	HIV		Transduced CD34+ cells (minimum dose: 2.5 x 10^6 cells/kg)					Phase2, Phase1	Active not recruiting	2017-10-12	2025-10-01	2024-05-06	<= 5 Years	12	6	United States, United Kingdom	True			Grant : U01 AI087628 R01 AI082020 U01AI125051 Clinical Publications : PMID: 30261899 PMID: 25295500
NCT02247843	Sickle Cell Disease	Lenti/G-βAS3-FB (contains anti-sickling mutations: T87Q, G16D, E22A)	Donald B. Kohn, M.D.	Other	HBB	Gene transfer	Ex-vivo	Overexpression of protective allele/gene	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells					Phase2, Phase1	Active not recruiting	2014-09-20	2025-12	2024-05-16	>= 18 Years	4	1	United States	True	WO2020168004A1	Uses the same βAS3-globin as NCT03964792	Grant : DR3-06945 P30 AI028697 Preclinical Publications : PMID: 33186538 PMID: 30533448 PMID: 30198339 PMID: 25681747 PMID: 23863630 Clinical Publications : PMID: 23863630
NCT05444894	Beta-Thalassemia, transfusion-dependent	Renizgamglogene autogedtemcel (EDIT-301)	Editas Medicine, Inc.	Industry	HBG1/HBG2	Gene editing	Ex-vivo	Overexpression of protective allele/gene	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV	ASCas12a mRNA	Min: 5.7 x 10^6 CD34+ cells/kg	Max: 11.9 x 10^6 CD34+ cells/kg				Phase2, Phase1	Recruiting	2022-06-27	2025-12	2024-01-29	18 Years - 35 Years	9	8	Canada, United States	True	WO2017160890A1	RMAT designation in 2023	News and Press Releases : https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-present-clinical-data-ruby-and-edithal-trials-0 Clinical Publications : https://ir.editasmedicine.com/static-files/dc33c7c9-a186-4136-ac7b-0de29a36a940
NCT04853576	Sickle Cell Disease	Renizgamglogene autogedtemcel (EDIT-301)	Editas Medicine, Inc.	Industry	HBG1/HBG2	Gene editing	Ex-vivo	Overexpression of protective allele/gene	Intravenous	Autologous cells	CD34+ cells			ASCas12a	Min: 2.9 x 10^6 CD34+ cells/kg	Max: 10.0 x 10^6 CD34+ cells/kg				Phase2, Phase1	Active not recruiting	2021-04-16	2025-08	2024-10-08	12 Years - 50 Years	45	24	Canada, United States	True	US12031132B2; US20200299661A1;	Paused development and initiated efforts to outlicense reni-cel; possible BLA submission by Q1 2025	Preclinical Publications : https://www.editasmedicine.com/wp-content/uploads/2019/12/ASH-2019-Poster.pdf https://www.editasmedicine.com/wp-content/uploads/2019/10/FINAL-Chang-et-al-EHA-2019-Poster.pdf https://www.editasmedicine.com/wp-content/uploads/2019/10/Editas-2019-CSHL-genome-egineering-FINAL.pdf News and Press Releases : https://ir.editasmedicine.com/static-files/fdb9bc2a-11f6-4b9e-b890-6cb6e2cebfdb https://ir.editasmedicine.com/static-files/6d28e1be-216b-432b-bc39-0acb094b8f54 https://ir.editasmedicine.com/news-releases/news-release-details/editas-medicine-announces-second-quarter-2024-results-and Clinical Publications : https://library.ehaweb.org/eha/2024/eha2024-congress/422389/rabi.hanna.reni-cel.the.first.ascas12a.gene-edited.cell.therapy.led.to.html?f=listing%3D0%2Abrowseby%3D8%2Asortby%3D1%2Asearch%3Dhanna https://www.editasmedicine.com/wp-content/uploads/2024/06/Reni-cel_RUBY_oral_presentation_EHA2024.pdf https://ir.editasmedicine.com/static-files/dc33c7c9-a186-4136-ac7b-0de29a36a940
NCT05518188	Spastic Paraplegia Type 50 (SPG50)	MELPIDA (scAAV9-AP4M1)	Elpida Therapeutics SPC	Industry	AP4M1	Gene transfer	In-vivo	Functional gene replacement	Intrathecal	Viral vector		Viral transduction	AAV9		1 x 10^15 vg					Phase2, Phase1	Recruiting	2022-08-24	2030-10-01	2024-10-08	4 Months - 10 Years	4	1	United States	True	WO2022226183A3	Phase III Study approved, begins August 2024	Preclinical Publications : PMID: 36951961 Clinical Publications : PMID: 38942994 PMID: 36951961 https://www.elpidatx.com/documents Protocol : https://www.elpidatx.com/documents
NCT05419492	Dravet Syndrome, SCN1A-Associated	ETX101 (rAAV9-reGABA-eTFSCN1A)	Encoded Therapeutics	Industry	SCN1A	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intracerebroventricular	Viral vector	GABAergic inhibitory interneurons	Viral transduction	AAV9		Undisclosed low dose	Undisclosed high dose				Phase2, Phase1	Recruiting	2022-06-10	2031-04	2024-08-21	6 Months - 35 Months	22	2	United States	True	US20230203531A1; US20200397917A1; US20220136009A1; US20220168449A1; US20220170910A1	US IND cleared February 2024	Preclinical Publications : PMID: 35435735 https://aesnet.org/abstractslisting/etx101--a-gabaergic-interneuron-selective-aav-mediated-gene-therapy-for-the-treatment-of-scn1apositive-dravet-syndrome--biodistribution-and-safety-in-non-human-primates News and Press Releases : https://encoded.com/press-releases/encoded-therapeutics-announces-us-ind-clearance-and-australian-cta-approval-for-dravet-syndrome-gene-therapy-candidate-etx101/
NCT06399107	Sickle Cell Disease	BAH243	Essen Biotech	Other	HBB	Gene transfer	Ex-vivo	Overexpression of protective allele/gene	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells					Phase2, Phase1	Recruiting	2024-04-29	2025-12-28	2024-11-05	2 Years - 90 Years	85	1	China	False
NCT05144386	HIV-1 Infection, subgroup B	EBT-101 (AAV9-SaCas9-GagD-LTR1)	Excision BioTherapeutics	Industry	HIV genes	Gene editing	In-vivo	Gene excision	Intravenous	Viral vector		Viral transduction	AAV9	Cas9	0.9 x 10^12 vg/kg	3.0 x 10^12 vg/kg				Phase1	Active not recruiting	2021-11-22	2025-05	2024-07-29	18 Years - 70 Years	6	3	United States	True	US20190367924A1; US20200392487A1; US20240139294A1	Safe, but does not provide long-term viral suppression	Grant : R01 MH115860 R01 MH110360 R01 HL114152 R56 AI143647 R56 AI150772 R01 DA042706 CLIN2-13310 R01 NS087971 Preclinical Publications : PMID: 37587230 PMID: 33247091 PMID: 31266936 PMID: 30195766 PMID: 28366764 PMID: 27194423 News and Press Releases : https://www.excision.bio/news/press-releases/detail/43/excision-biotherapeutics-announces-data-from-the-phase-12
NCT05903794	Neovascular (Wet) Age-Related Macular Degeneration	EXG102-031	Exegenesis Bio	Industry	ABD-VEGFR	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Subretinal	Viral vector		Viral transduction	AAV8		Undisclosed dose 1	Undisclosed dose 2				Phase1	Recruiting	2023-06-06	2025-12-31	2024-09-19	>= 50 Years	12	2	United States	True	US20240190943A1	FDA cleared IND 1/18/23	Preclinical Publications : https://iovs.arvojournals.org/article.aspx?articleid=2786761 News and Press Releases : https://exegenesisbio.com/wp-content/uploads/2023/01/Exegenesis-Bio-FDA-IND-Approval-PR-Jan-18-2023.pdf
NCT04418414	Hemophilia A	CD68-ET3	Expression Therapeutics, LLC	Industry	F8	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells					Phase1	Not yet recruiting	2020-05-28	2039-08	2024-02-20	>= 18 Years	7	0		True	WO2022165390A1		Grant : R01 HL135853 U54 HL141981 R01 HL092179 Preclinical Publications : PMID: 37452013 PMID: 36591257 PMID: 34141826 PMID: 32258210 PMID: 31585952 PMID: 26909355 PMID: 26015976
NCT05757245	Alpha-Thalassemia	GMCN-508A	First Affiliated Hospital of Guangxi Medical University	Other	HBA	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells					Phase1	Recruiting	2023-02-24	2030-12-31	2023-04-18	5 Years - 35 Years	5	1	China	False
NCT05762510	Beta-Thalassemia, transfusion-dependent	GMCN-508B	First Affiliated Hospital of Guangxi Medical University	Other	HBB	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells					Early phase1	Recruiting	2022-01-06	2030-10-31	2023-05-09	5 Years - 35 Years	5	1	China	False
NCT03837483	Wiskott-Aldrich Syndrome	Etuvetidigene autotemcel (formerly OTL-103)	Fondazione Telethon	Other	WAS	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells (4.4 - 14.5 x 10^6 cells/kg)					Phase3	Active not recruiting	2019-02-08	2027-09	2024-01-31	<= 65 Years	10	2	United States, Italy	True	EP3973996A1	Telethon intends to seek regulatory approval in Europe	Preclinical Publications : PMID: 22371846 PMID: 19259069 PMID: 15509508 News and Press Releases : https://www.pharmaceutical-technology.com/features/inside-the-effort-to-rescue-a-gene-therapy-for-a-rare-disease/?cf-view Clinical Publications : PMID: 37244942 PMID: 35075289 PMID: 30981783 PMID: 25898053 PMID: 25792466 PMID: 23845947 https://ashpublications.org/blood/article/134/Supplement_1/3346/423707/Lentiviral-Hematopoietic-Stem-and-Progenitor-Cell
NCT03173521	Mucopolysaccharidosis Type IV	AAV2/8.TBG.hARSB	Fondazione Telethon	Other	ARSB	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV2/8		6 x 10^11 gc/kg	2 x 10^12 gc/kg	6 x 10^12 gc/kg			Phase2, Phase1	Active not recruiting	2017-04-12	2025-09-30	2021-12-06	4 Years - 65 Years	9	2	Turkey, Italy	False	US20190343929A1		Preclinical Publications : PMID: 28932756 PMID: 24725025 Clinical Publications : PMID: 38319253
NCT05739643	Krabbe Disease	FBX-101	Forge Biologics, Inc	Industry	GALC	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAVrh10		1.6 x 10^13 gc/kg	Undisclosed dose 2				Phase2, Phase1	Recruiting	2023-02-13	2026-07	2024-08-09	<= 18 Years	9	3	United States	True	WO2018136710; WO2020132385A1	ASGCT 2024: Update given on Phase 1b data; dose escalation to highest dose approved, will start 2Q24	Grant : F32 NS093898 K99 HD096115 R01 NS096087 Preclinical Publications : PMID: 22850681 PMID: 37952085 PMID: 29316812 PMID: 26329589 PMID: 25533112 News and Press Releases : https://www.forgebiologics.com/forge-biologics-novel-aav-gene-therapy-fbx-101-for-patients-with-krabbe-disease-is-granted-uks-innovation-passport-designation/ Clinical Publications : https://annualmeeting.asgct.org/global/am24/asgct24_lba_final.aspx, Abstract #8
NCT05324943	Gaucher Disease, Type 1	FLT201	Freeline Therapeutics	Industry	GBA1	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAVS3		4.5 x 10^11 vg/kg					Phase1	Active not recruiting	2022-02-28	2025-01-31	2024-08-07	>= 18 Years	18	10	United States, Brazil, United Kingdom, Israel, Germany, Spain	True	EP4118200A2; US20220387558A1; US20220162642A1;	RMAT designation and PRIME designations granted Q2 2024; Phase 3 trial planned in 2025	Preclinical Publications : PMID:36631545 (used same AAV) News and Press Releases : https://spurtherapeutics.com/static/Spur-Therapeutics-ESGCT-Data-Release-10-23-24.pdf https://spurtherapeutics.com/static/Spur-Therapeutics-FLT201-Update-Press-Release-07-08-2024.pdf Clinical Publications : PMID: 35857660 (used same AAV) Protocol : https://www.tandfonline.com/doi/full/10.2217/frd-2022-0019
NCT06492876	Diabetic Macular Edema	FT-003	Frontera Therapeutics	Industry	Aflibercept	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Intravitreal	Viral vector		Viral transduction	AAV2.7m8		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Phase2, Phase1	Recruiting	2024-07-01	2028-11-15	2024-07-09	18 Years - 74 Years	78	1	China	False	US20230295243A1
NCT06492863	Neovascular (Wet) Age-Related Macular Degeneration	FT-003	Frontera Therapeutics	Industry	Aflibercept	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Intravitreal	Viral vector		Viral transduction	AAV2.7m8		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Phase2, Phase1	Recruiting	2024-07-01	2028-10-15	2024-07-09	50 Years - 80 Years	78	1	China	False	US20230295243A1		News and Press Releases : https://fronteratherapeutics.com/frontera-therapeutics-doses-first-patient-in-a-clinical-trial-of-ft-003-gene-therapy-for-the-treatment-of-wet-amd/
NCT05858983	Inherited Retinal Dystrophy Associated With RPE65 Mutations	FT-001 (AAV2-hRPE65)	Frontera Therapeutics	Industry	RPE65	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2		1.5 x 10^10 vg/eye	7.5 x 10^10 vg/eye	15 x 10^10 vg/eye			Phase2, Phase1	Recruiting	2023-04-29	2029-11-30	2023-05-15	8 Years - 45 Years	9	1	China	False	US20230321280A1		News and Press Releases : https://fronteratherapeutics.com/frontera-therapeutics-initiates-phase-ii-clinical-trial-for-ft-001-in-hereditary-retinopathy-treatment/ Clinical Publications : https://iovs.arvojournals.org/article.aspx?articleid=2799447&resultClick=1
NCT06492850	Retinitis Pigmentosa, X-Linked	FT-002 (AAV5-GRK1-hRPGRORF15)	Frontera Therapeutics	Industry	RPGRORF15	Gene transfer	In-vivo	Functional gene replacement	Intraocular	Viral vector	Photoreceptors	Viral transduction	AAV5		5 x 10^10 vg/eye	10 x 10^10 vg/eye	20 x 10^10 vg/eye			Phase2, Phase1	Recruiting	2024-07-01	2026-02-01	2024-07-09	8 Years - 45 Years	32	1	China	False	CA3186826A1		Preclinical Publications : https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=59880 News and Press Releases : https://fronteratherapeutics.com/frontera-therapeutics-unveils-preliminary-clinical-results-for-ft-002-injection-at-retinal-cell-and-gene-therapy-innovation-summit-2024/ Clinical Publications : https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=59895
NCT02652767	Leber Hereditary Optic Neuropathy (mt-ND4)	LUMEVOQ (lenadogene nolparvovec)	GenSight Biologics	Industry	ND4G11778A	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV2		9 x 10^10 vg in 90uL					Phase3	Completed	2016-01-07	2019-07-04	2022-07-29	>= 15 Years	39	7	United States, Italy, United Kingdom, France, Germany		WO2019241206A1	EAP due to commence Q3 2024, France only; MAA in UK goal in 2026	Preclinical Publications : PMID: 34729378 PMID: 26029714 News and Press Releases : https://www.gensight-biologics.com/wp-content/uploads/2024/07/Gensight_Newsletter_NEW_20240711-2.pdf Clinical Publications : PMID: 36496192 PMID: 36449262 PMID: 36350566 PMID: 35820885 PMID: 34415265 PMID: 33566264 PMID: 33298565
NCT03326336	Retinitis Pigmentosa, Non-Syndromic	GS030 (rAAV2.7m8-CAG-ChrimsonR-tdTomato)	GenSight Biologics	Industry	ChR-tdT	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravitreal	Viral vector	Retinal ganglion	Viral transduction	AAV2.7m8		5 x 10^10 vg/eye	1.5 x 10^11 vg/eye	5 x 10^11 vg/eye			Phase2, Phase1	Recruiting	2017-10-11	2025-12	2022-07-26	18 Years - 75 Years	15	3	United States, United Kingdom, France	True	US20240238613A1; US20240165198A1		Preclinical Publications : PMID: 34977267 PMID: 34131223 PMID: 33504896 Clinical Publications : PMID: 34031601 https://iovs.arvojournals.org/article.aspx?articleid=2776227&resultClick=1
NCT05835895	Osteoarthritis, Knee	GNSC-001 (CMV promotor)	Genascence Corporation	Industry	IL1RN	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraarticular	Viral vector		Viral transduction	AAV2.5		1 x 10^11 vg/knee	1 x 10^12 vg/knee	1 x 10^13 vg/knee			Phase1	Active not recruiting	2023-04-19	2029-05	2024-05-03	40 Years - 75 Years	67	9	United States	True	US20220016213A1	Initial data for Phase 1b trial expected Q4 2024	Grant : CLIN2-14265 R01 AR051085 R01 AR048566 Preclinical Publications : PMID: 35260797 PMID: 29869535 PMID: 2617025 News and Press Releases : https://www.prnewswire.com/news-releases/genascence-announces-initiation-of-phase-1b-clinical-trial-of-gnsc-001-gene-therapy-for-knee-osteoarthritis-oa-302022071.html Clinical Publications : https://www.liebertpub.com/doi/10.1089/hum.2023.29258.abstracts (Abstract #P553)
NCT06199531	NGLY1 Deficiency	GS-100	Grace Science, LLC	Industry	NGLY1	Gene transfer	In-vivo	Functional gene replacement	Intracerebroventricular	Viral vector		Viral transduction	AAV9		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Phase2, Phase1	Recruiting	2023-11-21	2028-01-31	2024-06-04	2 Years - 18 Years	12	2	United States	True	EP4329824A1	Selected for START program (6/3/24)	Preclinical Publications : PMID: 36320418 News and Press Releases : https://www.fastcompany.com/40490486/this-father-founded-a-medical-research-startup-to-save-his-kids-life https://finance.yahoo.com/news/grace-science-llc-selected-fda-120300702.html
NCT05207657	Chronic Granulomatous Disease, p47phox	PCHIM-p47	Great Ormond Street Hospital for Children NHS Foundation Trust	Other	NCF1	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells					Phase2, Phase1	Recruiting	2021-06-24	2029-04-01	2023-05-16	>= 23 Months	5	1	United Kingdom	False
NCT06178432	Glycogen Storage Disease Type 2 (Late-Onset Pompe Disease)	BBM-G102	Huashan Hospital	Other	GAA	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV		Undisclosed dose 1					Early phase1	Not yet recruiting	2023-11-30	2028-12	2023-12-21	>= 18 Years	6	1	China	False
NCT05906953	Inherited Retinal Disease Associated With RPE65 Mutations	HG004	HuidaGene Therapeutics Co., Ltd.	Industry	RPE65	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV9		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Phase2, Phase1	Recruiting	2023-05-09	2025-12	2024-09-19	6 Years - 50 Years	20	3	United States, China	True		FDA granted RPDD on 8/7/23	Preclinical Publications : ARVO 2024 Annual meeting, Abstract # 5091-A0348 News and Press Releases : https://www.huidagene.com/new/news/50 Clinical Publications : ARVO 2024 Annual Meeting, Abstract #3286
NCT06031727	Neovascular (Wet) Age-Related Macular Degeneration	HG202	HuidaGene Therapeutics Co., Ltd.	Industry	VEGFA mRNA	Gene editing	In-vivo	Gene inactivation	Subretinal	Viral vector		Viral transduction	AAV	hfCas13Y	Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Early phase1	Recruiting	2023-09-01	2025-06-27	2023-11-13	50 Years - 80 Years	12	2	China	False	WO2023185878A1	Preclinical/proof-of-concept data presented at ASGCT 2024	Preclinical Publications : ARVO 2024 Annual meeting, Abstract # 6517 PMID: 35953673, https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=59984 News and Press Releases : https://www.huidagene.com/new/news/43 Clinical Publications : ARVO 2024 Annual Meeting, Abstract #4357-A0076
NCT05454566	Osteoarthritis, Knee	ICM-203 (hNkx3.2-D2 CDS)	ICM Co. Ltd.	Industry	NKX3-2	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraarticular	Viral vector		Viral transduction	AAV5.2		6 x 10^12 vg	2 x 10^13 vg	6 x 10^13 vg			Phase2, Phase1	Not yet recruiting	2022-07-04	2026-11	2024-10-15	50 Years - 80 Years	18	0		True		Update on Phase 1/2a study given at ASGCT 2024	Clinical Publications : https://www.cell.com/action/showPdf?pii=S1525-0016%2824%2900237-5 (Abstract #146)
NCT06289452	Retinoschisis, X-linked	IVB102	InnoVec Biotherapeutics Inc.	Industry	RS1	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Early phase1	Recruiting	2024-02-25	2029-12-31	2024-05-07	>= 8 Years	18	1	China	False	WO2023143606A1
NCT06196840	Neovascular (Wet) Age-Related Macular Degeneration	LX102	Innostellar Biotherapeutics Co.,Ltd	Industry	VEGF-trap	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Subretinal	Viral vector		Viral transduction	AAV2		2 x 10^10 vg	Undisclosed dose 2				Phase2	Recruiting	2023-12-25	2029-05	2024-01-09	50 Years - 89 Years	50	3	China	False			Clinical Publications : https://iovs.arvojournals.org/article.aspx?articleid=2798798
NCT06196827	Leber Congenital Amaurosis-Type 2	LX101	Innostellar Biotherapeutics Co.,Ltd	Industry	RPE65	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2		Undisclosed low dose (0.3mL/eye)	Undisclosed high dose (0.3mL/eye)				Phase2, Phase1	Active not recruiting	2023-12-25	2027-12	2024-01-09	>= 6 Years	9	2	China	False	US11970519B2
NCT04186650	Dystrophic Epidermolysis Bullosa	EF1a-COL7A1-SIN retroviral vector transduced autologous skin	Institut National de la Santé Et de la Recherche Médicale, France	Other gov	COL7A1	Gene transfer	Ex-vivo	Exon skipping/splice editor	Skin graft	Autologous cells	Keratinocytes	Viral transduction	RV		Up to 6 grafts of 50 cm^2 each					Phase2, Phase1	Active not recruiting	2019-11-25	2027-06-09	2021-10-19	>= 18 Years	3	1	France	False	EP2766480B1; WO2022122900A1		Preclinical Publications : PMID: 26994967 PMID: 25381930 PMID: 20485266 News and Press Releases : https://cure-eb.org/research-portfolio/ebgraft/ Clinical Publications : PMID: 31557321 PMID: 24933565
NCT06238908	Hemophilia A	NGGT003	Institute of Hematology & Blood Diseases Hospital, China	Other	F8	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV8		4 x 10^11 vg/kg	1x10^12 vg/kg	2.5x10^12 vg/kg			Early phase1	Not yet recruiting	2024-01-26	2030-01-31	2024-02-02	>= 18 Years	6	1	China	False	CN116715752A
NCT05641610	Hemophilia B	ZS801	Institute of Hematology & Blood Diseases Hospital, China	Other	F9	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV		2.0 x 10^12 vg/kg	5.0 x 10^12 vg/kg	1.0 x 10^13 vg/kg			Phase2, Phase1	Not yet recruiting	2022-11-25	2028-12	2023-02-09	>= 18 Years	21	1	China	False
NCT06634420	Hereditary Angioedema	NTLA-2002	Intellia Therapeutics	Industry	KLKB1	Gene editing	In-vivo	Gene inactivation	Intravenous	MRNA, LNP	Hepatocyte	Lipid encapsulation	LDLR	Cas9 mRNA	50 mg					Phase3	Recruiting	2024-10-07	2027-09	2024-10-22	>= 18 Years	60	1	United States	True	US20230203480A1; US20240018496A1; WO2024011206A1	BLA submission planned 2026; Phase II study met all primary and secondary endpoints, selected pivotal dose	News and Press Releases : https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-second-quarter-2024-financial Clinical Publications : PMID: 39445704 PMID: 38294975 Protocol : https://www.nejm.org/doi/suppl/10.1056/NEJMoa2309149/suppl_file/nejmoa2309149_protocol.pdf
NCT06128629	Transthyretin Amyloidosis with Cardiomyopathy/Polyneuropathy	Nexiguran ziclumeran (NTLA-2001)	Intellia Therapeutics	Industry	TTR	Gene editing	In-vivo	Gene inactivation	Intravenous	MRNA, LNP	Hepatocyte	Lipid encapsulation	LNP	Cas9 mRNA	55mg					Phase3	Recruiting	2023-11-08	2028-04	2024-11-04	18 Years - 90 Years	765	60	Argentina, Singapore, United States, United Kingdom, Spain, New Zealand, Canada, Sweden, Taiwan, Denmark, Italy, Australia, France, Germany	True	US20230257747A1; WO2017173054A1; US20240076636A1; US20230287400A1; US20210087568A1; WO2020219876A1; US20220009878A1	Initiate Phase III study for ATTRv-PN by year end	Preclinical Publications : PMID: 29490262 News and Press Releases : https://ir.intelliatx.com/static-files/a57908a6-aaca-404d-9072-32471cd3d41e Clinical Publications : PMID: 34215024 PMID: 37928601 https://www.intelliatx.com/wp-content/uploads/Intellia_Follow_On-Dosing_PNS-2024_06.25.24.pdf Protocol : https://www.nejm.org/doi/suppl/10.1056/NEJMoa2107454/suppl_file/nejmoa2107454_protocol.pdf
NCT05120830	Hereditary Angioedema	NTLA-2002	Intellia Therapeutics	Industry	KLKB1	Gene editing	In-vivo	Gene inactivation	Intravenous	MRNA, LNP	Hepatocyte	Lipid encapsulation	LDLR	Cas9 mRNA	25 mg	50 mg	75 mg	50 mg (pivotal dose)		Phase2, Phase1	Active not recruiting	2021-11-03	2026-03-31	2024-09-19	>= 18 Years	37	9	New Zealand, Netherlands, United Kingdom, Australia, France, Germany	False	US20230203480A1; US20240018496A1; WO2024011206A1	BLA submission planned 2026; Phase II study met all primary and secondary endpoints, selected pivotal dose	News and Press Releases : https://ir.intelliatx.com/news-releases/news-release-details/intellia-therapeutics-announces-second-quarter-2024-financial Clinical Publications : PMID: 38294975 https://www.intelliatx.com/wp-content/uploads/Intellia-IR-EAACI-NTLA-2002-Presentation_6.3.24.pdf Protocol : https://www.nejm.org/doi/suppl/10.1056/NEJMoa2309149/suppl_file/nejmoa2309149_protocol.pdf
NCT06622668	Alpha 1-Antitrypsin Deficiency	NTLA-3001	Intellia Therapeutics	Industry	SERPINA1	Gene editing	In-vivo	Functional gene replacement	Intravenous	MRNA, LNP	Liver	Lipid encapsulation	LNP	Cas9 mRNA	Undisclosed low dose	Undisclosed intermediate dose	Undisclosed high dose			Phase2, Phase1	Recruiting	2024-09-30	2030-05	2024-10-30	18 Years - 75 Years	30	1	New Zealand	False
NCT04671433	Retinitis Pigmentosa, X-Linked	Botaretigene sparoparvovec (AAV2/5-RPGR)	Janssen Research & Development, LLC	Industry	RPGR	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2/5		2.0 x 10^10 vg/mL	2.0 x 10^11 vg/mL	2.0 x 10^12 vg/mL			Phase3	Active not recruiting	2020-11-05	2024-09-20	2024-09-19	>= 3 Years	97	27	Canada, Netherlands, Belgium, United States, Denmark, Italy, United Kingdom, Israel, France, Switzerland, Spain	True	WO2024079655A1; US11045558B2	Enrollment completed	Preclinical Publications : https://meiragtx.com/wp-content/uploads/2022/05/Human-retinal-organoid-vector-efficacy-Poster-ARVO-2022.pdf News and Press Releases : https://investors.meiragtx.com/news-releases/news-release-details/meiragtx-reports-first-quarter-2024-financial-and-operational Clinical Publications : PMID:38871269 https://investors.meiragtx.com/static-files/09375a9f-d98f-4b9d-b30c-bf1c89a2cc0f
NCT05811351	Geographic Atrophy	JNJ-1887 (AAVCAGsCD59)	Janssen Research & Development, LLC	Industry	CD59	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravitreal	Viral vector		Viral transduction	AAV2		3.56 x 10^11 vg/eye	1.071 x 10^12 vg/mL				Phase2	Active not recruiting	2023-03-07	2026-03-08	2024-10-24	>= 60 Years	305	162	Hungary, United States, Czechia, United Kingdom, Portugal, Switzerland, Spain, Canada, Netherlands, Sweden, Turkey, Belgium, Denmark, Poland, Italy, Australia, Germany	True	WO2023089564A1		Clinical Publications : PMID: 38909914 https://euretina.softr.app/amsterdam-abstract?recordId=recSojB3Cs4Bx4GBD https://iovs.arvojournals.org/article.aspx?articleid=2786197 https://retinalphysician.com/issues/2020/april/clinical-trial-download-data-on-a-gene-therapy-for-dry-and-wet-amd/
NCT04819841	Sickle Cell Disease	KMAU-001 (nulabeglogene autogedtemcel)	Kamau Therapeutics	Industry	HBB	Gene editing	Ex-vivo	Mutation correction	Intravenous	Autologous cells	CD34+ cells	Viral transduction	AAV6	Cas9 mRNA	Transduced CD34+ cells					Phase2, Phase1	Recruiting	2021-03-24	2027-07-31	2024-10-09	12 Years - 40 Years	15	3	United States	True	US11851652B2	Product was previously developed by Graphite Bio	Grant : R01 AI097320 R01 AI120766 R01 HL135607 R01 HL152314 Preclinical Publications : PMID: 37637384 PMID: 34135108 PMID: 30195800 PMID: 27820943 Clinical Publications : https://kamautx.com/wp-content/uploads/2023/12/ASH_poster_v3_1.pdf
NCT06280378	Beta-Thalassemia, transfusion-dependent	KL003 (βA-T87Q-globin)	Kanglin Biotechnology (Hangzhou) Co., Ltd.	Industry	HBB	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells					Phase2, Phase1	Not yet recruiting	2024-02-20	2027-05	2024-02-28	3 Years - 35 Years	41	2	China	False		Received NMPA approval on 1/3/24	News and Press Releases : https://www.kanglinbio.com/#/newsdetail?id=57&newsTypeId=15
NCT05504837	Cystic Fibrosis	KB-407	Krystal Biotech, Inc.	Industry	CFTR	Gene transfer	In-vivo	Functional gene replacement	Inhalational	Viral vector		Viral transduction	HSV-1		Undisclosed dose (single administration)	Undisclosed dose (2 administrations)	Undisclosed dose (4 administrations)			Phase1	Recruiting	2022-08-15	2024-07	2024-04-09	>= 18 Years	12	2	United States	True	WO2020163703A1; WO2021127524A1	Completed dosing in first two cohorts and on track to start third in Q2 2024	Preclinical Publications : https://ir.krystalbio.com/static-files/4daf5858-70fc-4193-93d4-6ccb85fb53ef https://ir.krystalbio.com/static-files/04c0187d-1f2f-439d-935e-20980ed6729d https://ir.krystalbio.com/static-files/efd3ab11-ea4a-4bf0-a5d5-66db0e8bc4e5 News and Press Releases : https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-announces-first-quarter-2024-financial-results Clinical Publications : https://ir.krystalbio.com/static-files/8b395c9d-3d0a-4e8e-be20-023ff9c475bb
NCT06049082	Alpha 1-Antitrypsin Deficiency	KB-408	Krystal Biotech, Inc.	Industry	SERPINA1	Gene transfer	In-vivo	Functional gene replacement	Inhalational	Viral vector	Lung	Viral transduction	HSV-1		10^9 PFU	10^10 PFU	10^11 PFU			Phase1	Recruiting	2023-09-15	2026-05	2024-05-06	18 Years - 70 Years	12	1	United States	True		Phase I Interim data expected 2H 2024	Preclinical Publications : https://ir.krystalbio.com/static-files/ca6beb29-db59-49ac-9738-b07378e5fadd https://ir.krystalbio.com/static-files/b8a30065-0028-4980-b676-3f05427959b6 News and Press Releases : https://ir.krystalbio.com/static-files/8b395c9d-3d0a-4e8e-be20-023ff9c475bb
NCT05735158	Autosomal Recessive Congenital Ichthyoses, TGM1-deficient	KB-105	Krystal Biotech, Inc.	Industry	TGM1	Gene transfer	In-vivo	Functional gene replacement	Topical	Viral vector		Viral transduction	HSV-1		Undisclosed single dose formulated as a topical gel, weekly application					Phase2	Not yet recruiting	2023-02-09	2024-04	2023-02-23	>= 6 Months	15	1	United States	True	WO2019200163A1; EP3377637B1; US11717547B2; US20200101123A1	Initiate Phase 2 cohort in 2024	News and Press Releases : https://ir.krystalbio.com/static-files/8b395c9d-3d0a-4e8e-be20-023ff9c475bb
NCT02852213	AADC Deficiency	AAV2-hAADC	Krzysztof Bankiewicz	Other	DDC	Gene transfer	In-vivo	Functional gene replacement	CED	Viral vector	Substantia nigra & ventral tegmental area	Viral transduction	AAV2		Dose: 1.3x10 11 vg (<160μL); concentration of 8.3x10^11 vg/mL	4.2 x 10^11 vg (160ul)	1.6 x 10^12 vg (60uL)	1.3 x 10^12 vg (500uL)		Phase1	Recruiting	2016-07-20	2031-07	2024-08-27	>= 24 Months	42	3	United States	True	US20230330267A1		Grant : R01 NS094292 R01 NS073940 1R01NS094292 5R01NS094292 Preclinical Publications : PMID: 25541617 PMID: 38198147 Clinical Publications : PMID: 34253733 Protocol : https://cdn.clinicaltrials.gov/large-docs/13/NCT02852213/Prot_SAP_002.pdf
NCT06109181	Arrhythmogenic Right Ventricular Cardiomyopathy	LX2020	Lexeo Therapeutics	Industry	PKP2	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAVrh10		Starting dose: 2.0 x10^13 gc/kg					Phase2, Phase1	Recruiting	2023-10-25	2027-02	2024-10-02	18 Years - 65 Years	10	4	United States	True		FTD and ODD granted 12/2023; cohort 1 interim data expected 2H 2024	Preclinical Publications : https://doi.org/10.1038/s44161-023-00370-3 News and Press Releases : https://ir.lexeotx.com/news-releases/news-release-details/lexeo-therapeutics-reports-fourth-quarter-and-full-year-2023
NCT05445323	Friedreich's Ataxia, with Cardiomyopathy	LX2006	Lexeo Therapeutics	Industry	FXN	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAVrh10		1.8 x 10^11 vg/kg	5.6 x 10^11 vg/kg	1.2 x 10^12 vg/kg			Phase2, Phase1	Recruiting	2022-06-23	2029-09	2024-05-17	18 Years - 50 Years	9	6	Canada, United States	True	WO2023150563A1	Fast Track designation granted 4/16/24; update given at ASGCT 2024	Grant : R33HL151355 Preclinical Publications : PMID: 38600238 PMID: 37166361 PMID: 32646255 News and Press Releases : https://ir.lexeotx.com/news-releases/news-release-details/lexeo-therapeutics-announces-positive-interim-phase-12-clinical
NCT03634007	Alzheimer's Disease	LX1001 (AAVrh.10hAPOE2-HA)	Lexeo Therapeutics	Industry	APOE2	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intrathecal	Viral vector		Viral transduction	AAVrh10		1.4 x 10^10 gc/mL CSF	4.4 x 10^10 gc/mL CSF	1.4 x 10^11 gc/mL CSF	1.4 x 10^14 gc (fixed dose)		Phase2, Phase1	Active not recruiting	2018-08-03	2024-11	2023-11-15	>= 50 Years	15	4	United States	True	WO2021108809A1; WO2024011237A1; WO2021076941A1; WO2021022208A1	Interim Phase 1/2 data readout (all cohorts) 2H 2024	Grant : R01 NS090934 R01 AG047644 Preclinical Publications : PMID: 29409358 PMID: 27318144 News and Press Releases : https://ir.lexeotx.com/news-releases/news-release-details/lexeo-therapeutics-reports-first-quarter-2024-financial-results
NCT05040217	Alzheimer's Disease \| Mild Cognitive Impairment	AAV2-BDNF	Mark Tuszynski	Other	BDNF	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraparenchymal	Viral vector	Entorhinal cortex & hippocampus	Viral transduction	AAV2		3 x 10^11 vg/mL	1 x 10^12 vg/mL				Phase1	Recruiting	2021-06-21	2027-10-01	2024-08-27	50 Years - 80 Years	12	2	United States	True	US6683058B1; US20030124095A1; WO2023196575A1		Grant : U01 AG043416 P01 AG010435 R01 AG066080 R01 AG071656 5R01AG066080 5R01AG071656 Preclinical Publications : PMID: 36309123 PMID: 29535375 PMID: 24068826 News and Press Releases : https://today.ucsd.edu/story/first-in-human-clinical-trial-to-assess-gene-therapy-for-alzheimers-disease Clinical Publications : PMID: 26302439 PMID: 15852017 PMID: 12212782
NCT04774536	Sickle Cell Disease	CRISPR_SCD001	Mark Walters, MD	Other	HBB	Gene editing	Ex-vivo	Mutation correction	Intravenous	Autologous cells	CD34+ cells	Electroporation	none	Cas9 mRNA	Transduced CD34+ cells (range: 3 - 20 x 10^6 cells/kg)					Phase2, Phase1	Recruiting	2021-02-17	2029-03-01	2024-09-20	12 Years - 35 Years	9	2	United States	True			Grant : CLIN2SCD-11722
NCT03818763	Hemophilia A	Pleightlet (Auto CD34+PBSCs -889ITGA2B-BDDFVIII-WPTS(MUT6)(VSVg))	Medical College of Wisconsin	Other	F8	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	Megakaryocytes	Viral transduction	VSV-G		Transduced CD34+ cells (not to exceed 20ml/kg body weight)					Phase1	Recruiting	2019-01-15	2033-05-01	2024-04-26	>= 18 Years	5	1	United States	True	WO2014066663A1	First patient enrolled in March 2022, only 2 patients dosed so far 8/16/24	Grant : R01 HL102035 R01 HL068138 R01 HL142791 Preclinical Publications : PMID: 34245090 PMID: 26787735 PMID: 24253479 PMID: 22632092 PMID: 16823491 PMID: 14675082 PMID: 10449749 https://www.platelettargetedtherapeutics.com/abstracts/NHF%20Poster%2001%20-%201%20NHF%20poster%2038.pdf News and Press Releases : https://www.platelettargetedtherapeutics.com/
NCT03001310	Achromatopsia	Entacingene turiparvovec (AAV8-hCARp.hCNGB3)	MeiraGTx UK II Ltd	Industry	CNGB3	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector	Cone cells	Viral transduction	AAV2/8		0.1 × 10^12 vg/mL; 0.5 mL	0.3 × 10^12 vg/mL; 0.5mL	0.6 × 10^12 vg/mL; 0.5mL	1.0 × 10^12 vg/mL; 0.5mL		Phase2, Phase1	Completed	2016-11-22	2019-10-25	2023-03-08	>= 3 Years	23	2	United States, United Kingdom	True			Preclinical Publications : PMID: 21576125 https://meiragtx.com/wp-content/uploads/2018/08/ESGCT-2017-CNGB3-poster-32-1.pdf News and Press Releases : https://meiragtx.com/research-development/pipeline/ Clinical Publications : PMID: 37172884 PMID: 35998912 Protocol : https://cdn.clinicaltrials.gov/large-docs/10/NCT03001310/Prot_000.pdf https://cdn.clinicaltrials.gov/large-docs/10/NCT03001310/SAP_001.pdf
NCT03758404	Achromatopsia	AAV-CNGA3 (AAV8-hG1.7p.co.CNGA3)	MeiraGTx UK II Ltd	Industry	CNGA3	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector	Cone cells	Viral transduction	AAV2/8		Undisclosed low dose	Undisclosed intermediate dose	Undisclosed high dose			Phase2, Phase1	Completed	2018-11-27	2021-06-10	2022-12-01	>= 3 Years	11	2	United States, United Kingdom	True			Preclinical Publications : https://meiragtx.com/wp-content/uploads/2019/04/ARVO-2019-CNGA3-Poster-Revised.pdf News and Press Releases : https://meiragtx.com/research-development/pipeline/ Clinical Publications : PMID: 35998912 Protocol : https://cdn.clinicaltrials.gov/large-docs/04/NCT03758404/Prot_000.pdf https://cdn.clinicaltrials.gov/large-docs/04/NCT03758404/SAP_001.pdf
NCT02781480	Leber Congenital Amaurosis-Type 2	Cevaretigene ritoparvovec (AAV2/5-RPE65P/OPTIRPRE65)	MeiraGTx UK II Ltd	Industry	RPE65	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2/5		1.0 x 10^11 vg/mL	3.0 x 10^11 vg/mL	1.0 x 10^12 vg/mL			Phase2, Phase1	Completed	2016-04-28	2018-12	2021-07-12	>= 3 Years	15	2	United States, United Kingdom	True	WO2024079655A1		Preclinical Publications : PMID: 27653967 https://meiragtx.com/wp-content/uploads/2018/08/ESGCT-2017-OPTIRPE65-poster-31-1.pdf Protocol : https://cdn.clinicaltrials.gov/large-docs/80/NCT02781480/Prot_SAP_000.pdf
NCT05603312	Parkinson's Disease	AAV-GAD	MeiraGTx, LLC	Industry	GAD1/2	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraparenchymal	Viral vector		Viral transduction	AAV2		1 x 10^12 vg/mL					Phase2, Phase1	Completed	2022-10-12	2024-09-06	2024-10-08	25 Years - 85 Years	14	6	United States	True	WO2024079655A1; WO2021119615A1; US8017385B2	Initiation of Phase III design discussions in H2 2024	Preclinical Publications : PMID: 12376704 News and Press Releases : https://investors.meiragtx.com/news-releases/news-release-details/meiragtx-reports-first-quarter-2024-financial-and-operational Clinical Publications : PMID: 30487248 PMID: 28405611 PMID: 25036712 PMID: 21419704 PMID: 18042721 PMID: 17586305
NCT05926765	Radiation-induced Xerostomia	AAV-hAQP1	MeiraGTx, LLC	Industry	AQP1	Gene transfer	In-vivo	Overexpression of protective allele/gene	Parotid	Viral vector		Viral transduction	AAV2		0.4 x 10^12 vg/gland	1.2 x 10^12 vg/gland				Phase2	Recruiting	2023-06-22	2025-07	2024-10-08	>= 18 Years	120	22	Canada, United States, United Kingdom	True	WO2024079655A1; WO2024079657A1	FDA in support of Phase II study as pivotal for BLA submission	News and Press Releases : https://investors.meiragtx.com/news-releases/news-release-details/meiragtx-reports-first-quarter-2024-financial-and-operational Clinical Publications : https://investors.meiragtx.com/static-files/ba644eb0-16b6-444f-bcb3-c6e9cb6fe4fa
NCT04833907	Canavan Disease	MYR101 (rAAV-Olig001-ASPA)	Myrtelle Inc.	Industry	ASPA	Gene transfer	In-vivo	Functional gene replacement	Intracerebroventricular	Viral vector	Oligodendrocytes	Viral transduction	AAVOlig001		3.7 x 10^13 vg					Phase2, Phase1	Recruiting	2021-03-24	2027-08-31	2024-04-11	3 Months - 60 Months	24	1	United States	True	US9636370B2; US10532110B2	Selected for START program 7/2024	Grant : R21 NS059518 R15 NS088763 R41 AG044890 Preclinical Publications : PMID: 33614826 PMID: 27628693 PMID: 27717881 PMID:22750077 PMID: 20040913 News and Press Releases : https://myrtellegtx.com/fda-selects-myrtelle-for-start-pilot-program/ https://myrtellegtx.com/now-available-myrtelle-asgct-symposium-presentation/
NCT06332807	Phenylketonuria	NGGT002	NGGT INC.	Industry	PAH	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV8		6.0 x 10^12 vg/kg	1.0 x 10^13 vg/kg	2.0 x 10^13 vg/kg			Phase2, Phase1	Recruiting	2024-02-07	2030-12-30	2024-10-02	18 Years - 55 Years	12	1	United States	True	WO2024094044A1	Recieved Orphan Drug Designation from the FDA in Jan 2023	Clinical Publications : https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=59381
NCT04945772	Retinitis Pigmentosa	Sonpiretigene isteparvovec (MCO-010)	Nanoscope Therapeutics Inc.	Industry	MCO	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravitreal	Viral vector	Bipolar cells	Viral transduction	AAV2		0.9 x 10^11 gc/eye	1.2 x 10^11 gc/eye				Phase2	Completed	2021-06-15	2024-01-18	2024-03-22	>= 18 Years	27	6	Puerto Rico, United States	True		Anticipated BLA filing Q1 2025	Grant : R01 EY028216 R01EY025717 R44 EY025905 Preclinical Publications : PMID:34418301 News and Press Releases : https://nanostherapeutics.com/2024/10/10/nanoscope-announces-plans-to-submit-bla-for-mco-010-to-treat-retinitis-pigmentosa/ Clinical Publications : https://abstracts.euretina.org/2024/ca24-2740-4044/r/rec9aVeQGG85rFV4N Ho A. Longitudinal BCVA analysis of low- or high-dose MCO-010 mutation agnostic optogenetic therapy for retinitis pigmentosa: 12-month results from a Phase 2b/3 randomized, sham-controlled, patient- and assessor-masked clinical trial (RESTORE). Presented at: Association for Research in Vision and Ophthalmology meeting May 5-9, 2024 Seattle PMID:34418301
NCT05417126	Stargardt Disease	Sonpiretigene isteparvovec (MCO-010)	Nanoscope Therapeutics Inc.	Industry	MCO	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravitreal	Viral vector	Bipolar cells	Viral transduction	AAV2		1.2 x 10^11 gc/eye					Phase2	Completed	2022-06-09	2023-09-28	2023-11-09	>= 16 Years	6	2	United States	True		Phase III Study expected to begin 2025	Grant : R01 EY028216 R01EY025717 R44 EY025905 Preclinical Publications : PMID:34418301 News and Press Releases : https://nanostherapeutics.com/2024/09/12/nanoscope-therapeutics-announces-end-of-phase-2-meeting-with-u-s-fda-and-plan-to-initiate-a-phase-3-clinical-trial-of-mco-010-to-treat-stargardt-macular-degeneration/ Clinical Publications : PMID:34418301
NCT03952637	GM1 Gangliosidosis	AAV9-GLB1	National Human Genome Research Institute (NHGRI)	NIH	GLB1	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV9		1.5 x 10^13 vg.kg	4.5 x 10^13 vg/kg				Phase2, Phase1	Recruiting	2019-05-15	2028-01-01	2024-11-08	6 Months - 12 Years	45	1	United States	True			Grant : R01 HD060576 R01 NS076991 Preclinical Publications : PMID: 35249485 PMID: 31534909 PMID: 25964428
NCT06325709	Chronic Granulomatous Disease, X-linked	Base-edited autologous CD34+ cells	National Institute of Allergy and Infectious Diseases (NIAID)	NIH	CYBB c.676 C>T	Gene editing	Ex-vivo	Mutation correction	Intravenous	Autologous cells	CD34+ cells	Electroporation	none	ABE8e-SpRY	Transduced CD34+ cells					Phase2, Phase1	Recruiting	2024-03-21	2032-12-31	2024-11-04	18 Years - 75 Years	10	1	United States	True		First clinical data expected 2025	Grant : Z01-Al-00644 Z01-AI-00645 Z01-Al-00988 Preclinical Publications : PMID: 39413163
NCT01306019	Severe Combined Immunodeficiency, X-linked	VSV-G pseudotyped CL20-i4-EF1α-hγc-OPT vector	National Institute of Allergy and Infectious Diseases (NIAID)	NIH	IL2RG	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	VSV-G		Transduced CD34+ cells (median dose: 6.73 x 10^6 cells/kg; range: 4.46-15.10 x 10^6 cells/kg)					Phase2, Phase1	Recruiting	2011-02-26	2032-12-31	2024-11-08	2 Years - 40 Years	40	1	United States	True		Developed in partnership with St. Jude, and licensed to Mustang Bio, development paused by Mustang Bio	Grant : CLIN2-09504 P01 HL053749 National Cancer Institute (CA21765) NIAID (Z01-AI-00988 & U54-AI082973) Preclinical Publications : PMID: 20457870 PMID: 19286997 News and Press Releases : https://ir.mustangbio.com/news-events/press-releases/detail/159/mustang-bio-announces-strategic-manufacturing-partnership Clinical Publications : PMID: 30995372 Protocol : https://www.nejm.org/doi/suppl/10.1056/NEJMoa1815408/suppl_file/nejmoa1815408_protocol.pdf
NCT05984927	Neovascular (Wet) Age-Related Macular Degeneration	NG101	Neuracle Genetics, Inc	Industry	Codon optimized aflibercept	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Subretinal	Viral vector		Viral transduction	AAV8		1 x 10^9 vg	3 x 10^9 vg	8 x 10^9 vg			Phase2, Phase1	Recruiting	2023-07-24	2030-01	2024-10-23	50 Years - 89 Years	18	4	Canada, United States	True	WO2022010277A1; KR20230167312A;	Plans to expand trial into the USA	Preclinical Publications : https://neuraclegen.com/en/notice/?uid=17&mod=document&pageid=1 News and Press Releases : https://www.thebionews.net/news/articleView.html?idxno=5269
NCT05228145	Batten Disease (Late Infantile Neuronal Ceroid Lipofuscinosis)	NGN-101	Neurogene Inc.	Industry	CLN5	Gene transfer	In-vivo	Functional gene replacement	Intracerebroventricular	Viral vector		Viral transduction	AAV9		Undisclosed low dose	Undisclosed intermediate dose	Undisclosed high dose			Phase2, Phase1	Active not recruiting	2021-12-17	2028-11	2024-08-12	3 Years - 9 Years	6	2	United States, United Kingdom	True		Preliminary data expected 2H 2024	Preclinical Publications : PMID: 30078766 PMID: 37942487 PMID: 37614821 PMID: 33930398 News and Press Releases : https://ir.neurogene.com/static-files/b8cb0b91-fad6-4e91-93b0-fa7ba3d2e412
NCT05898620	Rett Syndrome	NGN-401 (scAAV9.P546.MECP2)	Neurogene Inc.	Industry	MECP2	Gene transfer	In-vivo	Functional gene replacement	Intracerebroventricular	Viral vector		Viral transduction	AAV9		1 x 10^15 vg	3 x 10^15 vg				Phase2, Phase1	Recruiting	2023-06-01	2029-10	2024-10-28	4 Years - 10 Years	16	8	United States, United Kingdom, Australia	True	US9415121B2	Selected for START program (6/5/24); Interim data expected 4Q:24	Preclinical Publications : PMID: 37481701 PMID: 29019980 PMID: 28497075 PMID: 28497072 News and Press Releases : https://ir.neurogene.com/static-files/b8cb0b91-fad6-4e91-93b0-fa7ba3d2e412 https://ir.neurogene.com/news-releases/news-release-details/neurogene-reports-second-quarter-2024-financial-results-and Clinical Publications : https://ir.neurogene.com/static-files/51d2d22f-5f21-4af2-86eb-b4d3d031d4c9 https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=59597
NCT05293626	Leber Hereditary Optic Neuropathy (mt-ND4)	OPVIKA (esonadogene imvoparvovec)	Neurophth Therapeutics Inc	Other	ND4G11778A	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV2		Dose de-escalation: 0.5 x 10^9, 0.05mL	Starting dose: 1.5 x 10^9 vg, 0.05ml/eye	Intermediate dose: 3.0 x 10^9, 0.05mL	High dose: 4.5 x 10^9, 0.05mL		Phase2, Phase1	Active not recruiting	2021-12-09	2029-12	2024-09-04	18 Years - 75 Years	12	3	United States	True	WO2020038352A1; US11332741B1; EP4382601A1	Granted ODD by FDA and EMA, completed US enrollment	Preclinical Publications : PMID: 24396955 PMID: 22612072 News and Press Releases : https://www.clinicaltrialsarena.com/news/neurophth-trial-lhon-therapy/?cf-view Clinical Publications : https://www.asgct.org/global/documents/asgct20_abstracts_may8?_zs=S2i4b&_zl=U9052 (Abstract #1307) PMID: 32284191 PMID: 31237206 PMID: 30968497 PMID: 29188523 PMID: 27749593 PMID: 27426279 PMID: 26892229
NCT05820152	Leber Hereditary Optic Neuropathy (mt-ND1)	NFS-02	Neurophth Therapeutics Inc	Other	ND1G3460A	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV2		5.0 x 10^7 vg, 0.05mL/eye	Starting dose: 1.5 x 10^8 vg, 0.05mL/eye	5.0 x 10^8 vg, 0.05mL/eye	1.5 x 10^9 vg, 0.05mL/eye		Phase2, Phase1	Terminated	2023-04-06	2024-06-24	2024-09-20	18 Years - 75 Years	11	4	United States, China	True	WO2021115317A1	First patient dosed 8/17/23	News and Press Releases : https://www.neurophth.com/en/NewsD-393.html
NCT06018558	Geographic Atrophy	OCU410	Ocugen	Industry	RORA	Gene transfer	In-vivo	Overexpression of protective allele/gene	Subretinal	Viral vector		Viral transduction	AAV5		2.5 x 10^10 vg/mL	5 x 10^10 vg/mL	1.5 x 10^11 vg/mL			Phase2, Phase1	Recruiting	2023-06-30	2025-09-23	2024-10-01	>= 50 Years	63	9	United States	True	US20210123077A1	Dosing of 3rd cohort completed July 2024	Preclinical Publications : https://iovs.arvojournals.org/article.aspx?articleid=2778912 News and Press Releases : https://ir.ocugen.com/news-releases/news-release-details/ocugen-provides-business-update-second-quarter-2024-financial
NCT06388200	Retinitis Pigmentosa	OCU400	Ocugen	Industry	NR2E3	Gene transfer	In-vivo	Overexpression of protective allele/gene	Subretinal	Viral vector		Viral transduction	AAV5		2.5 x 10^10 vg/eye					Phase3	Recruiting	2024-04-09	2026-10-30	2024-10-01	>= 8 Years	150	5	United States	True	US20210123077A1	First patient dosed 6/20/24; on track for 2026 BLA and MAA approval targets	Preclinical Publications : PMID: 32123325 News and Press Releases : https://ir.ocugen.com/news-releases/news-release-details/ocugen-provides-business-update-second-quarter-2024-financial
NCT05956626	Stargardt Disease (Juvenile Macular Degeneration)	OCU410ST	Ocugen	Industry	RORA	Gene transfer	In-vivo	Overexpression of protective allele/gene	Subretinal	Viral vector		Viral transduction	AAV5		3.75 x 10^10 vg/mL	7.5 x 10^10 vg/mL	2.25 x 10^11 vg/mL			Phase2, Phase1	Recruiting	2023-06-30	2025-10-28	2024-03-12	6 Years - 65 Years	42	6	United States	True	US20210123077A1	FDA granted ODD, moving to Phase 2 with the medium and high doses	Preclinical Publications : https://iovs.arvojournals.org/article.aspx?articleid=2778912 News and Press Releases : https://ir.ocugen.com/news-releases/news-release-details/data-and-safety-monitoring-board-approves-initiation-phase-2 https://ir.ocugen.com/news-releases/news-release-details/ocugen-provides-business-update-second-quarter-2024-financial
NCT05616793	Leber Congenital Amaurosis - Type 5	OPGx-LCA5 (AAV8.hLCA5)	Opus Genetics, Inc	Industry	LCA5	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV8		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Phase2, Phase1	Recruiting	2022-11-07	2028-01-30	2024-07-01	>= 13 Years	15	1	United States	True		Received Rare Pediatric Disease Designation from FDA	Preclinical Publications : PMID: 32428231 News and Press Releases : https://opusgtx.com/companynews/opus-genetics-receives-rare-pediatric-disease-designation-from-the-u-s-fda-for-ocular-gene-therapy-opgx-lca5-to-treat-rare-inherited-retinal-disease-lca5/
NCT06149403	Mucopolysaccharidosis Type I (Hurler Syndrome)	OTL-203 (Autologous CD34+ cells transduced with IDUA lentiviral vector)	Orchard Therapeutics	Industry	IDUA	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Mean dose: 20.9 x 10^6 transduced CD34+ cells/kg					Phase3	Recruiting	2023-11-17	2031-03	2024-09-19	28 Days - 30 Months	40	7	Netherlands, United States, Italy, United Kingdom	True		First patient randomized 2/2024	Grant : R01 DK066448 Preclinical Publications : PMID: 20847202 News and Press Releases : https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-announces-positive-clinical-and-preclinical/ Clinical Publications : PMID: 34788506 PMID: 38691622 Protocol : https://www.nejm.org/doi/suppl/10.1056/NEJMoa2106596/suppl_file/nejmoa2106596_protocol.pdf
NCT04903288	AADC Deficiency	UPSTAZA (eladocagene exuparvovec)	PTC Therapeutics	Industry	DDC	Gene transfer	In-vivo	Functional gene replacement	Intraparenchymal	Viral vector	Putamen	Viral transduction	AAV2		1.8×10^11 vg/dose, 320 μl/dose					Phase2	Active not recruiting	2021-05-21	2028-04-30	2024-10-21	1 Year - 17 Years	13	6	United States, Taiwan, Israel	True	US-20190000991-A1; US-10898585-B2; US-20210236653-A1; US-11865188-B2; US-20240100186-A1	BLA Submitted: Priority Review Action Date (11/13/24)	Preclinical Publications : PMID: 36472076 News and Press Releases : https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-announces-fda-acceptance-and-priority-review Clinical Publications : PMID: 37803205 PMID: 37205256 PMID: 34763085 Protocol : https://www.ema.europa.eu/en/documents/product-information/upstaza-epar-product-information_en.pdf
NCT04119687	Osteoarthritis, Knee	PCRX-201, FX-201 (AdV-NK-KB-IL1RA) enekinragene inzadenovec	Pacira Pharmaceuticals, Inc	Industry	IL1RA	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraarticular	Viral vector		Viral transduction	Ad5		1.4 x 10^10 gc/knee	Undisclosed medium dose	Undisclosed high dose			Phase1	Active not recruiting	2019-10-03	2026-11-28	2024-02-02	30 Years - 80 Years	72	7	United States	True	US20190376080A1	FDA granted RMAT designation March 2024	Preclinical Publications : PMID: 34963343 News and Press Releases : https://investor.pacira.com/news-releases/news-release-details/pacira-biosciences-announces-pcrx-201-granted-regenerative Clinical Publications : https://www.cell.com/action/showPdf?pii=S1525-0016%2821%2900206-9 (Abstract #594)
NCT04747431	Frontotemporal Dementia	PBFT02	Passage Bio, Inc.	Industry	GRN	Gene transfer	In-vivo	Functional gene replacement	Intracisterna magna	Viral vector		Viral transduction	AAV1		3.3 x 10^10 GC/g brain weight	1.1 x 10^11 GC/g brain weight	2.2 x 10^11 GC/g brain weight			Phase2, Phase1	Recruiting	2021-02-02	2027-08	2024-08-20	35 Years - 75 Years	15	8	Canada, United States, Brazil, Italy, Portugal	True	WO2024081551A1		Preclinical Publications : PMID: 32937039 News and Press Releases : https://www.passagebio.com/investors-and-news/press-releases-and-statements/news-details/2024/Passage-Bio-Announces-Positive-Feedback-from-FDA-on-Expansion-of-upliFT-D-Trial-of-PBFT02-to-Include-FTD-C9orf72-Patients/default.aspx
NCT04713475	GM1 Gangliosidosis	PBGM01	Passage Bio, Inc.	Industry	GLB1	Gene transfer	In-vivo	Functional gene replacement	Intracisterna magna	Viral vector		Viral transduction	AAVhu68		3.3 x 10^10 GC/g brain weight	1.1 x 10^11 GC/g brain weight	2.2 x 10^11 GC/g brain weight	TBD		Phase2, Phase1	Active not recruiting	2021-01-04	2029-02	2024-05-28	1 Month - 24 Months	26	9	Canada, Turkey, United States, Brazil, United Kingdom	True	WO2024081551A1	Out-licensed development to Gemma Biotherapeutics, uncertain development status	News and Press Releases : https://www.passagebio.com/investors-and-news/press-releases-and-statements/news-details/2024/Passage-Bio-Out-licenses-Three-Pediatric-Gene-Therapy-Programs-to-GEMMA-Biotherapeutics-and-Enters-New-Research-Collaboration/default.aspx
NCT06392724	Duchenne Muscular Dystrophy	GEN6050X	Peking Union Medical College Hospital	Other	DMD	Gene editing	In-vivo	Exon skipping/splice editor	Intravenous	Viral vector		Viral transduction	AAV9	eTAM	5 x 10^13 vg/kg body weight					Early phase1	Recruiting	2024-04-26	2027-12	2024-07-08	4 Years - 10 Years	3	1	China	False	CA3191505A1; WO2017215619A1		Preclinical Publications : PMID: 30293782 PMID: 27723754
NCT05805007	Retinitis Pigmentosa	ZVS203e	Peking University Third Hospital	Other	RHO	Gene editing	In-vivo	Gene inactivation	Subretinal	Viral vector		Viral transduction	AAV	Cas9 mRNA	Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Early phase1	Recruiting	2023-03-14	2026-04	2023-10-24	>= 18 Years	9	1	China	False
NCT04370054	Hemophilia A	Giroctogogene fitelparvovec	Pfizer	Industry	F8	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector	Liver	Viral transduction	AAV2/6		3 × 10^13 vg/kg					Phase3	Active not recruiting	2020-04-21	2028-10-25	2024-07-24	18 Years - 64 Years	76	36	Republic of, United States, Japan, United Kingdom, Spain, Greece, Saudi Arabia, Canada, Sweden, Turkey, Taiwan, Brazil, Italy, Australia, France, Germany, Korea	True	WO2017074526	BLA and MAA submissions expected early 2025	Grant : R01 HL084220 Preclinical Publications : PMID: 21126217 News and Press Releases : https://www.pfizer.com/news/press-release/press-release-detail/pfizer-announces-positive-topline-results-phase-3-study#xd_co_f=YWEzODI0M2MtZmJjZC00YjkxLTllNTktMTI2OWNhMDAyYjZk Clinical Publications : PMID: 37871576 https://investor.sangamo.com/static-files/1ea27793-cd7d-44d6-9e9c-65ba6a07da00
NCT04408625	Frontotemporal Dementia	LY3884963 (PR006) AAV9-CMVe-CBA-GRN	Prevail Therapeutics	Industry	GRN	Gene transfer	In-vivo	Functional gene replacement	Intracisterna magna	Viral vector		Viral transduction	AAV9		2.1 x 10^13 vg	4.2 x 10^13 vg				Phase2, Phase1	Recruiting	2020-05-21	2029-08-31	2024-10-21	30 Years - 85 Years	30	11	Belgium, United States, United Kingdom, Australia, France, Spain	True	US20210261981A1; WO2022082017A2		Preclinical Publications : PMID: 30559071 PMID: 29378861 PMID: 28379303 https://www.asgct.org/global/documents/asgct20_abstracts_may8.aspx?_ga=2.195561324.1360682713.1722530946-1234197496.1714069342&_gl=11jj0ddw_gaMTIzNDE5NzQ5Ni4xNzE0MDY5MzQy_ga_Q37QKR6TCJMTcyMjU0MzAxMC4yMi4xLjE3MjI1NDMwMjkuMC4wLjA._gcl_auMTY3NjYzNjM2Mi4xNzIyNTMwOTQ2_ga_6FB6X4L6XFMTcyMjU0MzAxMC4yMi4xLjE3MjI1NDMwMjkuNDEuMC4w News and Press Releases :* https://www.prevailtherapeutics.com/prevail-therapeutics-announces-first-patient-dosed-in-phase-1-2-proclaim-clinical-trial-evaluating-pr006-for-the-treatment-of-frontotemporal-dementia-patients-with-grn-mutations/ Clinical Publications : PMID: 38745011
NCT04411654	Gaucher Disease, Type 1	LY3884961 (PR001) AAV9-CMV-CBA-GBA1	Prevail Therapeutics	Industry	GBA1	Gene transfer	In-vivo	Functional gene replacement	Intracisterna magna	Viral vector		Viral transduction	AAV9		Undisclosed low dose	Undisclosed high dose				Phase2, Phase1	Active not recruiting	2020-05-11	2028-05	2024-03-21	0 Months - 24 Months	15	5	United States, United Kingdom	True	WO2022082017A2; US20200318115A1; US11903985B2; US20220211871A1		Preclinical Publications : https://alz-journals.onlinelibrary.wiley.com/doi/abs/10.1002/alz.043614 https://www.asgct.org/global/documents/asgct20_abstracts_may8.aspx?_ga=2.195561324.1360682713.1722530946-1234197496.1714069342&_gl=11jj0ddw_gaMTIzNDE5NzQ5Ni4xNzE0MDY5MzQy_ga_Q37QKR6TCJMTcyMjU0MzAxMC4yMi4xLjE3MjI1NDMwMjkuMC4wLjA._gcl_auMTY3NjYzNjM2Mi4xNzIyNTMwOTQ2_ga_6FB6X4L6XFMTcyMjU0MzAxMC4yMi4xLjE3MjI1NDMwMjkuNDEuMC4w News and Press Releases :* https://www.prevailtherapeutics.com/our-progress/proceed-update/ Clinical Publications : PMID:34151863
NCT06559176	Chronic Granulomatous Disease, p47phox	PM359	Prime Medicine, Inc.	Industry	NCF1 (c.75_76delGT)	Gene editing	Ex-vivo	Mutation correction	Intravenous	Autologous cells	CD34+ cells	Electroporation		prime editor	Transduced CD34+ cells					Phase2, Phase1	Recruiting	2024-07-11	2029-12	2024-10-15	>= 6 Years	12	5	Canada, United States, United Kingdom	True	US20230357766A1; US11795452B2; US20230220374A1		Grant : R00 HL163805 R01EB031172 R35 GM118062 U01AI142756 R01 GM065400 F32 GM 112366 RM1 HG009490 Preclinical Publications : PMID: 37142705 PMID: 27096365 https://primemedicine.com/wp-content/uploads/2024/01/Prime-Medicine-JPM-2024-Deck.pdf https://ashpublications.org/blood/article/142/Supplement%201/7129/500818/Prime-Editing-Efficiently-and-Precisely-Corrects News and Press Releases : https://investors.primemedicine.com/news-releases/news-release-details/prime-medicine-presents-preclinical-data-demonstrating-ability
NCT03566043	Mucopolysaccharidosis Type II (Hunter Syndrome)	RGX-121 (AAV9-IDS)	REGENXBIO Inc.	Industry	IDS	Gene transfer	In-vivo	Functional gene replacement	Intracisterna magna	Viral vector		Viral transduction	AAV9		1.3 x 10^10 GC/g brain mass	6.5 x 10^10 GC/g brain mass	2.9 x 10^11 GC/g brain mass			Phase3, Phase2	Active not recruiting	2018-05-01	2025-05	2023-11-18	4 Months - 5 Years	48	5	United States, Brazil	True	US20180036388A1; US20240108761A1	BLA filing planned for Q3 2024	Preclinical Publications : PMID: 36541357 PMID: 28478695 PMID: 27510804 News and Press Releases : https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-successful-pre-bla-meeting-fda-support https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-reports-first-quarter-2024-financial-results-and Clinical Publications : https://www.regenxbio.com/getattachment/353ec3e0-e8ab-4545-bc5f-2b54e4b23c17/HARMATZ-RGX-121-WORLD-web-FINAL.pdf?lang=en-US&ext=.pdf https://ir.regenxbio.com/static-files/c8d99eac-95bf-4fae-8d6c-2ace6fe11b0e
NCT05693142	Duchenne Muscular Dystrophy	RGX-202 (AAV8-microdystrophin)	REGENXBIO Inc.	Industry	Micro-dystrophin	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravenous	Viral vector		Viral transduction	AAV8		1 x 10^14 GC/kg body weight	2 x 10^14 GC/kg body weight				Phase2, Phase1	Recruiting	2023-01-04	2025-12	2024-07-23	1 Year - 11 Years	15	5	United States	True	US20230270886A1	Pivotal trial initiation expected Q3-Q4 2024; EOP2 meeting with FDA in July 2024	News and Press Releases : https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-new-positive-data-affinity-duchenner-trial https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-announces-expansion-affinity-duchenner-trial-include https://ir.regenxbio.com/news-releases/news-release-details/regenxbio-reports-first-quarter-2024-financial-results-and Clinical Publications : https://www.regenxbio.com/getattachment/0647f60f-a68d-4ca0-86e7-57c979ec7f35/RGX-202-ASGCT24.pdf?lang=en-US&ext=.pdf
NCT06460844	Retinitis Pigmentosa	RTx-015	Ray Therapeutics, Inc.	Industry	Codon optimized ChR-3M	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravitreal	Viral vector		Viral transduction	AAV.7m8		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Phase1	Recruiting	2024-06-10	2026-05	2024-10-01	>= 18 Years	9	2	United States	True			Preclinical Publications : PMID: 39237697 PMID: 31010741 News and Press Releases : https://raytherapeutics.com/video-overview/ https://bakarlabs.berkeley.edu/on-a-personal-mission-for-his-daughter-paul-bresge-launches-another-eye-disease-biotech-with-100m-for-ray-therapeutics/
NCT05788536	Congenital Hearing Loss	DB-OTO, Myo15 promotor	Regeneron Pharmaceuticals	Industry	OTOF	Gene transfer	In-vivo	Functional gene replacement	Intracochlear	Viral vector	Hair cell	Viral transduction	dual AAV1		3 x 10^13 vg/ml	Undisclosed high dose	Undisclosed expansion dose			Phase2, Phase1	Recruiting	2023-03-15	2031-04-19	2024-09-04	<= 17 Years	22	12	United States, United Kingdom, Spain	True		Phase I/II interim data presented at ASGCT on 2 patients	Preclinical Publications : PMID: 38952419 https://decibeltx.wpenginepowered.com/wp-content/uploads/ASGCT-2021_OTOF-program.pdf News and Press Releases : https://investor.regeneron.com/news-releases/news-release-details/latest-db-oto-results-show-dramatically-improved-hearing-normal Clinical Publications : https://investor.regeneron.com/news-releases/news-release-details/latest-db-oto-results-show-dramatically-improved-hearing-normal
NCT06511349	Primary Hyperoxaluria, Type I	YOLT-203	RenJi Hospital	Other	HAO1	Gene editing	In-vivo	Gene inactivation	Intravenous	MRNA, LNP		Lipid encapsulation	LNP	YolCas12	0.3 mg/kg	0.6 mg/kg	1.0 mg/kg			Early phase1	Recruiting	2024-07-04	2026-01-31	2024-08-21	>= 2 Years	7	1	China	False		First patient dosed (8/5/24), ODD and RPDD granted by FDA in September 2024	Preclinical Publications : PMID: 38874509 News and Press Releases : https://www.yoltx.com/news/press-release/
NCT06092034	Danon Disease	RP-A501	Rocket Pharmaceuticals Inc.	Industry	LAMP2B	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV9		6.7 x 10^13 GC/kg					Phase2	Active not recruiting	2023-10-11	2029-09	2024-09-25	>= 8 Years	12	6	United States, Italy, United Kingdom, Germany	True	US10703797B2; US20210379201A1	Phase II enrollment completed 09/2024	Grant : CLIN2-15218 Preclinical Publications : PMID: 32188720 News and Press Releases : https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-announces-completion-enrollment-phase-2 https://ir.rocketpharma.com/static-files/cd0d9ae3-5f6d-44ac-8874-27c46d0ed05c Clinical Publications : https://ir.rocketpharma.com/static-files/cd0d9ae3-5f6d-44ac-8874-27c46d0ed05c
NCT04105166	Pyruvate Kinase Deficiency	RP-L301	Rocket Pharmaceuticals Inc.	Industry	PKLR	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	PGK-coPRK-WPRE		Dose range: 2.3 x 10^6 - 6.5 x 10^6 transduced CD34+ cells/kg					Phase1	Active not recruiting	2019-09-24	2025-05	2024-02-05	8 Years - 50 Years	5	3	United States, Spain	True	CA3106241A1; WO2020037249A1	Phase I data presented at ASGCT 2024; Global Phase II planned late 2024/early 2025	Preclinical Publications : PMID: 27138040 News and Press Releases : https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-presents-positive-data-lv-hematology Clinical Publications : https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-presents-positive-data-lv-hematology https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=59989
NCT04248439	Fanconi Anemia, subtype A	Mozafancogene autotemcel (RP-L102) PGK-FANCA	Rocket Pharmaceuticals Inc.	Industry	FANCA	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Dose range: 2.0 x 10^5 - 4.1 x 10^6 transduced CD34+ cells/kg					Phase2	Active not recruiting	2020-01-24	2026-05	2024-04-10	>= 1 Year	5	2	United States	True	CA3093708A1; CA3106241A1; WO2020037249A1	BLA filing H1 2024; MAA accepted by EMA 4/2/24	Preclinical Publications : PMID: 28801449 PMID: 26415575 PMID: 20001454 News and Press Releases : https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-presents-positive-data-lv-hematology Clinical Publications : https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=59471 PMID: 31501599 https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-presents-positive-data-lv-hematology
NCT03812263	Leukocyte Adhesion Defect - Type I	KRESLADI (mametegragene autotemcel) RP-L201	Rocket Pharmaceuticals Inc.	Industry	ITGB2	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	Chim.hCD18-LV		Dose range: 2.8-10 x 10^6 transduced CD34+ cells/kg	Min dose: 2.0 x 10^6 CD34+ cells/kg				Phase2, Phase1	Completed	2019-01-18	2023-09-12	2023-11-15	>= 3 Months	9	3	United States, United Kingdom, Spain	True	EP3830248A1; CA3106241A1; WO2020037249A1	BLA Submitted: Pending FDA (????)	Grant : CIRM grant: CLIN2-11480 Preclinical Publications : PMID: 36092365 PMID: 27056660 News and Press Releases : https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-provides-regulatory-update-kresladitm Clinical Publications : https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=59948 https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-presents-positive-data-lv-hematology https://ashpublications.org/blood/article/140/Supplement%201/7774/491308/Interim-Results-from-an-Ongoing-Phase-1-2-Study-of
NCT05885412	Arrhythmogenic Right Ventricular Cardiomyopathy	RP-A601 (AAVrh.74-PKP2a)	Rocket Pharmaceuticals Inc.	Industry	PKP2A	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAVrh.74		Starting dose: 8 x 10^13 GC/kg					Phase1	Recruiting	2023-05-22	2026-09	2024-10-04	>= 18 Years	9	3	United States	True		5/2024: Received Orphan Medicial Product designation from EMA	Preclinical Publications : PMID: 38288614 News and Press Releases : https://ir.rocketpharma.com/static-files/cd0d9ae3-5f6d-44ac-8874-27c46d0ed05c https://ir.rocketpharma.com/news-releases/news-release-details/rocket-pharmaceuticals-receives-orphan-medicinal-product
NCT02610582	Achromatopsia	RAAV.hCNGA3 (AAV8-hArr3-hCNGA3-WPREm)	STZ eyetrial	Other	CNGA3	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector	Cone cells	Viral transduction	AAV8		1x10^11 vg/dose					Phase2, Phase1	Active not recruiting	2015-09-18	2027-06	2024-04-18	>= 6 Years	13	1	Germany		US20180353619; WO2017144080A1; WO2016146669A1; WO2018010965A1		Preclinical Publications : PMID: 20628362 Clinical Publications : PMID: 34006508 PMID: 32352493 PMID: 30187779
NCT04611503	Retinitis Pigmentosa	RAAV.hPDE6A	STZ eyetrial	Other	PDE6A	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV8		1 x 10^9 vg	5 x 10^9 vg	1 x 10^10 vg	5 x 10^10 vg		Phase2, Phase1	Active not recruiting	2020-05-20	2027-07	2024-04-18	>= 18 Years	9	1	Germany	False			Preclinical Publications : PMID: 29212391 PMID: 29212382
NCT04046224	Fabry Disease	Isaralgagene civaparvovec (ST-920)	Sangamo Therapeutics	Industry	GLA	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV2/6		0.26 x 10^13 vg/kg	0.53 x 10^13 vg/kg	1.58 x 10^13 vg/kg	2.63 x 10^13 vg/kg		Phase2, Phase1	Active not recruiting	2019-08-01	2025-09	2024-05-09	>= 18 Years	34	18	Canada, United States, Taiwan, Italy, United Kingdom, Australia, Germany	True	US20200231989A1; US9877988B2;	Recent Type B meeting with FDA allowed ongoing Phase 1/2 study to serve as the primary basis for approval under Accelerated Approval Program, BLA submission H2 2025	Preclinical Publications : PMID: 32775495 News and Press Releases : https://investor.sangamo.com/news-releases/news-release-details/sangamo-therapeutics-announces-alignment-fda-accelerated https://investor.sangamo.com/news-releases/news-release-details/sangamo-therapeutics-announces-us-fda-alignment-abbreviated Clinical Publications : https://investor.sangamo.com/static-files/c42a663e-3c1a-4f98-90b7-f61a3c50daf0
NCT05972629	Phenylketonuria	SAR444836	Sanofi	Industry	PAH	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV SNY001		Undisclosed dose 1					Phase2, Phase1	Recruiting	2023-06-23	2027-07-31	2024-11-01	18 Years - 65 Years	32	5	Turkey, United States, Israel	True		Deprioritized by Sponsor	News and Press Releases : https://www.sanofi.com/assets/dotcom/content-app/events/quaterly-results/2024/2024-q2-2024-results/q2-2024-presentation-en.pdf
NCT06061549	Heart Failure with Preserved Ejection Fraction	AAV1-CMV-SERCA2a (SRD-001)	Sardocor Corp.	Industry	SERCA2a	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraarterial	Viral vector		Viral transduction	AAV1		3 x 10^13 vg					Phase1	Recruiting	2023-07-30	2029-08	2023-09-29	>= 50 Years	10	2	United States	True	WO2011130552A2; WO2020176732A1	First patients dosed Q1 2024, product/indication combo granted Fast Track designation	Grant : P50 HL112324 R01 HL088434 R43 HL075934 Preclinical Publications : PMID: 18342232 PMID: 30371209 PMID: 21406682 News and Press Releases : https://www.einpresswire.com/article/688652471/medera-s-sardocor-announces-fast-track-designation-and-dosing-of-3-patients-in-first-in-human-hfpef-gene-therapy-trial Clinical Publications : PMID: 24065463 PMID: 32669717
NCT06224660	DMD-Associated Dilated Cardiomyopathy	AAV1-CMV-SERCA2a (SRD-001)	Sardocor Corp.	Industry	SERCA2a	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraarterial	Viral vector		Viral transduction	AAV1		1 x 10^13 vg	3 x 10^13 vg				Phase1	Not yet recruiting	2024-01-11	2030-02	2024-01-25	>= 18 Years	12	0		True		Granted Orphan Drug Designation by FDA 2/28/24	Grant : R01 AR070517 R01 AR069107 Preclinical Publications : PMID: 31981493 News and Press Releases : https://www.einpresswire.com/article/692002837/medera-s-sardocor-granted-fda-orphan-drug-designation-for-dmd-associated-cardiomyopathy-gene-therapy
NCT04703842	Heart Failure with Reduced Ejection Fraction	AAV1-CMV-SERCA2a (SRD-001)	Sardocor Corp.	Industry	SERCA2a	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraarterial	Viral vector		Viral transduction	AAV1		3 x 10^13 vg	4.5 x 10^13 vg				Phase2, Phase1	Recruiting	2021-01-07	2028-12	2024-03-26	18 Years - 80 Years	57	5	United States	True	WO2011130552A2; WO2020176732A1	Update given at ASGCT 2024	Grant : P20 HL100396 P50 HL112324 R01 HL078731 R01 HL088434 R43 HL075934 Preclinical Publications : PMID: 30371209 PMID: 21406682 PMID: 18342232 Clinical Publications : PMID: 32669717 PMID: 26803443 PMID: 24622121 PMID: 24065463 PMID: 21709064 PMID: 19327618
NCT01976091	Limb-Girdle Muscular Dystrophy, Type 2D/R3	Patidistrogene bexoparvovec (SRP-9004) rAAVrh74-tMCK-SGCA	Sarepta Therapeutics, Inc.	Industry	SGCA	Gene transfer	In-vivo	Functional gene replacement	Isolated limb infusion	Viral vector		Viral transduction	AAVrh74		1 x 10^12 vg/kg/limb	3 x 10^12 vg/kg/limb				Phase2, Phase1	Completed	2013-07-24	2019-03-14	2023-06-15	>= 7 Years	6	0		True	US20220370639A1; WO2021257595A1		Grant : U01 AR060911 U54 NS055958 F32 AR055008 Preclinical Publications : PMID: 33349138 PMID: 18525034 News and Press Releases : https://www.youtube.com/watch?v=IaMvWxNaauY&t=3s Clinical Publications : PMID: 30838895 PMID: 21031578 PMID: 19798725 Protocol : https://cdn.clinicaltrials.gov/large-docs/91/NCT01976091/Prot_000.pdf https://cdn.clinicaltrials.gov/large-docs/91/NCT01976091/SAP_001.pdf
NCT05906251	Limb-Girdle Muscular Dystrophy Type 2B/R2	SRP-6004 (rAAVrh74-MHCK7-DYSF)	Sarepta Therapeutics, Inc.	Industry	DYSF	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAVrh74		2 x 10^12 vg	6 x 10^12 vg				Phase1	Active not recruiting	2023-06-07	2028-08-31	2024-08-20	18 Years - 50 Years	2	1	United States	True	WO2021257595A1		Grant : U54 HD066409 Preclinical Publications : PMID: 28707952 PMID: 25815352 News and Press Releases : https://www.youtube.com/watch?v=IaMvWxNaauY&t=3s https://www.sarepta.com/community-letter-community-update-lgmd-programs-development
NCT06246513	Limb-Girdle Muscular Dystrophy, Type 2E/R4	Bidridistrogene xeboparvovec (SRP-9003) AAVrh74-MHCK7-SGCB	Sarepta Therapeutics, Inc.	Industry	SGCB	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAVrh74		5 x 10^13 vg/kg					Phase3	Recruiting	2024-01-30	2029-11-30	2024-05-29	>= 4 Years	15	4	United States	True	US20230241252A1; WO2021257595A1; EP4219726A1	FDA granted Fast Track designation	Preclinical Publications : PMID: 28284983 PMID: 26214262 News and Press Releases : https://www.youtube.com/watch?v=IaMvWxNaauY&t=3s https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-therapeutics-initiates-screening-emergene-phase-3 https://www.sarepta.com/community-letter-community-update-lgmd-programs-development Clinical Publications : PMID: 38177855
NCT06370351	Congenital Hearing Loss	SENS-501	Sensorion	Industry	OTOF	Gene transfer	In-vivo	Functional gene replacement	Intracochlear	Viral vector		Viral transduction	AAV		Undisclosed low dose	Undisclosed high dose	Undisclosed expansion dose			Phase2, Phase1	Recruiting	2024-04-09	2031-07	2024-09-26	6 Months - 31 Months	12	2	Australia, France	False		First patient dosed Q3 2024	Preclinical Publications : PMID: 30782832 https://www.sensorion.com/wp-content/uploads/2024/02/20240126_ARO_2024_Poster_Otof.pdf News and Press Releases : https://s27.q4cdn.com/232015521/files/doc_news/Sensorion-Reports-New-Positive-Clinical-Results-Presented-at-the-World-Congress-of-Audiology-2024.pdf https://s27.q4cdn.com/232015521/files/doc_news/2024/02/2024-01-18_SENS_Sensorion-Announces-Approval-to-initiate-Audiogene-in-France_Final.pdf
NCT06474442	Herpes Simplex Virus Type I Stromal Keratitis	BD111	Shanghai BDgene Co., Ltd.	Industry	HSV genes	Gene editing	In-vivo	Gene excision	Intrastromal	Viral vector		Viral transduction	LV	SpCas9 mRNA	1.25 x 10^6 TU/eye	2.5 x 10^6 TU/eye	5.0 x 10^6 TU/eye	10 x 10^6 TU/eye		Phase2	Not yet recruiting	2024-06-13	2026-12	2024-06-25	18 Years - 70 Years	40	1	China	False	WO2024011980A1		Preclinical Publications : PMID: 33432198 Clinical Publications : PMID: 37658603
NCT06465550	Beta-Thalassemia, transfusion-dependent	BD211 (βA-T87Q-globin)	Shanghai BDgene Co., Ltd.	Industry	HBB	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells (> 5 x 10^6 cells/kg)					Phase1	Recruiting	2024-06-12	2026-12	2024-06-24	3 Years - 35 Years	9	3	China	False	WO2023130911A1		Clinical Publications : PMID: 38759653
NCT05203679	Hemophilia B	BBM-H901	Shanghai Belief-Delivery BioMed Co., Ltd	Industry	F9	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV843		1 x 10^13 vg/kg					Phase3	Active not recruiting	2021-12-29	2028-06-30	2024-07-24	>= 18 Years	32	9	China	False	WO2019241324A1; WO2022222869A1; WO2021180118A1	Dosing completed of all Phase III subjects	Grant : K08 HL146991 News and Press Releases : https://www.beliefbiomed.com/en/news-center/bbm-news/belief-biomed-announces-a-key-milestone-of-dosing-completion-for-all-subjects-in-its-registrational-clinical-trial-of-bbm-h901/ Clinical Publications : PMID: 36306204 PMID: 35598604 Protocol : https://d1niluoi1dd30v.cloudfront.net/23523026/S2352302622X00070/S2352302622001132/mmc1.pdf?Expires=1724087113&Key-Pair-Id=APKAICLNFGBCWWYGVIZQ&Signature=Itisx-~E3Wj7Wv4jZf3EFCIrPPCqq2Z4W05POv-Vu3jx~zVEAuHDBT-mWryh907i2YlkU4BYThhNfI03tjhVMK55XbKWIYKnLqpvJbxvkOEAX9~bBIeeezTdlFlTUvijV0pXBeLu3Mkffd3pVGB4MGHSqXBjuVyzfbuFlimUpNA_
NCT06111638	Hemophilia A	BBM-H803	Shanghai Belief-Delivery BioMed Co., Ltd	Industry	F8	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV		Undisclosed dose 1					Phase2, Phase1	Recruiting	2023-10-27	2030-06-30	2024-07-23	>= 18 Years	12	1	China	False	WO2022222869A1; WO2021180118A1	First patient dosed January 2024	News and Press Releases : https://www.prnewswire.com/apac/news-releases/belief-biomed-successfully-completed-dosing-of-first-subject-in-the-registrational-clinical-trial-for-hemophilia-a-302025243.html
NCT05765981	AADC Deficiency	VGN-R09b (AADC + NTF)	Shanghai Jiao Tong University School of Medicine	Other	DDC	Gene transfer	In-vivo	Functional gene replacement	Intraparenchymal	Viral vector	Striatum	Viral transduction	AAV9		Undisclosed single dose					Early phase1	Recruiting	2023-01-18	2029-02-20	2023-03-13	24 Months - 7 Years	6	1	China	False	WO2023202637	NMPA IND accepted 1/24/24; FDA IND accepted 7/26/24	News and Press Releases : http://www.vitalgen-biomed.com/en/details.php?id=24
NCT06141460	Neovascular (Wet) Age-Related Macular Degeneration	RRG001	Shanghai Refreshgene Technology Co., Ltd.	Industry	Anti-VEGF	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Subretinal	Viral vector		Viral transduction	AAV		Undisclosed dose 1	Undisclosed dose 2	Undisclosed dose 3	Undisclosed dose 4	Dose range: 1 x 10^8 - 1 x 10^13 vg/eye	Phase2, Phase1	Recruiting	2023-11-10	2030-12-31	2024-01-03	>= 50 Years	48	1	China	False	WO2024002076A1
NCT05441553	Hemophilia B	VGB-R04	Shanghai Vitalgen BioPharma Co., Ltd.	Industry	F9	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV8		Undisclosed dose (ranging from 4x10^11 - 2x10^12 vg/kg)					Phase2, Phase1	Not yet recruiting	2022-06-15	2025-01	2022-07-01	18 Years - 65 Years	26	1	China	False	WO2023280323A1; WO2023072181A1	Granted ODD in December 2021
NCT06480461	Parkinson's Disease	VGN-R09b (AADC + NTF)	Shanghai Vitalgen BioPharma Co., Ltd.	Industry	DDC	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intraparenchymal	Viral vector	Striatum	Viral transduction	AAV9		8.0 x 10^11 vg	1.6 x 10^12 vg	3.2 x 10^12 vg			Phase2, Phase1	Not yet recruiting	2024-06-14	2031-07-01	2024-06-28	40 Years - 75 Years	39	0		False	WO2024017387A1
NCT05694598	Bietti Crystalline Dystrophy	VGR-R01 (rAAV2/8-hCYP4V2)	Shanghai Vitalgen BioPharma Co., Ltd.	Industry	CYP4V2	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2/8		6.0 x 10^10 vg/eye	1.2 x 10^11 vg/eye	2.0 x 10^11 vg/eye	1.2 x 10^11 vg/eye (expansion dose)		Phase1	Not yet recruiting	2023-01-11	2025-09-01	2023-01-23	18 Years - 69 Years	12	1	China	False	WO2023284873A1; US20240018541A1	Phase I/II preliminary results presented at ASGCT 2024	Preclinical Publications : PMID: 35925866 News and Press Releases : http://www.vitalgen-biomed.com/en/ Clinical Publications : PMID: 38653979 https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=98138
NCT06217861	Glutaric Acidemia Type I	VGM-R02b	Shanghai Vitalgen BioPharma Co., Ltd.	Industry	GCDH	Gene transfer	In-vivo	Functional gene replacement	Intracerebroventricular	Viral vector		Viral transduction	AAV9		Undisclosed dose					Phase1	Recruiting	2023-12-12	2026-08	2024-05-17	<= 6 Years	12	1	China	False	WO2023221942A1; WO2024017387A1	Granted CTA approval by NMPA on 7/13/23	News and Press Releases : http://www.vitalgen-biomed.com/en/details.php?id=19
NCT02559830	Adrenoleukodystrophy	CD34+ hematopoietic stem cells transduced with ABCD1	Shenzhen Second People's Hospital	Other	ABCD1	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		2x10^6 transduced CD34+ cells/kg (maximum 20x10^6)					Phase2, Phase1	Recruiting	2015-08-12	2025-10	2022-05-31	1 Year - 16 Years	50	1	China
NCT05986864	Neovascular (Wet) Age-Related Macular Degeneration	SKG0106	Skyline Therapeutics (US) Inc.	Industry	Anti-VEGF	Gene transfer	In-vivo	Genetic delivery of therapeutic protein	Intravitreal	Viral vector		Viral transduction	AAV		8 x 10^9 vg/eye	2.4 x 10^10 vg/eye	7.2 x 10^10 vg/eye			Phase2, Phase1	Recruiting	2023-08-03	2026-01-30	2024-06-28	>= 50 Years	68	8	United States, China	True	WO2023041015A1		Preclinical Publications : https://iovs.arvojournals.org/article.aspx?articleid=2799427 News and Press Releases : https://www.skytx.com/news-2023_07_03 Clinical Publications : https://www.cell.com/action/showPdf?pii=S1525-0016%2824%2900237-5 (Abstract #1626)
NCT06138639	Duchenne Muscular Dystrophy	SGT-003 (AAV-SLB101-CK8-microdystrophin containing R16-R17 nNOS binding domain)	Solid Biosciences Inc.	Industry	Micro-dystrophin	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravenous	Viral vector	Muscle cells	Viral transduction	AAV-SLB101		1 x 10^14 vg/kg					Phase2, Phase1	Recruiting	2023-11-14	2031-05-06	2024-10-15	4 Years - 11 Years	43	2	United States	True	US20230183740A1; US20220031865A1; WO2021072197A1	FDA granted Rare Pediatric Disease Designation 4/1/24; topline data expected Q4 2024	Preclinical Publications : https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=59885 News and Press Releases : https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-receives-rare-pediatric-disease-designation https://investors.solidbio.com/news-releases/news-release-details/solid-biosciences-provides-first-quarter-2024-business-update
NCT05748873	Retinitis Pigmentosa	SPVN06	SparingVision	Industry	NXNL1	Gene transfer	In-vivo	Overexpression of protective allele/gene	Subretinal	Viral vector		Viral transduction	AAV8		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Phase2, Phase1	Recruiting	2023-02-07	2029-03	2024-09-19	>= 18 Years	33	4	United States, France	True	WO2024084075A1; EP3728610B1	Enrollment of highest dose cohort is underway	Preclinical Publications : https://iovs.arvojournals.org/article.aspx?articleid=2786169&resultClick=1 https://iovs.arvojournals.org/article.aspx?articleid=2778934&resultClick=1 News and Press Releases : https://sparingvision.com/sparingvision-reports-progress-on-spvn06-at-major-ophthalmology-conferences-2/ Clinical Publications : https://iovs.arvojournals.org/article.aspx?articleid=2798292&resultClick=1
NCT06297486	Hemophilia A	Dirloctogene samoparvovec (SPK-8011) AAV LK03 capsid + TTRmut-hFVIII-X07	Spark Therapeutics, Inc.	Industry	F8	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	LK03		5 x 10^11 - 2 x 10^12 vg/kg					Phase3	Recruiting	2024-02-29	2035-09-04	2024-07-29	>= 18 Years	85	27	United States	True	WO2019028192A1; US11168124B2; WO2019006390A1; US20220362408A1		Grant : R01 HL126850 P01 HL064190 K08 HL140078 K08 HL146991 Preclinical Publications : PMID: 34977269 News and Press Releases : https://sparktx.com/press_releases/spark-therapeutics-announces-updated-phase-1-2-study-results-supporting-the-durability-of-investigational-gene-therapy-spk-8011-in-patients-with-hemophilia-a/ Clinical Publications : PMID: 34788507 PMID: 29211678
NCT06293729	Familial Hypercholesterolemia, Refractory	NGGT006	Suzhou Municipal Hospital	Other	LDLR	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravenous	Viral vector		Viral transduction	AAV		7.5 x 10^12 vg/kg	1.5 x 10^13 vg/kg	3 x 10^13 vg/kg			Early phase1	Not yet recruiting	2024-02-27	2029-03-01	2024-04-17	18 Years - 55 Years	9	0		False	CN117887723A
NCT05394064	Adrenomyeloneuropathy	SBT101	SwanBio Therapeutics, Inc.	Industry	ABCD1	Gene transfer	In-vivo	Functional gene replacement	Intrathecal	Viral vector		Viral transduction	AAV9		1.0x10^14 vg	3.0x10^14 vg				Phase2, Phase1	Recruiting	2022-05-16	2029-03-30	2023-09-13	18 Years - 65 Years	16	2	Netherlands, United States	True			Preclinical Publications : https://swanbiotx.com/investors-and-media/wp-content/themes/swanbio-theme/pdfs/SwanBio%20ASGCT%202022_Selection%20of%20clinical%20doses%20for%20SBT101%20for%20treatment%20of%20AMN_16May22.pdf News and Press Releases : https://www.synconaltd.com/media/2tdavwse/cell-and-gene-therapy-overview.pdf
NCT02362438	Giant Axonal Neuropathy	ScAAV9/JeT-GAN	Taysha Gene Therapies, Inc.	Industry	GAN	Gene transfer	In-vivo	Functional gene replacement	Intrathecal	Viral vector		Viral transduction	AAV9		3.5 x 10^13 vg	1.2 x 10^14 vg	1.8 x 10^14 vg	3.5 x 10^14 vg		Phase1	Active not recruiting	2015-02-12	2035-04-01	2023-11-21	3 Years - 99 Years	21	1	United States	True	US20240102050A1		Grant : R01NS087175 F32 NS095515 Preclinical Publications : PMID: 30709364 PMID: 33955818 PMID: 29766026 Clinical Publications : PMID: 38507752 Protocol : https://www.nejm.org/doi/suppl/10.1056/NEJMoa2307952/suppl_file/nejmoa2307952_protocol.pdf
NCT05606614	Rett Syndrome	TSHA-102	Taysha Gene Therapies, Inc.	Industry	MiniMECP2	Gene transfer	In-vivo	Functional gene replacement	Intrathecal	Viral vector		Viral transduction	scAAV9		5.7 x 10^14 vg	1 x 10^15 vg				Phase2, Phase1	Recruiting	2022-10-28	2032-01-05	2024-10-08	>= 12 Years	18	6	Canada, United States	True	US20240191254A1; US20240102050A1; US20190328804A1	Recieved RMAT designation in May 2024	Preclinical Publications : PMID: 38254921 News and Press Releases : https://ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-reports-second-quarter-2024-financial Clinical Publications : https://ir.tayshagtx.com/static-files/663b6ef4-52de-4bcd-97f2-97756d298cb3 https://ir.tayshagtx.com/news-releases/news-release-details/taysha-gene-therapies-announces-positive-clinical-data-across
NCT05836259	Hypertrophic Cardiomyopathy	TN-201	Tenaya Therapeutics	Industry	MYBPC3	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV9		3 x 10^13 vg/kg	6 x 10^13 vg/kg				Phase1	Recruiting	2023-04-18	2029-12	2024-11-01	18 Years - 65 Years	15	10	United States	True	WO2021163357A2; US20240084327A1	Initial data from cohort 1 available H2 2024; enrolling patients for high dose (Cohort 2)	Preclinical Publications : https://www.tenayatherapeutics.com/wp-content/uploads/HCMS-2023-Interim-Serostatus-Data-Desai.pdf https://www.tenayatherapeutics.com/wp-content/uploads/HCMS-2023-MyPeak-Trial-Design-Robertson.pdf News and Press Releases : https://investors.tenayatherapeutics.com/news-releases/news-release-details/tenaya-therapeutics-announces-updates-tn-201-gene-therapy https://investors.tenayatherapeutics.com/news-releases/news-release-details/tenaya-therapeutics-reports-second-quarter-2024-financial
NCT06228924	Arrhythmogenic Right Ventricular Cardiomyopathy	TN-401	Tenaya Therapeutics	Industry	PKP2	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV9		3 x 10^13 vg/kg	6 x 10^13 vg/kg				Phase1	Recruiting	2024-01-18	2029-10-01	2024-10-02	18 Years - 65 Years	15	7	United States	True	WO2022076648A1	Dosing expected to begin H2 2024	Preclinical Publications : PMID: 38499690 https://www.tenayatherapeutics.com/wp-content/uploads/04152022_PKP2-Presentation_HRS2022_v6.pdf News and Press Releases : https://investors.tenayatherapeutics.com/news-releases/news-release-details/tenaya-therapeutics-reports-first-quarter-2024-financial-results
NCT06107400	Hemoglobin H disease, Constant Spring type	RM-004	The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army	Other	(HBA2):c.427T>C (p.Ter143Gln)	Gene editing	Ex-vivo	Mutation correction	Intravenous	Autologous cells	CD34+ cells	Lipid encapsulation	LNP	SpRY-CBE	Transduced CD34+ cells					Early phase1	Recruiting	2023-10-25	2026-10-31	2024-06-03	12 Years - 35 Years	5	1	China	False	WO2023193616A1	First patient dosed (5/27/24)	News and Press Releases : https://reforgene.com/news/97-cn.html
NCT05345171	Ornithine Transcarbamylase Deficiency, Late Onset	Avalotcagene ontaparvovec (DTX301)	Ultragenyx Pharmaceutical Inc	Industry	OTC	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV8		1.0 x 10^13 GC/kg					Phase3	Recruiting	2022-04-18	2028-12	2024-10-21	>= 12 Years	50	25	Canada, Argentina, Netherlands, United States, Japan, Brazil, Italy, United Kingdom, France, Portugal, Germany, Spain	True	EP4038194A1	Enrollment expected to be completed H2 2024	Preclinical Publications : PMID: 35211641 News and Press Releases : https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-reports-second-quarter-2024-financial-results-and Clinical Publications : PMID: 37626723 Protocol : https://cdn.clinicaltrials.gov/large-docs/44/NCT02991144/SAP_001.pdf https://cdn.clinicaltrials.gov/large-docs/44/NCT02991144/Prot_000.pdf
NCT02716246	Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)	Rebisufligene etisparvovec (scAAV9.U1a.hSGSH)	Ultragenyx Pharmaceutical Inc	Industry	SGSH	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV9		0.5 x 10^13 vg/kg	1.0 x 10^13 vg/kg	3.0 x 10^13 vg/kg			Phase3, Phase2	Active not recruiting	2016-03-17	2025-01	2024-07-16		28	5	United States, Australia, Spain	True	US20220347298A1	Accelerated Approval BLA filing planned for 2024/early 2025	Grant : U01 NS064096 R21 NS081173 R01 NS093941 Preclinical Publications : PMID: 33339477 PMID: 32884151 PMID: 27331076 News and Press Releases : https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-plans-file-accelerated-approval-ux111 https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-reports-second-quarter-2024-financial-results-and
NCT05139316	Glycogen Storage Disease Type Ia	Pariglasgene brecaparvovec (DTX401)	Ultragenyx Pharmaceutical Inc	Industry	G6PC	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV8		1.0 x 10^13 GC/kg					Phase3	Recruiting	2021-07-14	2026-02	2024-10-26	>= 8 Years	52	20	Canada, Netherlands, United States, Japan, Brazil, Denmark, Italy, Germany, Spain	True	US20220017922A1	Marketing application expected 2025	Preclinical Publications : PMID: 30714174 PMID: 32430177 News and Press Releases : https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-positive-top-line-results-phase-3-study https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-reports-second-quarter-2024-financial-results-and Clinical Publications : PMID: 32655139 https://ir.ultragenyx.com/static-files/b8822aaa-3ce0-4342-8474-e1b71d237f18 Protocol : https://cdn.clinicaltrials.gov/large-docs/85/NCT03517085/Prot_000.pdf https://cdn.clinicaltrials.gov/large-docs/85/NCT03517085/SAP_001.pdf
NCT04884815	Wilson's Disease	Rivunatpagene miziparvovec (UX701)	Ultragenyx Pharmaceutical Inc	Industry	ATP7B	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV9		5.0 x 10^12 GC/kg	1.0 x 10^13 GC/kg	2.0 x 10^13 GC/kg	Undisclosed dose 4		Phase2, Phase1	Active not recruiting	2021-05-07	2031-11	2024-08-14	>= 18 Years	78	17	Canada, United States, United Kingdom, Portugal, Spain	True	EP3906066B1	All patients in Phase I have been dosed, data expected H2 2024, expansion dose selection to follow	News and Press Releases : https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-provides-update-stage-1-cohorts-pivotal-phase-123 https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-reports-second-quarter-2024-financial-results-and https://ir.ultragenyx.com/news-releases/news-release-details/ultragenyx-announces-completion-dosing-across-stage-1-cohorts
NCT06270316	Fabry Disease	AMT-191	UniQure Biopharma B.V.	Industry	GLA	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV5		6.0 x 10^13 gc/kg	3.0 x 10^14 gc/kg				Phase2, Phase1	Recruiting	2023-12-19	2027-04-29	2024-10-31	18 Years - 50 Years	12	2	United States	True	WO2015060722A1; WO2020104424A1	First patient dosed 8/15/24; FDA granted Orphan Drug Designation 09/2024	Preclinical Publications : https://www.liebertpub.com/doi/full/10.1089/hum.2021.29180.abstracts News and Press Releases : https://www.uniqure.com/investors-media/press-releases
NCT06100276	Amyotrophic Lateral Sclerosis	AMT-162	UniQure Biopharma B.V.	Industry	MiSOD1	Gene transfer	In-vivo	MiRNA knockdown of mutant/aberrant gene	Intrathecal	Viral vector		Viral transduction	AAVrh10		Undisclosed low dose	Undisclosed intermediate dose	Undisclosed high dose			Phase2, Phase1	Recruiting	2023-10-20	2031-03-30	2024-10-15	>= 18 Years	20	10	United States	True		First patient dosed October 2024	Grant : R21 NS098131 Preclinical Publications : PMID: 31970202 PMID: 32498636 https://www.liebertpub.com/doi/full/10.1089/hum.2021.29180.abstracts News and Press Releases : https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-and-apic-bio-enter-global-licensing-agreement-apb-102 https://www.uniqure.com/investors-media/press-releases
NCT05243017	Huntington's Disease, Early Manifest	AMT-130 (rAAV5-miHTT)	UniQure Biopharma B.V.	Industry	MiHTT	Gene transfer	In-vivo	MiRNA knockdown of mutant/aberrant gene	Intraparenchymal	Viral vector		Viral transduction	AAV5		6 x 10^12 gc/subject	6 x 10^13 gc/subject				Phase2, Phase1	Recruiting	2021-11-01	2029-10-07	2024-10-24	25 Years - 65 Years	14	6	Poland, United Kingdom, Germany	False	US20230119344A1; US20220213482A1; US20210371862A1	FDA granted RMAT designation Q2 2024	News and Press Releases : https://uniqure.gcs-web.com/news-releases/news-release-details/uniqure-announces-positive-interim-data-update-demonstrating Clinical Publications : PMID: 38489195 https://www.uniqure.com/assets/uploads/AMT-130-IA-update-July-2024.pdf
NCT04601974	Drug Resistant Epilepsy	Lenti-CAMK2A-KCNA1	University College, London	Other	KCNA1	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intracranial	Viral vector		Viral transduction	LV		Undisclosed dose (single administration)					Phase2, Phase1	Not yet recruiting	2020-10-06	2032-09	2023-05-15	>= 18 Years	10	0		False			Preclinical Publications : PMID: 30755487 PMID: 23147003
NCT05092685	Ornithine Transcarbamylase Deficiency	BGT-OTCD (AAVLK03hOTC)	University College, London	Other	OTC	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	LK03		6 x 10^11 vg/kg	2 x 10^12 vg/kg	6 x 10^12 vg/kg			Phase2, Phase1	Recruiting	2021-09-28	2027-06-30	2023-11-07	0 Days - 16 Years	12	1	United Kingdom	False	US20220372512A1; US20230093183A1	Granted ODD by FDA and EMA	Preclinical Publications : PMID: 36700121 PMID: 34703837 News and Press Releases : https://bloomsburygtx.com/bloomsbury-genetic-therapies-announces-initiation-of-recruitment-in-phase-1-2-horace-clinical-trial-of-investigational-gene-therapy-bgt-otcd-for-the-treatment-of-ornithine-transcarbamylase-deficiency/
NCT03001830	Hemophilia A	AAV2/8-HLP-FVIII-V3	University College, London	Other	F8	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector		Viral transduction	AAV2/8		6 x 10^11 vg/kg	2 x 10^12 vg/kg	4 x 10^12 vg/kg	6 x 10^12 vg/kg		Phase2, Phase1	Recruiting	2016-12-09	2029-12	2023-10-25	>= 18 Years	18	4	United States, United Kingdom	True			Clinical Publications : https://ashpublications.org/blood/article/142/Supplement%201/3624/500955/GO-8-Stable-Expression-of-Factor-VIII-over-5-Years
NCT02234934	Chronic Granulomatous Disease, X-linked	PCCLChimGp91/VSVg lentiviral vector (formerly OTL-102)	University of California, Los Angeles	Other	CYBB	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	VSV-G		Transduced CD34+ cells (6.5-32.6 x 10^6 cells/kg)					Phase2, Phase1	Recruiting	2014-09-04	2028-12	2023-11-18	>= 23 Months	16	3	United States	True	US20220378937A1	Commercial rights are owned by Orchard, which has deprioritized the program	Grant : CLIN2-08231 Preclinical Publications : PMID: 36332160 PMID: 29664709 PMID: 24557920 PMID: 20978475 News and Press Releases : https://ir.orchard-tx.com/news-releases/news-release-details/orchard-therapeutics-extends-runway-2024-focusing-hsc-gene/#:~:text=It%20was%20the%20first%20ex,and%20Drug%20Administration%20(FDA). Clinical Publications : PMID: 31988463
NCT05432310	Severe Combined Immunodeficiency, ADA-deficient	Simoladagene autotemcel (formerly OTL-101)	University of California, Los Angeles	Other	ADA	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells, minimum dose: Fresh (1 x 10^6 cells/kg); Cryopreserved (2 x 10^6 cells/kg)					Phase2, Phase1	Recruiting	2022-06-04	2026-12-31	2024-05-16	>= 1 Month	20	1	United States	True		Administered Under Compassionate Use	Grant : AC1-07675 (CIRM) U01 AI100801 CLIN2-09339 R01 AI074043 Preclinical Publications : PMID: 24256635 News and Press Releases : https://www.cnn.com/2023/04/27/health/bubble-boy-treatment-fight/index.html Clinical Publications : PMID: 33974366 Protocol : https://www.nejm.org/doi/suppl/10.1056/NEJMoa2027675/suppl_file/nejmoa2027675_protocol.pdf https://cdn.clinicaltrials.gov/large-docs/71/NCT01852071/Prot_000.pdf https://cdn.clinicaltrials.gov/large-docs/71/NCT01852071/SAP_001.pdf
NCT03897361	Cystinosis	CTNS-RD-04 or CTNS-RD-04-LB	University of California, San Diego	Other	CTNS	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Min dose: 3 x 10^6 CD34+ cells/kg					Phase2, Phase1	Active not recruiting	2019-02-06	2024-11	2024-06-12	>= 18 Years	6	1	United States	True	US20240009247A1	AVROBIO sold product to Novartis in May 2023	Grant : R01 DK099338 R21 DK090548 R01 DK090058 R01DK090058 CLIN2-11478 Preclinical Publications : PMID: 23089735 PMID: 25186209 News and Press Releases : https://www.fiercebiotech.com/biotech/novartis-buy-avrobio-gene-therapy-88m-leaving-rest-biotech-shelf Clinical Publications : PMID:34943781
NCT03538899	Severe Combined Immunodeficiency, DCLRE1C Deficient	AProArt (endogenous Artemis promotor)	University of California, San Francisco	Other	DCLRE1C	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Transduced CD34+ cells					Phase2, Phase1	Recruiting	2018-05-03	2038-06	2024-02-12	>= 2 Months	25	1	United States	True			Grant : CLIN2-10830 U54 AI082973 R01 AI063340 Preclinical Publications : PMID: 27611239 PMID: 25738323 PMID: 21663454 PMID: 20163250 News and Press Releases : https://clinicaltrials.ucsf.edu/trial/NCT03538899 Clinical Publications : PMID: 36546626 Protocol : https://www.nejm.org/doi/suppl/10.1056/NEJMoa2206575/suppl_file/nejmoa2206575_protocol.pdf
NCT02240407	Glycogen Storage Disease Type 2 (Late-Onset Pompe Disease)	RAAV9-DES-hGAA	University of Florida	Other	GAA	Gene transfer	In-vivo	Functional gene replacement	Intramuscular	Viral vector		Viral transduction	AAV9		4.6 x 10^13 vg/TA muscle					Phase1	Completed	2014-09-11	2021-08-26	2022-04-05	18 Years - 50 Years	2	1	United States	True	US20220347297A1		Grant : U01 HL121842 Preclinical Publications : PMID: 24021025 Clinical Publications : PMID: 26390092
NCT04201405	Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)	OTL-201 (Autologous CD34+ cells transduced with LV-CD11b-hSGSH)	University of Manchester	Other	SGSH	Gene transfer	Ex-vivo	Functional gene replacement	Intravenous	Autologous cells	CD34+ cells	Viral transduction	LV		Dose range: 4.37 - 22.7 x 10^6 CD34+ cells/kg					Phase2, Phase1	Active not recruiting	2019-12-05	2024-10-30	2024-02-13	3 Months - 24 Months	5	1	United Kingdom	False			Preclinical Publications : PMID: 31044143 PMID: 23748415 Clinical Publications : https://ir.orchard-tx.com/static-files/f67bb3e2-eff2-4bd6-9326-247884a1667c
NCT02317887	Retinoschisis, X-linked	RS1 AAV Vector (AAV8-scRS/IRBPhRS)	VegaVect, Inc.	Industry	RS1	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV8		1 x 10^9 vg/eye	1 x 10^10 vg/eye	1 x 10^11 vg/eye	< 3 x 10^11 vg/eye	< 6 x 10^11 vg/eye	Phase2, Phase1	Active not recruiting	2014-12-16	2025-07-31	2024-04-26	>= 18 Years	12	1	United States	True	US9873893B2; US20210290433A1; WO2019144077A1; US20180214577A1		Preclinical Publications : PMID: 36227606 PMID: 27409484 PMID: 27036983 Clinical Publications : PMID: 36227606 PMID: 34390869 PMID: 33601057 PMID: 30196853 PMID: 27409484 PMID: 27036983
NCT06164730	Familial Hypercholesterolemia, Heterozygous	VERVE-102	Verve Therapeutics, Inc.	Industry	PCSK9	Gene editing	In-vivo	Gene inactivation	Intravenous	MRNA, LNP		Lipid encapsulation	LDLR + GalNAc	base editor	Undisclosed dose 1	Undisclosed dose 2	Undisclosed dose 3	Undisclosed dose 4		Phase1	Recruiting	2023-12-01	2026-08	2024-06-24	18 Years - 65 Years	36	3	Canada, United Kingdom	True	US12029795B2; US20240010609A1; US20240131166A1; US20240011023A1	First patient dosed in Phase 1b trial May 2024	Preclinical Publications : https://www.vervetx.com/sites/default/files/2024-05/TIDES_5.17.24.pdf News and Press Releases : https://ir.vervetx.com/news-releases/news-release-details/verve-therapeutics-announces-dosing-first-patient-heart-2-phase https://ir.vervetx.com/news-releases/news-release-details/verve-therapeutics-announces-pipeline-progress-and-reports-1
NCT05398029	Familial Hypercholesterolemia, Heterozygous	VERVE-101	Verve Therapeutics, Inc.	Industry	PCSK9	Gene editing	In-vivo	Gene inactivation	Intravenous	MRNA, LNP	Hepatocyte	Lipid encapsulation	LDLR	ABE8.8m	0.1 mg/kg	0.3 mg/kg	0.45 mg/kg	0.6 mg/kg		Phase1	Active not recruiting	2022-05-19	2024-12	2024-04-08	18 Years - 75 Years	44	3	New Zealand, United Kingdom	True	US12029795B2; US20240010609A1; US20240131166A1; US20240011023A1	Update given during ASGCT 2024 on the off-target assessments done by VERVE	Preclinical Publications : PMID: 36314243 https://www.vervetx.com/sites/default/files/2024-05/VERVE-101%20Off-Target_ASGCT%20for%20website.pdf https://www.vervetx.com/sites/default/files/2024-07/Japan%20Atherosclerosis%20Conference_FINAL.pdf News and Press Releases : https://ir.vervetx.com/news-releases/news-release-details/verve-therapeutics-announces-pipeline-progress-and-reports-1
NCT06451770	Refractory Hypercholesterolemia	VERVE-201	Verve Therapeutics, Inc.	Industry	ANGPTL3	Gene editing	In-vivo	Gene inactivation	Intravenous	MRNA, LNP	Hepatocyte	Lipid encapsulation	LDLR + GalNAc	ABE	Undisclosed dose 1	Undisclosed dose 2	Undisclosed dose 3	Undisclosed dose 4		Phase1	Not yet recruiting	2024-06-04	2027-03	2024-06-11	18 Years - 70 Years	36	0		True	US20240010609A1; US20230340435A1; WO2023049299A2		Preclinical Publications : https://www.vervetx.com/sites/default/files/2024-05/EAS%202024%20201%20Nonclinical%20FINAL.pdf https://www.vervetx.com/sites/default/files/2023-08/ESC%202023_Khera_VERVE-201%20Slides_Website%20Upload.pdf News and Press Releases : https://ir.vervetx.com/news-releases/news-release-details/verve-therapeutics-announces-pipeline-progress-and-reports-first
NCT06291935	Retinitis Pigmentosa	VG901 (AAV2.NN-CNGA1)	ViGeneron GmbH	Industry	CNGA1	Gene transfer	In-vivo	Functional gene replacement	Intravitreal	Viral vector		Viral transduction	AAV2		Undisclosed low dose	Undisclosed high dose				Phase1	Recruiting	2024-02-01	2025-12	2024-03-04	>= 18 Years	6	1	Germany	False	US12043848B2; US20220409744A1	First patient dosed 4/10/24	Preclinical Publications : PMID: 24664765 PMID: 22802073 News and Press Releases : https://vigeneron.com/press/vigeneron-announces-first-patient-dosed-in-phase-1b-clinical-trial-of-vg901-for-the-intravitreal-treatment-of-retinitis-pigmentosa/
NCT06300476	Stargardt Disease (Juvenile Macular Degeneration)	JWK006	West China Hospital	Other	ABCA4	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV8		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Phase2, Phase1	Active not recruiting	2024-03-03	2029-12-30	2024-03-08	10 Years - 18 Years	9	1	China	False	CN115074369A	Investigator-initiated trial conducted at West China Hospital	Preclinical Publications : PMID: 37227014
NCT06114056	Duchenne Muscular Dystrophy	JWK007	West China Hospital	Other	Micro-dystrophin	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intravenous	Viral vector		Viral transduction	AAVrh74		1.0 x 10^14 vg/kg	2.0 x 10^14 vg/kg				Phase1	Recruiting	2023-10-29	2028-12-31	2024-01-17	5 Years - 10 Years	6	1	China	False		Investigator-initiated trial conducted at West China Hospital
NCT06345898	Retinoschisis, X-linked	JWK002	West China Hospital	Other	RS1	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV8		Undisclosed low dose	Undisclosed medium dose	Undisclosed high dose			Early phase1	Recruiting	2024-03-28	2029-12-30	2024-07-23	5 Years - 18 Years	18	1	China	False		Investigator-initiated trial conducted at West China Hospital
NCT06302608	Bietti Crystalline Dystrophy	NGGT001	Xiamen Ophthalmology Center Affiliated to Xiamen University	Other	CYP4V2	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2		1.5 x 10^11 vg/eye	3.0 x 10^11 vg/eye				Early phase1	Active not recruiting	2024-02-18	2028-05-29	2024-03-12	>= 18 Years	6	1	China	False	WO2023116745A1		News and Press Releases : https://www.cgtlive.com/view/nggt-biotechnology-gene-therapy-nggt001-improves-vision-patients-bietti-crystalline-dystrophy Clinical Publications : https://annualmeeting.asgct.org/abstracts/abstract-details?abstractId=59427
NCT06272149	Gaucher Disease, Type 2	VGN-R08b	Xinhua Hospital, Shanghai Jiao Tong University School of Medicine	Other	GBA1	Gene transfer	In-vivo	Functional gene replacement	Intracerebroventricular	Viral vector		Viral transduction	AAV9		Undisclosed low dose	Undisclosed high dose				Early phase1	Recruiting	2023-07-17	2029-02-28	2024-02-22	0 Months - 24 Months	6	1	China	False	WO2024017387A1	FIH interim data presented at ASGCT 2024	News and Press Releases : http://www.vitalgen-biomed.com/en/details.php?id=16
NCT04125732	Refractory Angina	Encoberminogene rezmadenovec (XC001)	XyloCor Therapeutics, Inc.	Industry	VEGF	Gene transfer	In-vivo	Overexpression of protective allele/gene	Intramyocardial	Viral vector		Viral transduction	Ad5		1 x 10^9 vp	1 x 10^10 vp	4 x 10^10 vp	1 x 10^11 vp (Expansion Dose)		Phase2, Phase1	Completed	2019-10-04	2023-05-30	2024-01-30	18 Years - 80 Years	41	16	United States	True	US20240115732A1		Preclinical Publications : PMID: 14741779 PMID: 37592080 PMID: 23692382 PMID: 16039163 News and Press Releases : https://www.xylocor.com/news.html Clinical Publications : PMID: 38696284 PMID: 37503661 PMID: 34224684
NCT06539208	Transthyretin Amyloidosis, with Polyneuropathy/Cardiomyopathy	YOLT-201	YolTech Therapeutics Co., Ltd	Industry	TTR	Gene editing	In-vivo	Gene inactivation	Intravenous	MRNA, LNP	Liver	Lipid encapsulation	LNP	Cas9 mRNA	Undisclosed dose ascension					Phase2, Phase1	Recruiting	2024-08-01	2026-06-30	2024-08-06	18 Years - 80 Years	31	3	China	False		First patient dosed (6/28/24)	Preclinical Publications : PMID: 38874509 News and Press Releases : https://www.yoltx.com/news/press-release/65 https://www.yoltx.com/
NCT03328130	Retinitis Pigmentosa	CTX-PDE6B (HORA-PDE6B)	eyeDNA Therapeutics	Industry	PDE6B	Gene transfer	In-vivo	Functional gene replacement	Subretinal	Viral vector		Viral transduction	AAV2/5		3.4 x 10^11 vg/eye	6.4 x 10^11 vg/eye				Phase2, Phase1	Recruiting	2017-10-05	2029-12	2024-03-07	>= 13 Years	23	1	France	False			Clinical Publications : https://iovs.arvojournals.org/article.aspx?articleid=2794387&resultClick=1
NCT06255782	Ornithine Transcarbamylase Deficiency, Neonatal Onset	ECUR-506	iECURE, Inc.	Industry	OTC	Gene transfer	In-vivo	Functional gene replacement	Intravenous	Viral vector	Hepatocyte	Viral transduction	AAV8	ARCUS	Undisclosed low dose	Undisclosed high dose				Phase2, Phase1	Active not recruiting	2023-12-19	2026-09	2024-08-23	24 Hours - 7 Months	13	2	United Kingdom	True	US20240101986A1; EP3288594B1; US9493788B2	FDA granted Fast Track designation 5/7/24	Preclinical Publications : PMID: 32095520 PMID: 26829317 https://precisionbiosciences.com/wp-content/uploads/2022/10/Precision_ARCUS_whitepaper_FINAL-October-2022.pdf News and Press Releases : https://iecure.com/news/iecure-receives-fda-fast-track-designation-for-ecur-506-for-the-treatment-of-neonatal-onset-ornithine-transcarbamylase-otc-deficiency/
NCT06063850	Temporal Lobe Epilepsy	AMT-260	uniQure France SAS	Industry	MiGRIK2	Gene transfer	In-vivo	MiRNA knockdown of mutant/aberrant gene	Intraparenchymal	Viral vector		Viral transduction	AAV9		Undisclosed low dose	Undisclosed high dose				Phase2, Phase1	Recruiting	2023-09-01	2027-06-30	2023-11-14	18 Years - 65 Years	12	2	United States	True			Preclinical Publications : https://www.liebertpub.com/doi/full/10.1089/hum.2021.29180.abstracts PMID: 37341588 https://www.uniqure.com/assets/uploads/11.29.22_uniQure_RD_Investor_Event.pdf News and Press Releases : https://www.uniqure.com/investors-media/press-releases

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