SCGE Platform - Gene Therapy Clinical Trials

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.

Disclaimer: The information on this dashboard has been collected for the convenience of patients and researchers. The SCGE team are not medical doctors and cannot provide medical advice. Please discuss with your provider the risks/benefits of participating in a clinical trial, and do not send us your personal medical information. The information contained within this table does not make use of any confidential or privileged information-all data is collected from publicly available sources. The SCGE makes no comment as to the efficacy and safety of the items listed, as these are not known at the time of publication. For the most up to date information, or to inquire about enrollment, please refer to clinicaltrials.gov or the Sponsor's website for contact information.

Updates in the last 14 days

NCT07282847

2026-05-28

Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD

Recruiting

FDA accepted IND in January 2026

NCT06461702

2026-05-28

Familial Hypercholesterolemia

Unknown

FDA cleared IND application in June 2025

NCT06856759

2026-05-28

Rett Syndrome

Active not recruiting

NCT07052929

2026-05-28

X-Linked Myotubular Myopathy

Recruiting

Astellas has restarted their XLMTM program by creating a new drug product ASP2957, with substantial modifications to the capsid to detarget the liver,...

NCT07592273

2026-05-28

Diabetic Retinopathy

Recruiting

This product is also under evaluation for wet AMD

Showing 1-100 of 134 results in ClinicalTrials

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NCT07282847 Recruiting Phase2, Phase1

View Report

Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD

Sponsor AskBio Inc (Industry)

Compound AB-1009

Therapy Type Gene transfer

Target Gene GAA

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT07052929 Recruiting Phase2, Phase1

View Report

X-Linked Myotubular Myopathy

Sponsor Astellas Gene Therapies (Industry)

Compound ASP2957

Therapy Type Gene transfer

Target Gene MTM1

Vector Type AAV3.8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT07592273 Recruiting Phase3, Phase2

View Report

Diabetic Retinopathy

Sponsor AbbVie (Industry)

Compound Surabgene lomparvovec

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT05419492 Recruiting Phase2, Phase1

View Report

Dravet Syndrome

Sponsor Encoded Therapeutics (Industry)

Compound ETX101

Therapy Type Gene transfer

Target Gene SCN1A

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06539208 Recruiting Phase2, Phase1

View Report

Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy

Sponsor YolTech Therapeutics Co., Ltd (Industry)

Compound YOLT-201

Therapy Type Gene editing

Target Gene TTR

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Gene inactivation

NCT06389877 Recruiting Phase2, Phase1

View Report

Alpha-1 Antitrypsin Deficiency (AATD)

Sponsor Beam Therapeutics Inc. (Industry)

Compound BEAM-302

Therapy Type Gene editing

Target Gene SERPINA1 (p.Glu366Lys)

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Mutation correction

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation

NCT06164730 Recruiting Phase1

View Report

Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease

Sponsor Verve Therapeutics, Inc. (Industry)

Compound VERVE-102

Therapy Type Gene editing

Target Gene PCSK9

Vector Type LDLR + GalNAc

Delivery System Endocytosis

Mechanism of Action Gene inactivation

FDA Designations Fast Track

NCT06451770 Recruiting Phase1

View Report

Hypercholesterolemia

Sponsor Verve Therapeutics, Inc. (Industry)

Compound VERVE-201

Therapy Type Gene editing

Target Gene ANGPTL3

Vector Type LDLR + GalNAc

Delivery System Endocytosis

Mechanism of Action Gene inactivation

NCT07007065 Recruiting Phase3

View Report

Neovascular Age-related Macular Degeneration

Sponsor AbbVie (Industry)

Compound ABBV-RGX-314

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT06735755 Recruiting Phase2, Phase1

View Report

Glycogen Storage Disease Type Ia

Sponsor Beam Therapeutics Inc. (Industry)

Compound BEAM-301

Therapy Type Gene editing

Target Gene G6PC1 (p.R83C)

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Mutation correction

NCT06128629 Recruiting Phase3

View Report

Transthyretin Amyloidosis (ATTR) with Cardiomyopathy

Sponsor Intellia Therapeutics (Industry)

Compound NTLA-2001

Therapy Type Gene editing

Target Gene TTR

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Gene inactivation

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation

NCT06680232 Recruiting Phase1

View Report

Chronic Hepatitis B

Sponsor Precision BioSciences, Inc. (Industry)

Compound PBGENE-HBV

Therapy Type Gene editing

Target Gene HBV DNA

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Gene inactivation

FDA Designations Fast Track

NCT06517888 Recruiting Phase2, Phase1

View Report

Vestibular Schwannoma

Sponsor Akouos, Inc. (Industry)

Compound AAVAnc80-antiVEGF

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAVAnc80

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT06839235 Recruiting Phase2, Phase1

View Report

Primary Hyperoxaluria Type 1 (PH1)

Sponsor Arbor Biotechnologies (Industry)

Compound ABO-101

Therapy Type Gene editing

Target Gene HAO1

Vector Type LNP

Delivery System Endocytosis

Mechanism of Action Gene inactivation

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT07226206 Recruiting Phase2, Phase1

View Report

Hemophilia A

Sponsor Hoffmann-La Roche (Industry)

Compound SPK-8011QQ

Therapy Type Gene transfer

Target Gene F8

Vector Type LK03

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT01306019 Recruiting Phase2, Phase1

View Report

X-linked Severe Combined Immunodeficiency (XSCID)

Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)

Compound VSV-G pseudotyped CL20-i4-EF1α-hγc-OPT vector

Therapy Type Gene transfer

Target Gene IL2RG

Vector Type VSV-G

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06817382 Recruiting Phase1

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Insmed Gene Therapy LLC (Industry)

Compound INS1201

Therapy Type Gene transfer

Target Gene Micro-dystrophin

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06217861 Recruiting Phase1

View Report

Glutaric Acidemia Type I

Sponsor Shanghai Vitalgen BioPharma Co., Ltd. (Industry)

Compound VGM-R02b

Therapy Type Gene transfer

Target Gene GCDH

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT07287397 Recruiting Phase2, Phase1

View Report

Amyotrophic Lateral Sclerosis

Sponsor Vector Y Therapeutics (Industry)

Compound VTx-002

Therapy Type Gene transfer

Target Gene Antibody to TDP-43

Vector Type AAV5.2

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

FDA Designations Fast Track

NCT05504837 Recruiting Phase1

View Report

Cystic Fibrosis

Sponsor Krystal Biotech, Inc. (Industry)

Compound KB407

Therapy Type Gene transfer

Target Gene CFTR

Vector Type HSV-1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT07160634 Recruiting Phase3

View Report

Duchenne Muscular Dystrophy (DMD)

Sponsor Solid Biosciences Inc. (Industry)

Compound SGT-003

Therapy Type Gene transfer

Target Gene Micro-dystrophin

Vector Type AAV-SLB101

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Fast Track, Rare Pediatric Disease Designation

NCT06255782 Recruiting Phase2, Phase1

View Report

Ornithine Transcarbamylase (OTC) Deficiency

Sponsor iECURE, Inc. (Industry)

Compound ECUR-506

Therapy Type Gene transfer

Target Gene OTC/PCSK9 locus

Vector Type dual AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Chemistry, Manufacturing, and Controls Development and Readiness Pilot (CDRP) Program, Regenerative Medicine Advanced Therapy (RMAT), Fast Track

NCT05682144 Recruiting Phase1

View Report

Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS

Sponsor Immusoft of CA, Inc. (Industry)

Compound ISP-001

Therapy Type Gene transfer

Target Gene IDUA

Vector Type Sleeping Beauty

Delivery System Electroporation

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05878860 Recruiting Phase3

View Report

X-Linked Retinoschisis

Sponsor Atsena Therapeutics Inc. (Industry)

Compound ATSN-201

Therapy Type Gene transfer

Target Gene RS1

Vector Type AAV.SPR

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06064890 Recruiting Phase2, Phase1

View Report

Frontotemporal Dementia, FTD, FTD-GRN

Sponsor AviadoBio Ltd (Industry)

Compound AVB-101

Therapy Type Gene transfer

Target Gene GRN

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT02716246 Recruiting Phase3, Phase2

View Report

Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)

Sponsor Ultragenyx Pharmaceutical Inc (Industry)

Compound UX111

Therapy Type Gene transfer

Target Gene SGSH

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06285643 Recruiting Phase2

View Report

Parkinson's Disease

Sponsor AskBio Inc (Industry)

Compound AB-1005

Therapy Type Gene transfer

Target Gene GDNF

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track

NCT05926765 Recruiting Phase2

View Report

Grade 2 and 3 Radiation-Induced Late Xerostomia

Sponsor MeiraGTx, LLC (Industry)

Compound AAV-hAQP1

Therapy Type Gene transfer

Target Gene AQP1

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation

NCT05788536 Recruiting Phase2, Phase1

View Report

Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)

Sponsor Regeneron Pharmaceuticals (Industry)

Compound OTARMENI

Therapy Type Gene transfer

Target Gene OTOF

Vector Type dual AAV1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06506461 Recruiting Phase1

View Report

Sickle Cell Disease

Sponsor St. Jude Children's Research Hospital (Other)

Compound Gene-modified CD34+ cells

Therapy Type Gene editing

Target Gene BCL11A

Delivery System Electroporation

Mechanism of Action Overexpression of protective allele/gene

NCT06826612 Recruiting Phase2, Phase1

View Report

Huntington's Disease

Sponsor Hoffmann-La Roche (Industry)

Compound RG6662

Therapy Type Gene transfer

Target Gene MiHTT

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Gene inactivation

NCT06092034 Recruiting Phase2

View Report

Danon Disease

Sponsor Rocket Pharmaceuticals Inc. (Industry)

Compound RP-A501

Therapy Type Gene transfer

Target Gene LAMP2B

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06765980 Recruiting Phase2, Phase1

View Report

Geographic Atrophy Secondary to Age-related Macular Degeneration

Sponsor Kriya Therapeutics, Inc. (Industry)

Compound KRIYA-825

Therapy Type Gene transfer

Target Gene CR2-CR1

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT06844214 Recruiting Phase2, Phase1

View Report

Myotonic Dystrophy

Sponsor Sanofi (Industry)

Compound SAR446268

Therapy Type Gene transfer

Target Gene DMPK

Vector Type AAV.SAN011

Delivery System Viral transduction

Mechanism of Action Gene inactivation

FDA Designations Fast Track, Orphan Drug Designation

NCT07180355 Recruiting Phase1

View Report

Friedreich's Ataxia

Sponsor Solid Biosciences Inc. (Industry)

Compound SGT-212

Therapy Type Gene transfer

Target Gene FXN

Vector Type AAVhu68

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06910813 Recruiting Phase2, Phase1

View Report

Nephropathic Cystinosis

Sponsor Novartis Pharmaceuticals (Industry)

Compound DFT383

Therapy Type Gene transfer

Target Gene CTNS

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06692712 Recruiting Phase3

View Report

Hereditary Spastic Paraplegia Type 50

Sponsor Elpida Therapeutics SPC (Industry)

Compound MELPIDA

Therapy Type Gene transfer

Target Gene AP4M1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT07223944 Recruiting Phase3

View Report

Gaucher Disease Type 1

Sponsor Spur Therapeutics (Industry)

Compound FLT201

Therapy Type Gene transfer

Target Gene GBA1

Vector Type AAVS3

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT04998396 Recruiting Phase2, Phase1

View Report

Canavan Disease

Sponsor Aspa Therapeutics (Industry)

Compound BBP-812

Therapy Type Gene transfer

Target Gene ASPA

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT04774536 Recruiting Phase2, Phase1

View Report

Sickle Cell Disease

Sponsor Mark Walters, MD (Other)

Compound CRISPR_SCD001

Therapy Type Gene editing

Target Gene HBB

Vector Type none

Delivery System Electroporation

Mechanism of Action Mutation correction

NCT07064759 Recruiting Phase3

View Report

Neovascular Age-Related Macular Degeneration (nAMD)

Sponsor 4D Molecular Therapeutics (Industry)

Compound 4D-150

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT06364774 Recruiting Phase2, Phase1

View Report

Beta-Thalassemia Major

Sponsor Children's Hospital of Philadelphia (Other)

Compound CHOP-ALS20

Therapy Type Gene transfer

Target Gene HBB

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05885412 Recruiting Phase1

View Report

PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)

Sponsor Rocket Pharmaceuticals Inc. (Industry)

Compound RP-A601

Therapy Type Gene transfer

Target Gene PKP2

Vector Type AAVrh.74

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation

NCT07048808 Recruiting Phase2

View Report

Refractory Angina, Coronary Artery Disease

Sponsor XyloCor Therapeutics, Inc. (Industry)

Compound XC001

Therapy Type Gene transfer

Target Gene VEGF

Vector Type Ad5

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT05432310 Recruiting Phase2, Phase1

View Report

Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Sponsor University of California, Los Angeles (Other)

Compound Simoladagene autotemcel

Therapy Type Gene transfer

Target Gene ADA

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05606614 Recruiting Phase3

View Report

Rett Syndrome

Sponsor Taysha Gene Therapies, Inc. (Industry)

Compound TSHA-102

Therapy Type Gene transfer

Target Gene MiniMECP2

Vector Type scAAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06325709 Recruiting Phase2, Phase1

View Report

X-Linked Chronic Granulomatous Disease

Sponsor National Institute of Allergy and Infectious Diseases (NIAID) (NIH)

Compound Base-edited autologous CD34+ cells

Therapy Type Gene editing

Target Gene CYBB c.676 C>T

Vector Type none

Delivery System Electroporation

Mechanism of Action Mutation correction

NCT06063850 Recruiting Phase2, Phase1

View Report

Mesial Temporal Lobe Epilepsy

Sponsor UniQure Biopharma B.V. (Industry)

Compound AMT-260

Therapy Type Gene transfer

Target Gene MiGRIK2

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action MiRNA knockdown of mutant/aberrant gene

NCT06458595 Recruiting Phase2, Phase1

View Report

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

Sponsor Chengdu Origen Biotechnology Co., Ltd. (Industry)

Compound KH658

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT06662188 Recruiting Phase2, Phase1

View Report

SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome

Sponsor Jaguar Gene Therapy, LLC (Industry)

Compound JAG201

Therapy Type Gene transfer

Target Gene SHANK3

Vector Type AAV2/9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Rare Pediatric Disease Designation

NCT06185673 Recruiting Phase2, Phase1

View Report

Oculopharyngeal Muscular Dystrophy

Sponsor Benitec Biopharma, Inc. (Industry)

Compound BB-301

Therapy Type Gene transfer

Target Gene PABPN1

Vector Type AAV9-PL

Delivery System Viral transduction

Mechanism of Action Functional gene replacement, Gene inactivation

FDA Designations Fast Track, Orphan Drug Designation

NCT06292650 Recruiting Early phase1

View Report

Retinitis Pigmentosa

Sponsor Zhongmou Therapeutics (Industry)

Compound ZM-02

Therapy Type Gene transfer

Target Gene Ch2.0

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Orphan Drug Designation

NCT05071222 Recruiting Phase2, Phase1

View Report

Artemis (DCLRE1C) Deficient Severe Combined Immunodeficiency

Sponsor Assistance Publique - Hôpitaux de Paris (Other)

Compound ARTEGENE

Therapy Type Gene transfer

Target Gene DCLRE1C

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT07185256 Recruiting Phase2, Phase1

View Report

ARB, BVMD, Autosomal-Dominant Bestrophinopathy, Best Vitelliform Macular Dystrophy

Sponsor Opus Genetics, Inc (Industry)

Compound OPGx-BEST1

Therapy Type Gene transfer

Target Gene BEST1

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05477563 Recruiting Phase3

View Report

Beta-Thalassemia, Sickle Cell Disease

Sponsor Vertex Pharmaceuticals Incorporated (Industry)

Compound CASGEVY

Therapy Type Gene editing

Target Gene BCL11A

Vector Type RNP

Delivery System Electroporation

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05836259 Recruiting Phase2, Phase1

View Report

Hypertrophic Cardiomyopathy

Sponsor Tenaya Therapeutics (Industry)

Compound TN-201

Therapy Type Gene transfer

Target Gene MYBPC3

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05898620 Recruiting Phase3

View Report

Rett Syndrome

Sponsor Neurogene Inc. (Industry)

Compound NGN-401

Therapy Type Gene transfer

Target Gene MECP2

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Support for Clinical Trials Advancing Rare Disease Therapeutics (START) Pilot, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT02852213 Recruiting Phase1

View Report

Aromatic L-amino Acid Decarboxylase (AADC) Deficiency

Sponsor Krzysztof Bankiewicz (Other)

Compound AAV2-hAADC

Therapy Type Gene transfer

Target Gene DDC

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06224660 Recruiting Phase1

View Report

DMD-Associated Dilated Cardiomyopathy

Sponsor Sardocor Corp. (Industry)

Compound SRD-001

Therapy Type Gene transfer

Target Gene SERCA2a

Vector Type AAV1

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Orphan Drug Designation

NCT03727555 Recruiting Na

View Report

X-linked Adrenoleukodystrophy

Sponsor Shenzhen Geno-Immune Medical Institute (Other)

Compound (LV) TYF-ABCD1

Therapy Type Gene transfer

Target Gene ABCD1

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05986864 Recruiting Phase2, Phase1

View Report

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

Sponsor Skyline Therapeutics (US) Inc. (Industry)

Compound SKG0106

Therapy Type Gene transfer

Target Gene Anti-VEGF

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT05761899 Recruiting Phase2, Phase1

View Report

Hereditary Pulmonary Alveolar Proteinosis

Sponsor Children's Hospital Medical Center, Cincinnati (Other)

Compound Gene-Corrected Macrophages

Therapy Type Gene transfer

Target Gene CSF2RA

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03311503 Recruiting Phase2, Phase1

View Report

X-linked Severe Combined Immunodeficiency (XSCID)

Sponsor David Williams (Other)

Compound G2SCID lentiviral vector transduced CD34+ cells

Therapy Type Gene transfer

Target Gene IL2RG

Vector Type HIV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06818838 Recruiting Phase2, Phase1

View Report

Gaucher Disease Type 1

Sponsor Lingyi Biotech Co., Ltd. (Industry)

Compound LY-M001

Therapy Type Gene transfer

Target Gene GBA1

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06399107 Recruiting Phase2, Phase1

View Report

Sickle Cell Disease, Sickle-Cell Disease With Crisis

Sponsor Essen Biotech (Other)

Compound BAH243

Therapy Type Gene transfer

Target Gene HBB

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

NCT06749639 Recruiting Early phase1

View Report

Inherited Retinal Diseases, RDH12 Retinopathy

Sponsor Peking Union Medical College Hospital (Other)

Compound PUMCH-E101

Therapy Type Gene transfer

Target Gene RDH12

Vector Type undisclosed

Delivery System Undisclosed

Mechanism of Action Functional gene replacement

NCT06474442 Recruiting Phase2

View Report

Herpes Simplex Virus Type I Stromal Keratitis

Sponsor Shanghai BDgene Co., Ltd. (Industry)

Compound BD111

Therapy Type Gene editing

Target Gene HSV genes

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Gene excision

FDA Designations Orphan Drug Designation

NCT06238908 Recruiting Early phase1

View Report

Hemophilia A

Sponsor Institute of Hematology & Blood Diseases Hospital, China (Other)

Compound NGGT003

Therapy Type Gene transfer

Target Gene F8

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT03952637 Recruiting Phase2, Phase1

View Report

GM1 Gangliosidosis

Sponsor National Human Genome Research Institute (NHGRI) (NIH)

Compound AXO-AAV-GM1

Therapy Type Gene transfer

Target Gene GLB1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05906953 Recruiting Phase2, Phase1

View Report

Leber Congenital Amaurosis, Inherited Retinal Diseases Caused by RPE65 Mutations

Sponsor HuidaGene Therapeutics Co., Ltd. (Industry)

Compound HG004

Therapy Type Gene transfer

Target Gene RPE65

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05230459 Recruiting Phase2, Phase1

View Report

Limb-Girdle Muscular Dystrophy, Type 2I/R9

Sponsor AskBio Inc (Industry)

Compound AB-1003

Therapy Type Gene transfer

Target Gene FKRP

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06831825 Recruiting Phase1

View Report

Heart Failure With Reduced Ejection Fraction

Sponsor YAP Therapeutics, Inc. (Industry)

Compound YAP101

Therapy Type Gene transfer

Target Gene SAV1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Gene inactivation

NCT06526923 Recruiting Phase2, Phase1

View Report

Cystic Fibrosis

Sponsor Spirovant Sciences, Inc. (Industry)

Compound SP-101

Therapy Type Gene transfer

Target Gene CFTRΔR

Vector Type AAV2.5T

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05672121 Recruiting Phase2, Phase1

View Report

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

Sponsor Chengdu Origen Biotechnology Co., Ltd. (Industry)

Compound KH631

Therapy Type Gene transfer

Target Gene VEGFR1/R2 fusion protein

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT05762510 Recruiting Early phase1

View Report

Beta-Thalassemia Major

Sponsor First Affiliated Hospital of Guangxi Medical University (Other)

Compound GMCN-508B

Therapy Type Gene transfer

Target Gene HBB

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06332807 Recruiting Phase2, Phase1

View Report

Phenylketonuria (PKU)

Sponsor NGGT INC. (Industry)

Compound NGGT002

Therapy Type Gene transfer

Target Gene PAH

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT06111638 Recruiting Phase3, Phase2

View Report

Hemophilia A

Sponsor Shanghai Xinzhi BioMed Co., Ltd. (Industry)

Compound BBM-H803

Therapy Type Gene transfer

Target Gene F8

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT05858983 Recruiting Phase2, Phase1

View Report

Biallelic RPE65 Mutation-associated Retinal Dystrophy

Sponsor Frontera Therapeutics (Industry)

Compound FT-001

Therapy Type Gene transfer

Target Gene RPE65

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06280378 Recruiting Phase2, Phase1

View Report

Beta-Thalassemia Major

Sponsor Kanglin Biotechnology (Hangzhou) Co., Ltd. (Industry)

Compound KL003

Therapy Type Gene transfer

Target Gene HBB

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT04676048 Recruiting Phase2, Phase1

View Report

Hemophilia A

Sponsor ASC Therapeutics (Industry)

Compound ASC618

Therapy Type Gene transfer

Target Gene F8

Vector Type AAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT06272149 Recruiting Early phase1

View Report

Type II Gaucher Disease

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

Compound VGN-R08b

Therapy Type Gene transfer

Target Gene GBA1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Rare Pediatric Disease Designation

NCT03645460 Recruiting Na

View Report

Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)

Sponsor Shenzhen Geno-Immune Medical Institute (Other)

Compound ADA lentiviral vector

Therapy Type Gene transfer

Target Gene ADA

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06207552 Recruiting Early phase1

View Report

Fabry Disease

Sponsor Children's Hospital of Fudan University (Other)

Compound BBM-F101

Therapy Type Gene transfer

Target Gene GLA

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06291935 Recruiting Phase1

View Report

Retinitis Pigmentosa

Sponsor VeonGen Therapeutics GmbH (Industry)

Compound VG901

Therapy Type Gene transfer

Target Gene CNGA1

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06049082 Recruiting Phase1

View Report

Alpha-1 Antitrypsin Deficiency (AATD)

Sponsor Krystal Biotech, Inc. (Industry)

Compound KB-408

Therapy Type Gene transfer

Target Gene SERPINA1

Vector Type HSV-1

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation

NCT04278131 Recruiting Phase2, Phase1

View Report

Retinitis Pigmentosa

Sponsor Bionic Sight LLC (Industry)

Compound BS01

Therapy Type Gene transfer

Target Gene ChronosFP

Vector Type AAV2

Delivery System Viral transduction

Mechanism of Action Overexpression of protective allele/gene

FDA Designations Regenerative Medicine Advanced Therapy (RMAT)

NCT06492850 Recruiting Phase2, Phase1

View Report

X-Linked Retinitis Pigmentosa (XLRP)

Sponsor Frontera Therapeutics (Industry)

Compound FT-002

Therapy Type Gene transfer

Target Gene RPGRORF15

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Fast Track, Orphan Drug Designation

NCT06288230 Recruiting Phase2, Phase1

View Report

Spinal Muscular Atrophy

Sponsor Lantu Biopharma (Industry)

Compound Vesemnogene lantuparvovec

Therapy Type Gene transfer

Target Gene SMN1

Vector Type AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06511349 Recruiting Early phase1

View Report

Type 1 Primary Hyperoxaluria

Sponsor RenJi Hospital (Other)

Compound YOLT-203

Therapy Type Gene editing

Target Gene HAO1

Vector Type LNP

Delivery System Lipid encapsulation

Mechanism of Action Gene inactivation

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06641154 Recruiting Phase2, Phase1

View Report

Crigler-Najjar Syndrome Type I

Sponsor Federal State Budget Institution Research Center for Obstetrics, Gynecology and Perinatology Ministry of Healthcare (Other gov)

Compound GT-UGT1A1-AAV8-02

Therapy Type Gene transfer

Target Gene UGT1A1

Vector Type rAAV2/8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT05616793 Recruiting Phase2, Phase1

View Report

Leber Congenital Amaurosis-Type 5

Sponsor Opus Genetics, Inc (Industry)

Compound OPGx-LCA5

Therapy Type Gene transfer

Target Gene LCA5

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06308159 Recruiting Phase2, Phase1

View Report

Beta-Thalassemia Major

Sponsor Lantu Biopharma (Industry)

Compound Vebeglogene autotemcel

Therapy Type Gene transfer

Target Gene HBB

Vector Type LV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06860672 Recruiting Early phase1

View Report

Snijders Blok-Campeau syndrome

Sponsor Yongguo Yu (Other)

Compound Dual Vector AAV-CHD3-R1025W Base Editor

Therapy Type Gene editing

Target Gene CDH3/R1025W

Vector Type dual AAV

Delivery System Viral transduction

Mechanism of Action Mutation correction

NCT05791864 Recruiting Phase2, Phase1

View Report

Neuronal Ceroid Lipofuscinosis Type 2

Sponsor Tern Therapeutics, LLC (Industry)

Compound TTX-381

Therapy Type Gene transfer

Target Gene TPP1

Vector Type AAV9

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06492863 Recruiting Phase2, Phase1

View Report

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

Sponsor Frontera Therapeutics (Industry)

Compound FT-003

Therapy Type Gene transfer

Target Gene Aflibercept

Vector Type AAV2.7m8

Delivery System Viral transduction

Mechanism of Action Genetic delivery of therapeutic protein

NCT06519552 Recruiting Phase1

View Report

Mucopolysaccharidosis Type I (Hurler Syndrome)

Sponsor West China Hospital (Other)

Compound JWK-008

Therapy Type Gene transfer

Target Gene IDUA

Vector Type AAV5

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06650319 Recruiting Early phase1

View Report

Wilson Disease

Sponsor Chaohui Yu (Other)

Compound LY-M003

Therapy Type Gene transfer

Target Gene ATP7B

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT06370351 Recruiting Phase2, Phase1

View Report

OTOF Gene Mutation, DFNB9, Congenital Deafness, Hearing Disorders

Sponsor Sensorion (Industry)

Compound SENS-501

Therapy Type Gene transfer

Target Gene OTOF

Vector Type dual AAV

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

NCT06345898 Recruiting Early phase1

View Report

X-Linked Retinoschisis

Sponsor West China Hospital (Other)

Compound JWK002

Therapy Type Gene transfer

Target Gene RS1

Vector Type AAV8

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

NCT05248230 Recruiting Phase2

View Report

Cystic Fibrosis

Sponsor 4D Molecular Therapeutics (Industry)

Compound 4D-710

Therapy Type Gene transfer

Target Gene CFTR

Vector Type AAV A101

Delivery System Viral transduction

Mechanism of Action Functional gene replacement

FDA Designations Orphan Drug Designation, Rare Pediatric Disease Designation

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AAV	Adeno-associated virus, e.g. AAV2, AAV5, AAV2/5 indicates virus containing the genome of serotype 2 packaged in the capsid from serotype 5
ABE	Adenine base editor
Ad	Adenovirus
Cas9	CRISPR associated protein 9
CBE	Cytosine base editor
CRISPR	Clustered regularly interspaced short palindromic repeats
Gc	Genome copies
HIV	Human immunodeficiency virus
HSV	Herpes simplex virus
LNP	Lipid nanoparticle
LV	Lentivirus
MRNA	Messenger ribonucleic acid
ODD	Orphan Drug Designation
PFU	Plaque forming units
RMAT	Regenerative Medicine Advanced Therapy
RNP	Ribonucleoprotein
RPDD	Rare Pediatric Disease Designation
RV	Retrovirus
START	Support for Clinical Trials Advancing Rare Disease Therapeutics
Vg	Vector genomes
VSV-G	Vesicular stomatitis virus G

Electroporation	Cell membrane permeablized by electrical field to allow gene therapy to enter the cell
Lipid encapsulation	Any lipid nanoparticle used to deliver editor, corrected gene
Microinjection	Drug is injected into individual cells
Plasmid	Any plasmid used to deliver editor, corrected gene
Viral transduction	Any virus used to deliver editor, corrected gene, etc.

Industry	All for-profit entities
NIH	U.S. National Institutes of Health
Other Non-Profit	Includes individuals, universities, community-based organizations

CED	Convection enhanced delivery to the brain
Inhalational	Delivered to the lungs in the form of a fine spray
Intraarterial	Into the lumen of an artery
Intraarticular	Into a joint space
Intracerebroventricular	Into the ventricles of the brain (ICV)
Intracisterna magna	Into the cisterna magna of the brain
Intracochlear	Into the cochlea of the ear
Intradermal	Into the dermal layer of the skin
Intragastric	Into the stomach
Intramuscular	Into a skeletal muscle
Intraocular	Administered into the eye
Intraparenchymal	Into the brain
Intraperitoneal	Into the peritoneal cavity
Intrastromal	Into the stroma of the cornea
Intrathecal	Into the spinal canal
Intravenous	Into a vein
Intravesicular	Into the bladder
Intravitreal	Into the eye (IVT)
Subcutaneous	Under the skin
Subretinal	Under the sensory retina
Suprachoroidal	Into the suprachoroidal space between the sclera and the choroid of the eye
Topical	Applied to the outer layer of the skin

Early Phase I	Describes exploratory trials conducted before traditional Phase I trials to investigate how or whether a drug affects the body, have no therapeutic or diagnostic goals
Phase I	Describes clinical trials that focus on the safety of a drug
Phase II	Describes clinical trials that gather preliminary data on effectiveness, continue to monitor safety
Phase I/II	Combination Phase I/Phase II clinical trial
Phase III	Pivotal experiments to gather data on safety and effectiveness
Phase II/III	Combination Phase II/Phase III clinical trial

Clinical Trials - Gene Therapy Trial Browser

Clinical Trials Daily Digest

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Accelerated Approval	These regulations allowed drugs for serious conditions that filled an unmet medical need to be approved based on a surrogate endpoint
Breakthrough Therapy	A process designed to expedite the development and review of drugs which may demonstrate substantial improvement over available therapy.
Fast Track	Fast track is a process designed to facilitate the development, and expedite the review of drugs to treat serious conditions and fill an unmet medical need
Orphan Drug	This designation is for drugs intended to treat rare diseases or conditions that affect a small number of people, offering incentives like tax credits and market exclusivity
Priority Review	A Priority Review designation means FDA’s goal is to take action on an application within 6 months.
Rare Pediatric Disease	The rare pediatric disease PRV program aims to incentivize drug development for rare pediatric diseases.
Regenerative Medicine Advanced Therapy	Facilitates the development and expedites the review of regenerative medicine therapies, including cell therapies, therapeutic tissue engineering products, and human cell and tissue products, for serious or life-threatening conditions.
Support for Clinical Trials Advancing Rare Disease Therapeutics	(START) Pilot Program is a program designed to accelerate the development of novel drug and biological products for rare diseases by providing sponsors with enhanced communication and guidance from FDA staff.

Active, Not Recruiting	Study has ongoing, but is not enrolling new participants
Completed	Study has concluded normally
Not Yet Recruiting	Study has not started enrollment
Recruiting	Study is actively looking for participants
Suspended	Study halted prematurely but has the potential to resume
Terminated	Study was halted prematurely and will not resume, participants are no longer recieving intervention
Unknown	Study has passed its completion date, but last known status was not listed as Completed, Terminated or Withdrawn. Status has not been verified within the past 2 years. Studies with an unknown status are considered closed studies

Actual Study Start Date (m/d/y)	The actual date on which the first participant was enrolled in a clinical study
Adult/Pediatric/Both	Variable on whether trial accepts patients who are adults, pediatric (<18 years of age) or both
Ages Eligible for Study	More specific age ranges eligible for the study
Clinical Centers in USA?	Binary variable on whether trial sites are located in the USA (Y) or not (N)
Clinical Publications	URL connections to clinical data on human subjects
Compound Name	The interventional compound given to the study subjects
Countries	List of countries that contain at least 1 clinical trial site
Current Stage	The stage of the clinical trial, determined based on the studies' objective
Date of Last Update	The most recent date on which changes to a study record were made available on ClinicalTrials.gov
Delivery System	Describes the nature of the drug substance or process that is used to deliver the editor or corrected gene
Development Status	Indicates whether or not the clinical development program is ongoing, or if the drug is approved
Dose levels (up to 5)	List of doses given in the indicated clinical trial, may be expressed in specific units, or a range of possible doses
Drug Product Type	Describes the nature of the drug product
Editor Type	For gene editing type therapies, the protein that will perform the gene correction
Estimated Primary Completion Date (m/d/y)	The anticipated date that the primary outcome measure data will be complete (last participant data collected)
FDA Designations	List of special FDA designations granted to the Sponsor for the development program (i.e. Orphan Drug Designation, Fast Track, Rare Pediatric Disease Designation, RMAT, etc.)
Funder Type	Describes the organization that provides support for the clinical study
Grants	URL connections to funding used to conduct preclinical or clinical studies, granted by NIH or other US institution
Has US IND?	Binary variable indicating whether the drug product is regulated by an approved Investigational New Drug application by the Food and Drug Administration of the United States
Indication	The disease, disorder, syndrome, illness, or injury that is being studied
Locations	List of countries that contain at least 1 clinical trial site
Mechanism of Action	Simplified description of how the drug product works
NCT Number	Unique identification code given to each clinical study upon registration at ClinicalTrials.gov
News and Press Releases	URL connections to press releases generated by drug product Sponsor
N trial sites	Number of clinical sites where the clinical trial is conducted
Patents	URL connections to patents, intellectual property related to the drug product
Phases	The stage of the clinical trial, determined based on the studies' objective
Preclinical Publications	URL connections to preclinical data (in vitro, animal data)
Protocols	URL connections to clinical trial protocols, study design papers
Recent Regulatory Updates	News, updates on recent or anticipated regulatory milestones
Recruitment Status	Indicates the current recruitment status or the expanded access status
Results Posted	Indicates if summary results are posted to the clinical trial record
Route of Administration	How the drug product is introduced to the body
Sexes Eligible for Study	A type of eligibility criteria that indicates the sex of people who may participate in a clinical study (all, female, male)
Sponsor	The organization or person who initiates the study and who has authority and control over the study
Sponsor Class	Describes the organization that provides support for the clinical study
Standard Ages	Variable on whether trial accepts patients who are adults, pediatric (<18 years of age) or both
Target Gene or Variant	The symbol of the gene that is corrected or replaced by the drug compound
Target Tissue or Cell	Lists target cells if the therapy is directed at a particular cell type (either by ex-vivo enrichment, or tissue-specific regulatory elements)
Therapy Route	Describes whether the gene therapy is introduced to cells in-vivo or ex-vivo
Therapy Type	Describes the nature of the gene therapy
Trial Enrollment	Number of study subjects planned or actually enrolled in the study
Vector Type	Gives additional specifics (if known) about delivery system (e.g. AAV serotype)

Ex-vivo	When the cells are modified outside the body
In-vivo	When the cells are modified inside the body

Gene editing	A gene therapy where disease-causing variant is corrected via a gene editor
Recent Regulatory Updates	A gene therapy where a corrected gene is administered to relevant cells/tissues via a delivery system

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Clinical Trials - Gene Therapy Trial Browser

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