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, meeting abstracts, and more to give a comprehensive overview of the gene therapy clinical trial landscape.
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SCGE Platform Gene Therapy Clinical Trials downloaded on: 2026/08/15 07:41:25; Please cite the Somatic Cell Genome Editing Consortium Platform when using publicly accessible data in formal presentation or publication.
Definitions
Abbreviation
| 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 |
Delivery Type
| 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. |
FDA Designation
| 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. |
Funder Type
| Industry | All for-profit entities |
| NIH | U.S. National Institutes of Health |
| Other Non-Profit | Includes individuals, universities, community-based organizations |
Route Of Administration
| 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 |
Stages
| 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 |
Study Status
| 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 |
Table Column Header
| 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) |
Therapy Route
| Ex-vivo | When the cells are modified outside the body |
| In-vivo | When the cells are modified inside the body |
Therapy Type
| 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 |
References
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Fabry Disease
Sponsor
Sangamo Therapeutics (Industry)
Compound
ST-920
Therapeutic Modality
Gene transfer
Target Gene
GLA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2019-08-01
Last Modified Date: 1786424400000
Enrollment: 36
Australia, Canada, Germany + 4 more
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor
Janssen Research & Development, LLC (Industry)
Compound
Botaretigene sparoparvovec
Therapeutic Modality
Gene transfer
Target Gene
RPGR
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation
First Submitted: 2020-11-05
Last Modified Date: 1786424400000
Enrollment: 105
Belgium, Canada, Denmark + 8 more
Alzheimer's Disease, Early Onset Alzheimer's Disease
Sponsor
Lexeo Therapeutics (Industry)
Compound
LX1001
Therapeutic Modality
Gene transfer
Target Gene
APOE2
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track
First Submitted: 2018-08-03
Last Modified Date: 1786424400000
Enrollment: 15
United States
Glycogen Storage Disease Type Ia
Sponsor
Ultragenyx Pharmaceutical Inc (Industry)
Compound
DTX401
Therapeutic Modality
Gene transfer
Target Gene
G6PC
Vector Type
Adeno-associated virus
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
First Submitted: 2021-07-14
Last Modified Date: 1786424400000
Enrollment: 49
Brazil, Canada, Denmark + 6 more
Parkinson's Disease
Sponsor
MeiraGTx, LLC (Industry)
Compound
AAV-GAD
Therapeutic Modality
Gene transfer
Target Gene
GAD1/2
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2022-10-12
Last Modified Date: 1786424400000
Enrollment: 14
United States
Diabetic Neuropathy
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2020-06-25
Last Modified Date: 1784869200000
Enrollment: 162
United States
Peripheral Artery Disease
Sponsor
Northwestern University (Other)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2017-11-30
Last Modified Date: 1784869200000
Enrollment: 39
United States
Critical Limb Ischemia
Sponsor
Beijing Northland Biotech. Co., Ltd. (Industry)
Compound
NL003
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2020-01-18
Last Modified Date: 1784869200000
Enrollment: 302
China
Osteoarthritis, Knee
Sponsor
Xalud Therapeutics, Inc. (Industry)
Compound
XT-150
Therapeutic Modality
Gene transfer
Target Gene
Recombinant IL10
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track
First Submitted: 2019-10-09
Last Modified Date: 1784869200000
Enrollment: 289
Australia, United States
Critical Limb Ischemia
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2010-02-04
Last Modified Date: 1784869200000
Enrollment: 52
South Korea, United States
Amyotrophic Lateral Sclerosis (ALS)
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track, Orphan Drug Designation
First Submitted: 2021-11-05
Last Modified Date: 1784869200000
Enrollment: 8
South Korea, United States
Ischemic Heart Disease
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2011-08-21
Last Modified Date: 1784869200000
Enrollment: 9
South Korea
Charcot-Marie-Tooth Disease 1A
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapeutic Modality
Gene transfer
Target Gene
HGF
Delivery System
Not Applicable
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2022-03-31
Last Modified Date: 1784869200000
Enrollment: 12
South Korea
Neovascular (Wet) Age-Related Macular Degeneration
Sponsor
Oxford BioMedica (Industry)
Compound
RetinoStat
Therapeutic Modality
Gene transfer
Target Gene
COL18A1+PLG
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
First Submitted: 2011-02-21
Last Modified Date: 1784782800000
Enrollment: 21
United States
Choroideremia
Sponsor
Spark Therapeutics, Inc. (Industry)
Compound
SPK-7001
Therapeutic Modality
Gene transfer
Target Gene
CHM
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation
First Submitted: 2015-01-12
Last Modified Date: 1784782800000
Enrollment: 15
United States
HIV-1-infection
Sponsor
Excision BioTherapeutics (Industry)
Compound
EBT-101
Therapeutic Modality
Gene editing
Target Gene
HIV genes
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Gene excision
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT), Fast Track
First Submitted: 2021-11-22
Last Modified Date: 1784782800000
Enrollment: 6
United States
Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor
Adverum Biotechnologies, Inc. (Industry)
Compound
ADVM-043
Therapeutic Modality
Gene transfer
Target Gene
SERPINA1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2014-06-10
Last Modified Date: 1784782800000
Enrollment: 6
United States
Advanced Retinitis Pigmentosa
Sponsor
AbbVie (Industry)
Compound
RST-001
Therapeutic Modality
Gene transfer
Target Gene
ChR2
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Orphan Drug Designation
First Submitted: 2015-09-21
Last Modified Date: 1784696400000
Enrollment: 14
United States
Leber Congenital Amaurosis
Sponsor
Nantes University Hospital (Other)
Compound
RAAV2/4.hRPE65
Therapeutic Modality
Gene transfer
Target Gene
RPE65
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2011-11-24
Last Modified Date: 1784696400000
Enrollment: 9
France
Mucopolysaccharidosis Type VI
Sponsor
Fondazione Telethon (Other)
Compound
AAV2/8.TBG.hARSB
Therapeutic Modality
Gene transfer
Target Gene
ARSB
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2017-04-12
Last Modified Date: 1784696400000
Enrollment: 9
Italy, Turkey (Türkiye)
Leber Congenital Amaurosis-Type 2
Sponsor
Hadassah Medical Organization (Other)
Compound
RAAV2-hRPE65
Therapeutic Modality
Gene transfer
Target Gene
RPE65
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2009-01-12
Last Modified Date: 1784696400000
Enrollment: 3
Israel
Retinitis Pigmentosa
Sponsor
Novartis Pharmaceuticals (Industry)
Compound
CPK850
Therapeutic Modality
Gene transfer
Target Gene
RLBP1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2017-12-11
Last Modified Date: 1784696400000
Enrollment: 12
Sweden
Idiopathic Parkinson's Disease
Sponsor
Sangamo Therapeutics (Industry)
Compound
CERE-120
Therapeutic Modality
Gene transfer
Target Gene
NRTN
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2009-09-25
Last Modified Date: 1784696400000
Enrollment: 57
United States
CLN2 Batten Disease, Late-Infantile Neuronal Ceroid Lipofuscinosis
Sponsor
Weill Medical College of Cornell University (Other)
Compound
LX1004
Therapeutic Modality
Gene transfer
Target Gene
TPP1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2010-03-22
Last Modified Date: 1784696400000
Enrollment: 12
United States
Glycogen Storage Disease Type 2 (Pompe Disease)
Sponsor
University of Florida (Other)
Compound
AAV1-CMV-hGAA
Therapeutic Modality
Gene transfer
Target Gene
GAA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2009-07-13
Last Modified Date: 1784696400000
Enrollment: 9
United States
Acute Intermittent Porphyria
Sponsor
Digna Biotech S.L. (Industry)
Compound
RAAV2/5-PBGD
Therapeutic Modality
Gene transfer
Target Gene
PBGD
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2014-02-28
Last Modified Date: 1784696400000
Enrollment: 8
Spain
Pompe Disease
Sponsor
AskBio Inc (Industry)
Compound
ACTUS-101
Therapeutic Modality
Gene transfer
Target Gene
GAA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track
First Submitted: 2018-04-13
Last Modified Date: 1784696400000
Enrollment: 7
United States
Retinitis Pigmentosa, Retinal Dystrophies, Retinal Degeneration
Sponsor
Nanoscope Therapeutics Inc. (Industry)
Compound
MCO-010
Therapeutic Modality
Gene transfer
Target Gene
MCO
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track, Orphan Drug Designation
First Submitted: 2021-06-15
Last Modified Date: 1784696400000
Enrollment: 27
Puerto Rico, United States
Stargardt Disease
Sponsor
Nanoscope Therapeutics Inc. (Industry)
Compound
MCO-010
Therapeutic Modality
Gene transfer
Target Gene
MCO
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track, Orphan Drug Designation
First Submitted: 2022-06-09
Last Modified Date: 1784696400000
Enrollment: 6
United States
Sickle Cell Disease
Sponsor
Assistance Publique - Hôpitaux de Paris (Other)
Compound
DREPAGLOBE
Therapeutic Modality
Gene transfer
Target Gene
HBB
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2019-05-14
Last Modified Date: 1784696400000
Enrollment: 6
France
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor
Exegenesis Bio (Industry)
Compound
EXG102-031
Therapeutic Modality
Gene transfer
Target Gene
ABD-VEGFR
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
First Submitted: 2023-06-06
Last Modified Date: 1784696400000
Enrollment: 12
United States
X-Linked Retinoschisis
Sponsor
VegaVect, Inc. (Industry)
Compound
RS1 AAV Vector
Therapeutic Modality
Gene transfer
Target Gene
RS1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2014-12-16
Last Modified Date: 1784696400000
Enrollment: 12
United States
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor
Biogen (Industry)
Compound
BIIB112
Therapeutic Modality
Gene transfer
Target Gene
RPGR
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation
First Submitted: 2017-03-29
Last Modified Date: 1784696400000
Enrollment: 50
United Kingdom, United States
Variant Late-Infantile Neuronal Ceroid Lipofuscinosis (CLN6 Batten Disease)
Sponsor
Emily de los Reyes (Other)
Compound
AT-GTX-501
Therapeutic Modality
Gene transfer
Target Gene
CLN6
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-03-16
Last Modified Date: 1784696400000
Enrollment: 13
United States
Leukocyte Adhesion Defect - Type I
Sponsor
Rocket Pharmaceuticals Inc. (Industry)
Compound
KRESLADI
Therapeutic Modality
Gene transfer
Target Gene
ITGB2
Vector Type
Lentivirus
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
First Submitted: 2019-01-18
Last Modified Date: 1784696400000
Enrollment: 9
Spain, United Kingdom, United States
Achromatopsia
Sponsor
MeiraGTx UK II Ltd (Industry)
Compound
AAV-CNGA3
Therapeutic Modality
Gene transfer
Target Gene
CNGA3
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-11-27
Last Modified Date: 1784696400000
Enrollment: 11
United Kingdom, United States
Alzheimer's Disease
Sponsor
Sangamo Therapeutics (Industry)
Compound
CERE-110
Therapeutic Modality
Gene transfer
Target Gene
NGF
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2009-04-03
Last Modified Date: 1784696400000
Enrollment: 49
United States
Choroideremia
Sponsor
Biogen (Industry)
Compound
BIIB111
Therapeutic Modality
Gene transfer
Target Gene
CHM
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2018-03-07
Last Modified Date: 1784696400000
Enrollment: 169
Canada, Denmark, Finland + 5 more
Achromatopsia
Sponsor
MeiraGTx UK II Ltd (Industry)
Compound
AAV-CNGB3
Therapeutic Modality
Gene transfer
Target Gene
CNGB3
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-11-22
Last Modified Date: 1784696400000
Enrollment: 23
United Kingdom, United States
Hemophilia A
Sponsor
Christian Medical College, Vellore, India (Other)
Compound
CD68-ET3-LV
Therapeutic Modality
Gene transfer
Target Gene
F8
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2022-02-16
Last Modified Date: 1784696400000
Enrollment: 6
India
Hemophilia B
Sponsor
St. Jude Children's Research Hospital (Other)
Compound
ScAAV2/8-LP1-hFIXco
Therapeutic Modality
Gene transfer
Target Gene
F9
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2009-09-16
Last Modified Date: 1784696400000
Enrollment: 14
United Kingdom, United States
X-Linked Retinoschisis
Sponsor
Zhongmou Therapeutics (Industry)
Compound
ZM-01
Therapeutic Modality
Gene transfer
Target Gene
RS1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2023-09-25
Last Modified Date: 1784696400000
Enrollment: 2
China
Geographic Atrophy
Sponsor
Janssen Research & Development, LLC (Industry)
Compound
JNJ-1887
Therapeutic Modality
Gene transfer
Target Gene
CD59
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2023-03-07
Last Modified Date: 1784696400000
Enrollment: 305
Australia, Belgium, Canada + 14 more
Leber Hereditary Optic Neuropathy (LHON)
Sponsor
GenSight Biologics (Industry)
Compound
LUMEVOQ
Therapeutic Modality
Gene transfer
Target Gene
ND4 m.11778G>A
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2016-01-07
Last Modified Date: 1784696400000
Enrollment: 39
France, Germany, Italy + 2 more
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor
Genzyme, a Sanofi Company (Industry)
Compound
RAAV2-sFLT01
Therapeutic Modality
Gene transfer
Target Gene
SFLT01
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2009-12-02
Last Modified Date: 1784696400000
Enrollment: 19
United States
Parkinson's Disease
Sponsor
Neurocrine Biosciences (Industry)
Compound
VY-AADC01
Therapeutic Modality
Gene transfer
Target Gene
DDC
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Regenerative Medicine Advanced Therapy (RMAT)
First Submitted: 2018-05-16
Last Modified Date: 1784696400000
Enrollment: 14
United States
Duchenne Muscular Dystrophy (DMD)
Sponsor
Kevin Flanigan (Other)
Compound
RAAVrh74.MCK.GALGT2
Therapeutic Modality
Gene transfer
Target Gene
GALGT2
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2017-11-01
Last Modified Date: 1784696400000
Enrollment: 2
United States
Spinal Muscular Atrophy
Sponsor
Novartis Pharmaceuticals (Industry)
Compound
ITVISMA
Therapeutic Modality
Gene transfer
Target Gene
SMN1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Orphan Drug Designation
First Submitted: 2022-05-18
Last Modified Date: 1784696400000
Enrollment: 27
Australia, Belgium, Canada + 6 more
Retinal Disease, Retinitis Pigmentosa
Sponsor
King Khaled Eye Specialist Hospital (Other gov)
Compound
AAV2-VMD2-hMERTK
Therapeutic Modality
Gene transfer
Target Gene
MERTK
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2011-09-28
Last Modified Date: 1784696400000
Enrollment: 6
Saudi Arabia
Leber Congenital Amaurosis-Type 2
Sponsor
Beacon Therapeutics (Industry)
Compound
RAAV2-CB-hRPE65
Therapeutic Modality
Gene transfer
Target Gene
RPE65
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2008-09-08
Last Modified Date: 1784696400000
Enrollment: 12
United States
Hemophilia A
Sponsor
BioMarin Pharmaceutical (Industry)
Compound
ROCTAVIAN
Therapeutic Modality
Gene transfer
Target Gene
F8
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, Orphan Drug Designation
First Submitted: 2020-03-24
Last Modified Date: 1784696400000
Enrollment: 22
Australia, Brazil, Taiwan + 1 more
Duchenne Muscular Dystrophy (DMD)
Sponsor
Nationwide Children's Hospital (Other)
Compound
RAAV2.5-CMV-mini-Dystrophin
Therapeutic Modality
Gene transfer
Target Gene
Micro-dystrophin
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2007-01-26
Last Modified Date: 1784696400000
Enrollment: 6
United States
Metachromatic Leukodystrophy
Sponsor
Institut National de la Santé Et de la Recherche Médicale, France (Other gov)
Compound
AAVrh.10-CAG/cuARSA
Therapeutic Modality
Gene transfer
Target Gene
ARSA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2013-01-28
Last Modified Date: 1784696400000
Enrollment: 5
France
Leber Congenital Amaurosis
Sponsor
MeiraGTx UK II Ltd (Industry)
Compound
Cevaretigene ritoparvovec
Therapeutic Modality
Gene transfer
Target Gene
RPE65
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2016-04-28
Last Modified Date: 1784696400000
Enrollment: 15
United Kingdom, United States
Duchenne Muscular Dystrophy (DMD)
Sponsor
Megan Waldrop (Other)
Compound
AT702
Therapeutic Modality
Gene transfer
Target Gene
DMD (exon 2)
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Exon skipping/splice editor
First Submitted: 2020-01-22
Last Modified Date: 1784696400000
Enrollment: 3
United States
Spinal Muscular Atrophy
Sponsor
Novartis Pharmaceuticals (Industry)
Compound
ZOLGENSMA
Therapeutic Modality
Gene transfer
Target Gene
SMN1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-09-20
Last Modified Date: 1784696400000
Enrollment: 16
Argentina, Brazil
Beta-Thalassemia Major
Sponsor
Genetix Biotherapeutics Inc. (Industry)
Compound
ZYNTEGLO
Therapeutic Modality
Gene transfer
Target Gene
HBB
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2017-06-29
Last Modified Date: 1784696400000
Enrollment: 19
France, Germany, Greece + 3 more
Hemophilia B
Sponsor
CSL Behring (Industry)
Compound
HEMGENIX
Therapeutic Modality
Gene transfer
Target Gene
F9-R338L
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Priority Review, Orphan Drug Designation
First Submitted: 2018-06-14
Last Modified Date: 1784696400000
Enrollment: 67
Belgium, Denmark, Germany + 5 more
Diabetic Macular Edema, Diabetic Retinopathy
Sponsor
Adverum Biotechnologies, Inc. (Industry)
Compound
Ixoberogene soroparvovec
Therapeutic Modality
Gene transfer
Target Gene
Codon optimized aflibercept
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
First Submitted: 2020-05-28
Last Modified Date: 1784696400000
Enrollment: 36
Puerto Rico, United States
Sanfilippo Syndrome B
Sponsor
UniQure Biopharma B.V. (Industry)
Compound
AMT-110
Therapeutic Modality
Gene transfer
Target Gene
NAGLU
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2016-06-15
Last Modified Date: 1784696400000
Enrollment: 4
France
Leber Congenital Amaurosis-Type 2
Sponsor
University College, London (Other)
Compound
TgAAG76
Therapeutic Modality
Gene transfer
Target Gene
RPE65
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2008-03-20
Last Modified Date: 1784696400000
Enrollment: 12
United Kingdom
Leber Hereditary Optic Neuropathy (LHON)
Sponsor
Byron Lam (Other)
Compound
ScAAV2-P1ND4v2
Therapeutic Modality
Gene transfer
Target Gene
ND4
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2014-06-06
Last Modified Date: 1784696400000
Enrollment: 28
United States
Glycogen Storage Disease Type 2 (Pompe Disease)
Sponsor
University of Florida (Other)
Compound
RAAV9-DES-hGAA
Therapeutic Modality
Gene transfer
Target Gene
GAA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2014-09-11
Last Modified Date: 1784696400000
Enrollment: 2
United States
Cerebral Adrenoleukodystrophy (CALD)
Sponsor
Genetix Biotherapeutics Inc. (Industry)
Compound
SKYSONA
Therapeutic Modality
Gene transfer
Target Gene
ABCD1
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2019-02-13
Last Modified Date: 1784696400000
Enrollment: 35
France, Germany, Italy + 3 more
X-Linked Retinoschisis
Sponsor
Beacon Therapeutics (Industry)
Compound
RAAV2tYF-CB-hRS1
Therapeutic Modality
Gene transfer
Target Gene
RS1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation
First Submitted: 2015-04-02
Last Modified Date: 1784696400000
Enrollment: 27
United States
Unilateral OR Bilateral Severe to Profound Hearing Loss
Sponsor
Novartis Pharmaceuticals (Industry)
Compound
CGF166
Therapeutic Modality
Gene transfer
Target Gene
ATOH1
Vector Type
Adenovirus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2014-05-05
Last Modified Date: 1784696400000
Enrollment: 22
United States
Sickle Cell Disease
Sponsor
Donald B. Kohn, M.D. (Other)
Compound
LentiG-βAS3-FB (contains anti-sickling mutations: T87Q, G16D, E22A)
Therapeutic Modality
Gene transfer
Target Gene
HBB
Vector Type
Lentivirus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2014-09-20
Last Modified Date: 1784696400000
Enrollment: 4
United States
Congestive Heart Failure
Sponsor
Hammond, H. Kirk, M.D. (Indiv)
Compound
RT-100
Therapeutic Modality
Gene transfer
Target Gene
ADCY6
Vector Type
Adenovirus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track
First Submitted: 2008-11-06
Last Modified Date: 1784610000000
Enrollment: 56
United States
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor
Lions Eye Institute, Perth, Western Australia (Other)
Compound
RAAV.sFlt-1
Therapeutic Modality
Gene transfer
Target Gene
Soluble VEGFR1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
First Submitted: 2011-12-14
Last Modified Date: 1784610000000
Enrollment: 40
Australia
Limb-Girdle Muscular Dystrophy, Type 2C/R5
Sponsor
Genethon (Other)
Compound
AAV1.DES.hSGCG
Therapeutic Modality
Gene transfer
Target Gene
SGCG
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2011-03-17
Last Modified Date: 1784610000000
Enrollment: 9
France
Ischemic Cardiomyopathy, Non-ischemic Cardiomyopathy, Heart Failure, Cardiomyopathies
Sponsor
Celladon Corporation (Industry)
Compound
AAV1-CMV-Serca2a
Therapeutic Modality
Gene transfer
Target Gene
SERCA2A
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2012-07-16
Last Modified Date: 1784610000000
Enrollment: 250
Belgium, Denmark, Germany + 7 more
Familial Lipoprotein Lipase Deficiency
Sponsor
Amsterdam Molecular Therapeutics (Industry)
Compound
GLYBERA
Therapeutic Modality
Gene transfer
Target Gene
LPL
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation
First Submitted: 2009-04-30
Last Modified Date: 1784610000000
Enrollment: 5
Canada
Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease
Sponsor
Verve Therapeutics, Inc. (Industry)
Compound
VERVE-101
Therapeutic Modality
Gene editing
Target Gene
PCSK9
Delivery System
Endocytosis
Mechanism of Action
Gene inactivation
First Submitted: 2022-05-19
Last Modified Date: 1784610000000
Enrollment: 13
New Zealand, United Kingdom
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor
Krystal Biotech, Inc. (Industry)
Compound
VYJUVEK
Therapeutic Modality
Gene transfer
Target Gene
COL7A1
Vector Type
Herpesvirus
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
First Submitted: 2021-06-02
Last Modified Date: 1784610000000
Enrollment: 47
United States
X-Linked Chronic Granulomatous Disease
Sponsor
University of Zurich (Other)
Compound
Retroviral SF71-gp91phox transduced CD34+ cells
Therapeutic Modality
Gene transfer
Target Gene
CYBB
Vector Type
Retrovirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2009-06-22
Last Modified Date: 1784610000000
Enrollment: 2
Switzerland
Becker Muscular Dystrophy, Sporadic Inclusion Body Myositis
Sponsor
Nationwide Children's Hospital (Other)
Compound
RAAV1.CMV.huFollistatin344
Therapeutic Modality
Gene transfer
Target Gene
FST (FS344)
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2012-01-23
Last Modified Date: 1784610000000
Enrollment: 15
United States
Hemophilia A
Sponsor
Spark Therapeutics, Inc. (Industry)
Compound
SPK-8016
Therapeutic Modality
Gene transfer
Target Gene
F8
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2018-11-06
Last Modified Date: 1784610000000
Enrollment: 4
United States
Cystic Fibrosis
Sponsor
Imperial College London (Other)
Compound
PGM169/GL67A
Therapeutic Modality
Gene transfer
Target Gene
CFTR
Delivery System
Endocytosis
Mechanism of Action
Functional gene replacement
First Submitted: 2012-06-14
Last Modified Date: 1784610000000
Enrollment: 130
United Kingdom
Fanconi Anemia Complementation Group A
Sponsor
Boston Children's Hospital (Other)
Compound
MSCV-FANCA
Therapeutic Modality
Gene transfer
Target Gene
FANCA
Vector Type
Retrovirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2006-01-04
Last Modified Date: 1784610000000
Enrollment: 3
United States
Alpha-1 Antitrypsin Deficiency (AATD)
Sponsor
Beacon Therapeutics (Industry)
Compound
AGTC-0106
Therapeutic Modality
Gene transfer
Target Gene
SERPINA1
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2010-01-21
Last Modified Date: 1784610000000
Enrollment: 9
Ireland, United States
Limb-Girdle Muscular Dystrophy, Type 2D/R3
Sponsor
Nationwide Children's Hospital (Other)
Compound
RAAV1.tMCK.human-alpha-sarcoglycan
Therapeutic Modality
Gene transfer
Target Gene
SGCA
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2007-06-27
Last Modified Date: 1784610000000
Enrollment: 6
United States
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor
National Institute of Allergy and Infectious Diseases (NIAID) (NIH)
Compound
GALV MFGS-gc retrovirus
Therapeutic Modality
Gene transfer
Target Gene
IL2RG
Vector Type
Retrovirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2001-12-17
Last Modified Date: 1784610000000
Enrollment: 3
United States
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
Sponsor
Fondazione Telethon (Other)
Compound
STRIMVELIS
Therapeutic Modality
Gene transfer
Target Gene
ADA
Vector Type
Retrovirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2008-01-10
Last Modified Date: 1784610000000
Enrollment: 12
Israel, Italy
Parotid Salivary Dysfunction
Sponsor
National Institute of Dental and Craniofacial Research (NIDCR) (NIH)
Compound
AdhAQP1
Therapeutic Modality
Gene transfer
Target Gene
AQP1
Vector Type
Adenovirus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2006-09-02
Last Modified Date: 1784610000000
Enrollment: 17
United States
Duchenne Muscular Dystrophy (DMD)
Sponsor
Jerry R. Mendell (Other)
Compound
RAAV1.CMV.huFollistatin344
Therapeutic Modality
Gene transfer
Target Gene
FST (FS344)
Vector Type
Adeno-associated virus
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2015-01-26
Last Modified Date: 1784610000000
Enrollment: 3
United States
X-linked Severe Combined Immunodeficiency (XSCID)
Sponsor
Assistance Publique - Hôpitaux de Paris (Other)
Compound
Autologous CD34+ cells transduced with the self-inactivating (SIN) GAMMARETROVIRAL vector pSRS11.EFS.IL2RG.pre
Therapeutic Modality
Gene transfer
Target Gene
IL2RG
Vector Type
Retrovirus
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2011-06-21
Last Modified Date: 1784610000000
Enrollment: 5
France
Beta-Thalassemia Major
Sponsor
CorrectSequence Therapeutics Co., Ltd (Industry)
Compound
CS-101
Therapeutic Modality
Gene editing
Target Gene
BCL11A
Delivery System
Electroporation
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2024-02-23
Last Modified Date: 1781240400000
Enrollment: 9
China
Beta-Thalassemia Major
Sponsor
Sangamo Therapeutics (Industry)
Compound
ST-400
Therapeutic Modality
Gene editing
Target Gene
BCL11A
Delivery System
Electroporation
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Orphan Drug Designation
First Submitted: 2018-02-01
Last Modified Date: 1773723600000
Enrollment: 5
United States
Clinical Trials Daily Digest
Updates in the last 14 days
NCT06860672
1786510800000
Development status
Previous:
Active
Current:
Inactive
curator
Recent updates
Previous:
Current:
this clinical trial was designed for and conducted...
curator
News and press releases
Previous:
null
Current:
LinkName:A fatal reaction: A cutting-edge gene-edi...
curator
Last update post date
Previous:
2025-03-06
Current:
2026-07-30
curator
Estimated completion date
Previous:
2025-09
Current:
2025-03-31
curator
Overall status
Previous:
Recruiting
Current:
Terminated
curator
Enrollment type
Previous:
Estimated
Current:
Actual
curator
Vector type
Previous:
dual AAV
Current:
Adeno-associated virus
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* clinical diagnosis of snijd...
api
Record status
Previous:
Draft
Current:
Active
curator
Indication doid
Previous:
null
Current:
DOID:0070668
curator
Indication
Previous:
Developmental Delay Disorder, Intellectual Disabil...
Current:
Snijders Blok-Campeau syndrome
curator
Compound name
Previous:
null
Current:
Dual Vector AAV-CHD3-R1025W Base Editor
curator
Dose 1
Previous:
null
Current:
undisclosed
curator
Editor type
Previous:
null
Current:
TeABE
curator
Delivery system
Previous:
null
Current:
viral transduction
curator
Drug product type
Previous:
null
Current:
viral vector
curator
Route of administration
Previous:
null
Current:
intrathecal
curator
Mechanism of action
Previous:
null
Current:
mutation correction
curator
Therapy route
Previous:
null
Current:
in-vivo
curator
Therapy type
Previous:
null
Current:
gene editing
curator
Target gene
Previous:
null
Current:
CDH3/R1025W
curator
Preclinical publications
Previous:
null
Current:
LinkName:In vivo base editing of Chd3 rescues beha...
curator
Snijders Blok-Campeau syndrome
Yongguo Yu
Terminated
This clinical trial was designed for and conducted in an individual patient who died as a a result of the therapy
NCT06706427
1786510800000
Last update post date
Previous:
2025-05-25
Current:
2026-08-12
api
Vector type
Previous:
AAV2
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
1. age ≥ 18 years old.
2. male or female.
3. confi...
api
Bietti Crystalline Dystrophy
NGGT (Suzhou) Biotechnology Co., Ltd.
Active not recruiting
NCT07587021
1786510800000
Last update post date
Previous:
2026-05-14
Current:
2026-08-12
api
Overall status
Previous:
Not yet recruiting
Current:
Recruiting
api
Number of locations
Previous:
0
Current:
1
api
Locations
Previous:
Current:
United states
api
Description
Previous:
This study will be conducted to evaluate the effic...
Current:
This study will be conducted to evaluate the effic...
api
Vector type
Previous:
LNP
Current:
curator
Indication doid
Previous:
Current:
DOID:0111670
curator
Development status
Previous:
Current:
Active
curator
Record status
Previous:
Draft
Current:
Active
curator
Compound name
Previous:
Current:
YOLT-203
curator
Recent updates
Previous:
Current:
US IND application cleared November 2025
curator
Dose 1
Previous:
Current:
Early Phase 1 doses: 0.3 mg/kg, 0.6 mg/kg, 1.0 mg/...
curator
Editor type
Previous:
Current:
YolCas12
curator
Delivery system
Previous:
Current:
Endocytosis
curator
Drug product type
Previous:
Current:
Encapsulated RNP
curator
Route of administration
Previous:
Current:
Intravenous
curator
Mechanism of action
Previous:
Current:
Gene inactivation
curator
Therapy route
Previous:
Current:
in vivo
curator
Therapy type
Previous:
Current:
Gene editing
curator
Target gene
Previous:
Current:
HAO1
curator
Fda designation
Previous:
Current:
Orphan Drug Designation, Rare Pediatric Disease De...
curator
Related nctid
Previous:
null
Current:
LinkName:Early Phase 1: NCT06511349;LinkUrl:https:...
curator
News and press releases
Previous:
null
Current:
LinkName:YolTech Receives U.S. FDA ODD for YOLT-20...
curator
News and press releases
Previous:
null
Current:
LinkName:YolTech Therapeutics Receives FDA IND Cle...
curator
News and press releases
Previous:
null
Current:
LinkName:Success for YolTech's hyperoxaluria in vi...
curator
News and press releases
Previous:
null
Current:
LinkName:YolTech Therapeutics Administers First Pa...
curator
News and press releases
Previous:
null
Current:
LinkName:YolTech Receives U.S. FDA RPDD for YOLT-2...
curator
News and press releases
Previous:
null
Current:
LinkName:YolTech Therapeutics Receives FDA IND Cle...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Efficient and safe in vivo treatment of p...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Library-Assisted Evolution in Eukaryotic ...
curator
Primary Hyperoxaluria Type 1, PH1
YolTech Therapeutics Co., Ltd
Recruiting
US IND application cleared November 2025
NCT06255782
1786510800000
Last update post date
Previous:
2026-04-15
Current:
2026-08-12
api
Phases
Previous:
Phase1, Phase2
Current:
Phase3
api
Compound description
Previous:
Current:
non-replicating recombinant adeno-associated virus...
curator
Fda designation
Previous:
Chemistry, Manufacturing, and Controls Development...
Current:
Chemistry, Manufacturing, and Controls Development...
curator
Vector type
Previous:
dual AAV8
Current:
Adeno-associated virus
curator
Editor type
Previous:
ARCUS
Current:
ARCUS endonuclease
curator
Browse condition terms
Previous:
Amino acid metabolism, Inborn errors, Ammonia, Bra...
Current:
Amino acid metabolism, Inborn errors, Ammonia, Bra...
api
Estimated completion date
Previous:
2026-09
Current:
2027-12
api
Eligibility criteria
Previous:
Key inclusion criteria:
1. male sex
2. gestationa...
Current:
Key inclusion criteria:
1. male sex
2. gestationa...
api
Number of locations
Previous:
10
Current:
12
api
Enrollment count
Previous:
8
Current:
20
api
Description
Previous:
Ornithine transcarbamylase (otc) deficiency, The m...
Current:
Ornithine transcarbamylase (otc) deficiency, The m...
api
Recent updates
Previous:
Sponsor reports complete clinical response in firs...
Current:
Sponsor reports complete clinical response in firs...
curator
News and press releases
Previous:
null
Current:
LinkName:iECURE Selected for U.S. FDA Chemistry, M...
curator
News and press releases
Previous:
null
Current:
LinkName:iECURE Receives FDA Regenerative Medicine...
curator
Ornithine Transcarbamylase (OTC) Deficiency
iECURE, Inc.
Recruiting
Sponsor reports complete clinical response in first infant dosed, more data expected in the first half of 2026; ECUR-506 was selected to participate i...
NCT07429240
1786510800000
Record status
Previous:
Draft
Current:
Active
curator
Vector type
Previous:
AAV
Current:
Adeno-associated virus
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) ARCUS-Mediated Excision of Exons...
curator
Preclinical publications
Previous:
null
Current:
LinkName:High-efficiency homology-directed inserti...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) Treatment with PBGENE-DMD result...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) PBGENE-DMD gene editing treatmen...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Presentation) PBGENE-DMD gene editing dr...
curator
Indication doid
Previous:
Current:
DOID:11723
curator
Development status
Previous:
Current:
Active
curator
Compound name
Previous:
Current:
PBGENE-DMD
curator
Recent updates
Previous:
Current:
initial data from multiple patients (target enroll...
curator
Dose 1
Previous:
Current:
1E14 vg/kg
curator
Editor type
Previous:
Current:
ARCUS endonuclease
curator
Delivery system
Previous:
Current:
Viral transduction
curator
Drug product type
Previous:
Current:
Viral vector
curator
Route of administration
Previous:
Current:
Intravenous
curator
Mechanism of action
Previous:
Current:
Exon skipping/splice editor
curator
Therapy route
Previous:
Current:
in vivo
curator
Therapy type
Previous:
Current:
Gene editing
curator
Target gene
Previous:
Current:
DMD del ex45-55
curator
Fda designation
Previous:
Current:
Fast Track
curator
News and press releases
Previous:
null
Current:
LinkName:Precision BioSciences Receives FDA Fast T...
curator
News and press releases
Previous:
null
Current:
LinkName:(Corporate Presentation) Precision BioSci...
curator
Last update post date
Previous:
2026-04-30
Current:
2026-06-23
api
Number of locations
Previous:
1
Current:
2
api
Overall status
Previous:
Not yet recruiting
Current:
Recruiting
api
Locations
Previous:
Current:
United states
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. males, 2 to 7 years of age...
api
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
Precision BioSciences, Inc.
Recruiting
Initial data from multiple patients (target enrollment 3-5) anticipated by YE2026
All Recent Updates (52)
NCT06860672
Show Changes
1786510800000
Development status
Previous:
Active
Current:
Inactive
curator
Recent updates
Previous:
Current:
this clinical trial was designed for and conducted...
curator
News and press releases
Previous:
null
Current:
LinkName:A fatal reaction: A cutting-edge gene-edi...
curator
Last update post date
Previous:
2025-03-06
Current:
2026-07-30
curator
Estimated completion date
Previous:
2025-09
Current:
2025-03-31
curator
Overall status
Previous:
Recruiting
Current:
Terminated
curator
Enrollment type
Previous:
Estimated
Current:
Actual
curator
Vector type
Previous:
dual AAV
Current:
Adeno-associated virus
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* clinical diagnosis of snijd...
api
Record status
Previous:
Draft
Current:
Active
curator
Indication doid
Previous:
null
Current:
DOID:0070668
curator
Indication
Previous:
Developmental Delay Disorder, Intellectual Disabil...
Current:
Snijders Blok-Campeau syndrome
curator
Compound name
Previous:
null
Current:
Dual Vector AAV-CHD3-R1025W Base Editor
curator
Dose 1
Previous:
null
Current:
undisclosed
curator
Editor type
Previous:
null
Current:
TeABE
curator
Delivery system
Previous:
null
Current:
viral transduction
curator
Drug product type
Previous:
null
Current:
viral vector
curator
Route of administration
Previous:
null
Current:
intrathecal
curator
Mechanism of action
Previous:
null
Current:
mutation correction
curator
Therapy route
Previous:
null
Current:
in-vivo
curator
Therapy type
Previous:
null
Current:
gene editing
curator
Target gene
Previous:
null
Current:
CDH3/R1025W
curator
Preclinical publications
Previous:
null
Current:
LinkName:In vivo base editing of Chd3 rescues beha...
curator
Snijders Blok-Campeau syndrome
Yongguo Yu
Terminated
This clinical trial was designed for and conducted in an individual patient who died as a a result of the therapy
NCT06706427
Show Changes
1786510800000
Last update post date
Previous:
2025-05-25
Current:
2026-08-12
api
Vector type
Previous:
AAV2
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
1. age ≥ 18 years old.
2. male or female.
3. confi...
api
Bietti Crystalline Dystrophy
NGGT (Suzhou) Biotechnology Co., Ltd.
Active not recruiting
NCT07587021
Show Changes
1786510800000
Last update post date
Previous:
2026-05-14
Current:
2026-08-12
api
Overall status
Previous:
Not yet recruiting
Current:
Recruiting
api
Number of locations
Previous:
0
Current:
1
api
Locations
Previous:
Current:
United states
api
Description
Previous:
This study will be conducted to evaluate the effic...
Current:
This study will be conducted to evaluate the effic...
api
Vector type
Previous:
LNP
Current:
curator
Indication doid
Previous:
Current:
DOID:0111670
curator
Development status
Previous:
Current:
Active
curator
Record status
Previous:
Draft
Current:
Active
curator
Compound name
Previous:
Current:
YOLT-203
curator
Recent updates
Previous:
Current:
US IND application cleared November 2025
curator
Dose 1
Previous:
Current:
Early Phase 1 doses: 0.3 mg/kg, 0.6 mg/kg, 1.0 mg/...
curator
Editor type
Previous:
Current:
YolCas12
curator
Delivery system
Previous:
Current:
Endocytosis
curator
Drug product type
Previous:
Current:
Encapsulated RNP
curator
Route of administration
Previous:
Current:
Intravenous
curator
Mechanism of action
Previous:
Current:
Gene inactivation
curator
Therapy route
Previous:
Current:
in vivo
curator
Therapy type
Previous:
Current:
Gene editing
curator
Target gene
Previous:
Current:
HAO1
curator
Fda designation
Previous:
Current:
Orphan Drug Designation, Rare Pediatric Disease De...
curator
Related nctid
Previous:
null
Current:
LinkName:Early Phase 1: NCT06511349;LinkUrl:https:...
curator
News and press releases
Previous:
null
Current:
LinkName:YolTech Receives U.S. FDA ODD for YOLT-20...
curator
News and press releases
Previous:
null
Current:
LinkName:YolTech Therapeutics Receives FDA IND Cle...
curator
News and press releases
Previous:
null
Current:
LinkName:Success for YolTech's hyperoxaluria in vi...
curator
News and press releases
Previous:
null
Current:
LinkName:YolTech Therapeutics Administers First Pa...
curator
News and press releases
Previous:
null
Current:
LinkName:YolTech Receives U.S. FDA RPDD for YOLT-2...
curator
News and press releases
Previous:
null
Current:
LinkName:YolTech Therapeutics Receives FDA IND Cle...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Efficient and safe in vivo treatment of p...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Library-Assisted Evolution in Eukaryotic ...
curator
Primary Hyperoxaluria Type 1, PH1
YolTech Therapeutics Co., Ltd
Recruiting
US IND application cleared November 2025
NCT06255782
Show Changes
1786510800000
Last update post date
Previous:
2026-04-15
Current:
2026-08-12
api
Phases
Previous:
Phase1, Phase2
Current:
Phase3
api
Compound description
Previous:
Current:
non-replicating recombinant adeno-associated virus...
curator
Fda designation
Previous:
Chemistry, Manufacturing, and Controls Development...
Current:
Chemistry, Manufacturing, and Controls Development...
curator
Vector type
Previous:
dual AAV8
Current:
Adeno-associated virus
curator
Editor type
Previous:
ARCUS
Current:
ARCUS endonuclease
curator
Browse condition terms
Previous:
Amino acid metabolism, Inborn errors, Ammonia, Bra...
Current:
Amino acid metabolism, Inborn errors, Ammonia, Bra...
api
Estimated completion date
Previous:
2026-09
Current:
2027-12
api
Eligibility criteria
Previous:
Key inclusion criteria:
1. male sex
2. gestationa...
Current:
Key inclusion criteria:
1. male sex
2. gestationa...
api
Number of locations
Previous:
10
Current:
12
api
Enrollment count
Previous:
8
Current:
20
api
Description
Previous:
Ornithine transcarbamylase (otc) deficiency, The m...
Current:
Ornithine transcarbamylase (otc) deficiency, The m...
api
Recent updates
Previous:
Sponsor reports complete clinical response in firs...
Current:
Sponsor reports complete clinical response in firs...
curator
News and press releases
Previous:
null
Current:
LinkName:iECURE Selected for U.S. FDA Chemistry, M...
curator
News and press releases
Previous:
null
Current:
LinkName:iECURE Receives FDA Regenerative Medicine...
curator
Ornithine Transcarbamylase (OTC) Deficiency
iECURE, Inc.
Recruiting
Sponsor reports complete clinical response in first infant dosed, more data expected in the first half of 2026; ECUR-506 was selected to participate in the FDA's Chemistry, Manufacturing, and Controls...
NCT07429240
Show Changes
1786510800000
Record status
Previous:
Draft
Current:
Active
curator
Vector type
Previous:
AAV
Current:
Adeno-associated virus
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) ARCUS-Mediated Excision of Exons...
curator
Preclinical publications
Previous:
null
Current:
LinkName:High-efficiency homology-directed inserti...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) Treatment with PBGENE-DMD result...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) PBGENE-DMD gene editing treatmen...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Presentation) PBGENE-DMD gene editing dr...
curator
Indication doid
Previous:
Current:
DOID:11723
curator
Development status
Previous:
Current:
Active
curator
Compound name
Previous:
Current:
PBGENE-DMD
curator
Recent updates
Previous:
Current:
initial data from multiple patients (target enroll...
curator
Dose 1
Previous:
Current:
1E14 vg/kg
curator
Editor type
Previous:
Current:
ARCUS endonuclease
curator
Delivery system
Previous:
Current:
Viral transduction
curator
Drug product type
Previous:
Current:
Viral vector
curator
Route of administration
Previous:
Current:
Intravenous
curator
Mechanism of action
Previous:
Current:
Exon skipping/splice editor
curator
Therapy route
Previous:
Current:
in vivo
curator
Therapy type
Previous:
Current:
Gene editing
curator
Target gene
Previous:
Current:
DMD del ex45-55
curator
Fda designation
Previous:
Current:
Fast Track
curator
News and press releases
Previous:
null
Current:
LinkName:Precision BioSciences Receives FDA Fast T...
curator
News and press releases
Previous:
null
Current:
LinkName:(Corporate Presentation) Precision BioSci...
curator
Last update post date
Previous:
2026-04-30
Current:
2026-06-23
api
Number of locations
Previous:
1
Current:
2
api
Overall status
Previous:
Not yet recruiting
Current:
Recruiting
api
Locations
Previous:
Current:
United states
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. males, 2 to 7 years of age...
api
Duchenne Muscular Dystrophy With Mutations Amenable to PBGENE-DMD
Precision BioSciences, Inc.
Recruiting
Initial data from multiple patients (target enrollment 3-5) anticipated by YE2026
NCT06663878
Show Changes
1786510800000
Last update post date
Previous:
2024-10-29
Current:
2026-08-12
api
Overall status
Previous:
Not yet recruiting
Current:
Active not recruiting
api
Therapy route
Previous:
Undisclosed
Current:
In-vivo
curator
Therapy type
Previous:
Undisclosed
Current:
Gene transfer
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* male or female aged 18 and ...
api
Wilson Disease
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Active not recruiting
NCT04046224
Show Changes
1786424400000
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) A combined fertility, embr...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Isaralgagene civaparvovec ...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Isaralgagene civaparvovec ...
curator
News and press releases
Previous:
null
Current:
LinkName:Sangamo Therapeutics Enters Into Asset Sa...
curator
News and press releases
Previous:
LinkName:Sangamo Therapeutics Reports Recent Busin...
Current:
LinkName:Sangamo Therapeutics Reports Recent Busin...
curator
Clinical publications
Previous:
LinkName:(Poster) Isaralgagene civaparvovec (ST-92...
Current:
LinkName:(Poster) Isaralgagene civaparvovec (ST-92...
curator
Clinical publications
Previous:
LinkName:(Presentation) Isaralgagene civaparvovec ...
Current:
LinkName:(Presentation) Isaralgagene civaparvovec ...
curator
Clinical publications
Previous:
LinkName:Isaralgagene Civaparvovec (ST-920) Shows ...
Current:
LinkName:(Poster) Isaralgagene Civaparvovec (ST-92...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Isaralgagene civaparvovec ...
curator
Vector type
Previous:
AAV2/6
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Fda designation
Previous:
Orphan Drug Designation
Current:
Fast Track, Orphan Drug Designation, Regenerative ...
curator
With has results
Previous:
False
Current:
True
api
Last update post date
Previous:
2025-05-15
Current:
2026-04-14
api
Eligibility criteria
Previous:
Inclusion criteria:
* ≥ 18 years of age
* documen...
Current:
Inclusion criteria:
* ≥ 18 years of age
* documen...
api
Enrollment count
Previous:
33
Current:
36
api
Description
Previous:
This is the first in human treatment with st-920, ...
Current:
This is the first in human treatment with st-920, ...
api
Related nctid
Previous:
null
Current:
LinkName:Long Term Follow-Up: NCT05039866;LinkUrl:...
curator
Recent updates
Previous:
Recent Type B meeting with FDA allowed ongoing Pha...
Current:
Rolling BLA submission initiated December 2025, th...
curator
Fabry Disease
Sangamo Therapeutics
Completed
Rolling BLA submission initiated December 2025, the company expects submission to be completed 2Q2026
NCT06292650
Show Changes
1786424400000
Recent updates
Previous:
Two patients treated early 2024, IND cleared Decem...
Current:
Two patients treated early 2024, IND cleared Decem...
curator
News and press releases
Previous:
LinkName:Zhongmou Therapeutics Receives Orphan Dru...
Current:
LinkName:Zhongmou Therapeutics Receives Orphan Dru...
curator
Clinical publications
Previous:
LinkName:(Abstract #849) Safety and Efficacy of ZM...
Current:
LinkName:(Abstract #849) Safety and Efficacy of ZM...
curator
Vector type
Previous:
AAV
Current:
Adeno-associated virus
curator
Last update post date
Previous:
2025-12-04
Current:
2026-03-31
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
patients who meet all of the ...
api
Retinitis Pigmentosa
Zhongmou Therapeutics
Recruiting
Two patients treated early 2024, IND cleared December 2025; the company updated its international brand name to Augelux Therapeutics, but the name Zhongmou Therapeutics continues to be used in Mainlan...
NCT06217861
Show Changes
1786424400000
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Intracerebroventricular
Current:
Intraventricular
curator
Last update post date
Previous:
2024-05-17
Current:
2026-05-13
curator
Estimated completion date
Previous:
2026-08
Current:
2027-08
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. subjects must be ≤ 6 years...
api
Glutaric Acidemia Type I
Shanghai Vitalgen BioPharma Co., Ltd.
Recruiting
Granted CTA approval by NMPA on 7/13/23
NCT06272149
Show Changes
1786424400000
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Recent updates
Previous:
FIH interim data presented at ASGCT 2024
Current:
FIH interim data presented at ASGCT 2024, Orphan d...
curator
Fda designation
Previous:
Rare Pediatric Disease Designation
Current:
Orphan Drug Designation, Rare Pediatric Disease De...
curator
Dose 2
Previous:
Undisclosed high dose
Current:
curator
Dose 1
Previous:
Undisclosed low dose
Current:
Undisclosed dose escalation, 2 levels
curator
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Intracerebroventricular
Current:
Intraventricular
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. infants with age of ≤24 mo...
api
Type II Gaucher Disease
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine
Recruiting
FIH interim data presented at ASGCT 2024, Orphan drug designation granted 1/13/26
NCT07064759
Show Changes
1786424400000
Clinical publications
Previous:
LinkName:(Corporate Presentation) 4D-150 Business ...
Current:
LinkName:(Corporate Presentation) 4D-150 Business ...
curator
Clinical publications
Previous:
LinkName:(Corporate Presentation) 4D-150: PRISM Ph...
Current:
LinkName:(Corporate Presentation) July 2026;LinkUr...
curator
Vector type
Previous:
AAV
Current:
Adeno-associated virus
curator
Recent updates
Previous:
4D-150 is also being evaluated for the treatment o...
Current:
Sponsor announced positive 2-year data from Phase ...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) 2-Year Follow-Up: PRISM P...
curator
News and press releases
Previous:
null
Current:
LinkName:Corporate Presentation July 2026;LinkUrl:...
curator
News and press releases
Previous:
null
Current:
LinkName:4DMT Announces Positive 2-Year Data from ...
curator
Editor type
Previous:
none
Current:
curator
Last update post date
Previous:
2026-02-12
Current:
2026-04-16
api
Number of locations
Previous:
43
Current:
94
api
Locations
Previous:
Argentina, Latvia, Singapore, Hungary, United stat...
Current:
Argentina, Singapore, Hungary, United states, Japa...
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* ≥50 years of age at time of...
api
Neovascular Age-Related Macular Degeneration (nAMD)
4D Molecular Therapeutics
Recruiting
Sponsor announced positive 2-year data from Phase 2b trial in July 2026; 4D-150 is also being evaluated for the treatment of Diabetic macular edema in a Phase 2 trial (NCT05930561)
NCT06517888
Show Changes
1786424400000
News and press releases
Previous:
LinkName:SEC Form 10-K: 2024 Annual Report;LinkUrl...
Current:
LinkName:SEC Form 10-K: 2024 Annual Report;LinkUrl...
curator
Vector type
Previous:
AAVAnc80
Current:
Adeno-associated virus
curator
Dose 3
Previous:
Undisclosed dose 3
Current:
curator
Dose 2
Previous:
Undisclosed dose 2
Current:
curator
Dose 1
Previous:
Undisclosed dose 1
Current:
Undisclosed dose escalation, 3 levels
curator
Editor type
Previous:
none
Current:
curator
Drug product type
Previous:
Autologous cells
Current:
Viral vector
curator
Last update post date
Previous:
2026-03-31
Current:
2026-04-06
api
Eligibility criteria
Previous:
null
Current:
Criteria for inclusion:
1. presence of unilateral...
api
Vestibular Schwannoma
Akouos, Inc.
Recruiting
Eli Lilly acquired this company in 2022
NCT07160634
Show Changes
1786424400000
Clinical publications
Previous:
LinkName:(Corporate Presentation) SGT-003 INSPIRE ...
Current:
LinkName:(Corporate Presentation) August 2026;Link...
curator
Vector type
Previous:
AAV-SLB101
Current:
Adeno-associated virus
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster) Positive Preliminary Safety and ...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Update on the INSPIRE DUCH...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster) Lower POLARIS-101™ (AAV-SL...
curator
Recent updates
Previous:
Confirmatory trial is now recruiting, drug continu...
Current:
Drug continues to be well tolerated in 47 particip...
curator
Fda designation
Previous:
Fast Track, Rare Pediatric Disease Designation
Current:
Fast Track, Orphan Drug Designation, Rare Pediatri...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) Systemic Administration of a Rat...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) SGT-003 Gene Therapy Stabilizes ...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) Novel Pharmacokinetic Effects of...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Update on the INSPIRE DUCH...
curator
News and press releases
Previous:
null
Current:
LinkName:Solid Biosciences Reports First Quarter 2...
curator
Last update post date
Previous:
2026-06-02
Current:
2026-07-16
api
Number of locations
Previous:
5
Current:
6
api
Editor type
Previous:
none
Current:
curator
Locations
Previous:
Canada, Australia
Current:
Canada, United states, Australia
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* participant is ambulatory.
...
api
Record status
Previous:
Draft
Current:
Active
curator
News and press releases
Previous:
null
Current:
LinkName:Solid Biosciences Announces Positive Feed...
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 1/2: NCT06138639;LinkUrl:https://cl...
curator
Compound description
Previous:
null
Current:
AAV-SLB101-CK8-microdystrophin containing R16-R17 ...
curator
Indication doid
Previous:
null
Current:
DOID:11723
curator
Development status
Previous:
null
Current:
Active
curator
Indication
Previous:
Duchenne Muscular Dystrophy
Current:
Duchenne Muscular Dystrophy (DMD)
curator
Compound name
Previous:
null
Current:
SGT-003
curator
Dose 1
Previous:
null
Current:
1E14 vg/kg
curator
Delivery system
Previous:
null
Current:
Viral transduction
curator
Target tissue
Previous:
null
Current:
Muscle cells
curator
Drug product type
Previous:
null
Current:
Viral vector
curator
Route of administration
Previous:
null
Current:
Intravenous
curator
Mechanism of action
Previous:
null
Current:
Overexpression of protective allele/gene
curator
Therapy route
Previous:
null
Current:
In-vivo
curator
Therapy type
Previous:
null
Current:
Gene transfer
curator
Target gene
Previous:
null
Current:
Micro-dystrophin
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Abstract #1389) AAV-SLB101, a Novel Musc...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) Systemic Delivery of SGT-003 Mic...
curator
News and press releases
Previous:
null
Current:
LinkName:Solid Biosciences Reports Positive Initia...
curator
News and press releases
Previous:
null
Current:
LinkName:Solid Biosciences Receives FDA Fast Track...
curator
News and press releases
Previous:
null
Current:
LinkName:Solid Biosciences Receives Rare Pediatric...
curator
News and press releases
Previous:
null
Current:
LinkName:Solid Biosciences Granted FDA Orphan Drug...
curator
News and press releases
Previous:
null
Current:
LinkName:Solid Biosciences Awarded Innovation Pass...
curator
News and press releases
Previous:
null
Current:
LinkName:Solid Biosciences Reports Third Quarter 2...
curator
Duchenne Muscular Dystrophy (DMD)
Solid Biosciences Inc.
Recruiting
Drug continues to be well tolerated in 47 participants dosed as of 5/11/26, dosing is ongoing in Phase 3 trial
NCT06539208
Show Changes
1786424400000
News and press releases
Previous:
LinkName:YolTech Completes Dose Escalation in YOLT...
Current:
LinkName:YolTech Completes Dose Escalation in YOLT...
curator
Vector type
Previous:
LNP
Current:
curator
Dose 1
Previous:
Undisclosed dose ascension
Current:
Undisclosed dose escalation
curator
Delivery system
Previous:
Lipid encapsulation
Current:
Endocytosis
curator
Drug product type
Previous:
MRNA, LNP
Current:
Encapsulated mRNA
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. age 18 - 80 years old (inc...
api
Transthyretin Amyloidosis Polyneuropathy, Transthyrexin Amyloidosis Cardiomyopathy
YolTech Therapeutics Co., Ltd
Recruiting
Dose escalation completed December 2024
NCT06765980
Show Changes
1786424400000
Last update post date
Previous:
2026-04-30
Current:
2026-08-10
api
Number of locations
Previous:
3
Current:
4
api
Locations
Previous:
New zealand, Canada, Israel
Current:
New zealand, Canada, United states, Israel
api
Vector type
Previous:
AAV2
Current:
Adeno-associated virus
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* participant must be between...
api
Record status
Previous:
Draft
Current:
Active
curator
Preclinical publications
Previous:
LinkName:(Abstract #929) - ASGCT 2025;LinkUrl:
Current:
LinkName:(Abstract #929) Periorbital Delivery of A...
curator
Preclinical publications
Previous:
LinkName:(Abstract #327) - ASGCT 2025;LinkUrl:
Current:
LinkName:(Abstract #327) Stability of AAV2 Vector ...
curator
Preclinical publications
Previous:
LinkName:(Abstract #575) -ASGCT 2025;LinkUrl:
Current:
LinkName:(Abstract #575) KRIYA-825 (AAV2.CR2-CR1) ...
curator
Indication doid
Previous:
null
Current:
DOID:10871
curator
Development status
Previous:
null
Current:
Active
curator
Compound name
Previous:
null
Current:
KRIYA-825
curator
Dose 1
Previous:
null
Current:
undisclosed
curator
Delivery system
Previous:
null
Current:
viral transduction
curator
Drug product type
Previous:
null
Current:
viral vector
curator
Route of administration
Previous:
null
Current:
suprachoroidal
curator
Mechanism of action
Previous:
null
Current:
genetic delivery of therapeutic protein
curator
Therapy route
Previous:
null
Current:
in-vivo
curator
Therapy type
Previous:
null
Current:
gene transfer
curator
Target gene
Previous:
null
Current:
CR2-CR1
curator
Alias type
Previous:
null
Current:
proprietary name
curator
Alias value
Previous:
null
Current:
VV-14295
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Abstract) A Novel AAV Gene Therapy Compl...
curator
Geographic Atrophy Secondary to Age-related Macular Degeneration
Kriya Therapeutics, Inc.
Recruiting
NCT05693142
Show Changes
1786424400000
News and press releases
Previous:
LinkName:(Corporate Presentation) RGX-202: AFFINIT...
Current:
LinkName:(Corporate Presentation) Delivering the p...
curator
Vector type
Previous:
AAV8
Current:
Adeno-associated virus
curator
Last update post date
Previous:
2026-06-30
Current:
2026-07-21
api
Estimated completion date
Previous:
2028-08
Current:
2028-06
api
Overall status
Previous:
Recruiting
Current:
Active not recruiting
api
Eligibility criteria
Previous:
Part 1 - key inclusion criteria:
* the participan...
Current:
Part 1 - key inclusion criteria:
* the participan...
api
Number of locations
Previous:
17
Current:
23
api
Description
Previous:
Rgx-202 is a gene therapy designed to deliver a tr...
Current:
Rgx-202 is a gene therapy designed to deliver a tr...
api
Recent updates
Previous:
Pivotal topline data announced May 2026, Sponsor p...
Current:
Pivotal topline data announced May 2026, dosing co...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Completes Dosing in Confirmator...
curator
Editor type
Previous:
none
Current:
curator
Clinical publications
Previous:
null
Current:
LinkName:(Corporate Presentation) AFFINITY DUCHENN...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Announces Positive Topline Resu...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Highlights Key 2026 Catalysts a...
curator
Duchenne Muscular Dystrophy (DMD)
REGENXBIO Inc.
Active not recruiting
Pivotal topline data announced May 2026, dosing completed in confirmatory study. Sponsor plans to pursue accelerated approval and submit a BLA in Q3 2026
NCT05444894
Show Changes
1786424400000
Clinical publications
Previous:
LinkName:(Poster) Reni-cel, the first AsCas12a gen...
Current:
LinkName:(Poster) Reni-cel, the first AsCas12a gen...
curator
Clinical publications
Previous:
LinkName:Reni-cel, the first AsCas12a gene-edited ...
Current:
null
curator
Vector type
Previous:
LV
Current:
Lentivirus
curator
Clinical publications
Previous:
null
Current:
LinkName:CRISPR-Cas12a Gene Editing of HBG1 and HB...
curator
Eligibility criteria
Previous:
null
Current:
Key inclusion criteria:
diagnosis of transfusion ...
api
Beta-Thalassemia Major
Editas Medicine, Inc.
Active not recruiting
Ended development of this program due to inability to locate a financial partner
NCT07721025
Show Changes
1786424400000
Record status
Previous:
Draft
Current:
Active
curator
Compound description
Previous:
null
Current:
AAVrh.10-CAG-hFXN
curator
Indication doid
Previous:
null
Current:
DOID:0111218
curator
Development status
Previous:
null
Current:
Active
curator
Indication
Previous:
Friedreich Ataxia, Cardiomyopathy - Secondary
Current:
Friedreich Ataxia with Secondary Cardiomyopathy
curator
Compound name
Previous:
null
Current:
LX2006
curator
Recent updates
Previous:
null
Current:
Finalized pivotal trial protocol and statistical a...
curator
Dose 2
Previous:
null
Current:
Pivotal dose: 1.2E12 vg/kg
curator
Dose 1
Previous:
null
Current:
Dose escalation study: 1.8E11 vg/kg (n=6), 5.6E11 ...
curator
Vector type
Previous:
null
Current:
Adeno-associated virus
curator
Delivery system
Previous:
null
Current:
Viral transduction
curator
Drug product type
Previous:
null
Current:
Viral vector
curator
Route of administration
Previous:
null
Current:
Intravenous
curator
Mechanism of action
Previous:
null
Current:
Functional gene replacement
curator
Therapy route
Previous:
null
Current:
in vivo
curator
Therapy type
Previous:
null
Current:
Gene transfer
curator
Target gene
Previous:
null
Current:
FXN
curator
Fda designation
Previous:
Current:
Breakthrough Therapy, Fast Track, Orphan Drug Desi...
curator
News and press releases
Previous:
null
Current:
LinkName:Lexeo Therapeutics Announces Positive Int...
curator
News and press releases
Previous:
null
Current:
LinkName:Lexeo Therapeutics Announces Positive Int...
curator
News and press releases
Previous:
null
Current:
LinkName:(Corporate Presentation) Corporate Overvi...
curator
News and press releases
Previous:
null
Current:
LinkName:(Corporate Presentation) LX2006 Regulator...
curator
News and press releases
Previous:
null
Current:
LinkName:Lexeo Therapeutics Announces Progress in ...
curator
News and press releases
Previous:
null
Current:
LinkName:Lexeo Therapeutics Announces FDA Breakthr...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Corporate Presentation) Lexeo Therapeuti...
curator
Clinical publications
Previous:
null
Current:
LinkName:AAVrh.10hFXN Gene Therapy for the Cardiom...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Expression and processing of mature human...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Stress-Induced Mouse Model of the Cardiac...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Identification of Safe and Effective Intr...
curator
Protocol
Previous:
null
Current:
LinkName:Clinical Trial Protocol;LinkUrl:https://c...
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 1: NCT05302271;LinkUrl:https://www....
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 1/2: NCT05445323;LinkUrl:https://ww...
curator
Friedreich Ataxia with Secondary Cardiomyopathy
Lexeo Therapeutics
Recruiting
Finalized pivotal trial protocol and statistical analysis plan in June 2026, 1.2E12 will be pivotal dose, topline data readout expected 2H2027
NCT06392724
Show Changes
1786424400000
Preclinical publications
Previous:
LinkName:(Abstract #20) Drug Metabolism and Pharma...
Current:
LinkName:(Abstract #20) Drug Metabolism and Pharma...
curator
Preclinical publications
Previous:
LinkName:(Abstract #19) A Transformative DMD Cytos...
Current:
null
curator
Vector type
Previous:
dual AAV9
Current:
Adeno-associated virus
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. subject age: 4-10 years ol...
api
Duchenne Muscular Dystrophy (DMD)
Peking Union Medical College Hospital
Active not recruiting
Drug is being developed by GenAssist Ltd, initial results from 3 ambulatory patients announced November 2025
NCT05820152
Show Changes
1786424400000
News and press releases
Previous:
LinkName:Neurophth Announces First Patient Dosed i...
Current:
LinkName:Neurophth Announces First Patient Dosed i...
curator
Vector type
Previous:
AAV2
Current:
Adeno-associated virus
curator
Target gene
Previous:
ND1G3460A
Current:
ND1 m.3460G>A
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
age
1. age at the time of si...
api
Leber Hereditary Optic Neuropathy (LHON)
Neurophth Therapeutics Inc
Terminated
Study terminated due to sponsor circumstances and external reasons
NCT04671433
Show Changes
1786424400000
News and press releases
Previous:
LinkName:Corporate Presentation;LinkUrl:https://in...
Current:
LinkName:Corporate Presentation - June 2026;LinkUr...
curator
Vector type
Previous:
AAV2/5
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Related nctid
Previous:
null
Current:
LinkName:Long Term Follow-Up: NCT04671433;LinkUrl:...
curator
Last update post date
Previous:
2025-09-29
Current:
2026-03-16
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* male or female
* 3 years of...
api
Number of locations
Previous:
27
Current:
28
api
Enrollment count
Previous:
97
Current:
105
api
X-Linked Retinitis Pigmentosa (XLRP)
Janssen Research & Development, LLC
Completed
Phase 3 dosing complete, phase 3 study did not meet its primary efficacy endpoint
NCT06109181
Show Changes
1786424400000
News and press releases
Previous:
LinkName:(Corporate Presentation) Corporate Overvi...
Current:
LinkName:(Corporate Presentation) Corporate Overvi...
curator
Vector type
Previous:
AAVrh10
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Selected inclusion criteria:
* adults with a clin...
api
Arrhythmogenic Right Ventricular Cardiomyopathy
Lexeo Therapeutics
Active not recruiting
Completed enrollment of LX2020 Phase 1/2 trial, additional clinical data expected January 2026
NCT07148089
Show Changes
1786424400000
Last update post date
Previous:
2026-07-07
Current:
2026-08-10
api
Vector type
Previous:
AAV8
Current:
Adeno-associated virus
curator
Record status
Previous:
Draft
Current:
Active
curator
Recent updates
Previous:
Current:
Company anticipates dosing the first participant i...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) A Novel AAV Gene Therapy Strateg...
curator
News and press releases
Previous:
null
Current:
LinkName:SEC Form 10-K: Solid Biosciences Inc. FY ...
curator
News and press releases
Previous:
null
Current:
LinkName:Solid Biosciences Reports First Quarter 2...
curator
Compound description
Previous:
Current:
AAV8-DES-hCASQ2
curator
Indication doid
Previous:
Current:
DOID:0060675
curator
Development status
Previous:
Current:
Active
curator
Compound name
Previous:
Current:
SGT-501
curator
Dose 1
Previous:
Current:
undisclosed dose escalation, 2 levels
curator
Delivery system
Previous:
Current:
Viral transduction
curator
Target tissue
Previous:
Current:
cardiomyocytes
curator
Drug product type
Previous:
Current:
Viral vector
curator
Route of administration
Previous:
Current:
Intravenous
curator
Mechanism of action
Previous:
Current:
Overexpression of protective allele/gene
curator
Therapy route
Previous:
Current:
in vivo
curator
Therapy type
Previous:
Current:
Gene transfer
curator
Target gene
Previous:
Current:
CASQ2
curator
Fda designation
Previous:
Current:
Fast Track, Orphan Drug Designation, Rare Pediatri...
curator
News and press releases
Previous:
null
Current:
LinkName:Solid Biosciences Receives FDA Fast Track...
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
type of participant and disea...
api
Catecholaminergic Polymorphic Ventricular Tachycardia
Solid Biosciences Inc.
Recruiting
Company anticipates dosing the first participant in the Phase 1b ARTEMIS clinical trial evaluating SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT) in H2 2026
NCT07667387
Show Changes
1786424400000
Last update post date
Previous:
2026-08-07
Current:
2026-08-11
api
Estimated completion date
Previous:
2028-07
Current:
2028-09
api
Overall status
Previous:
Not yet recruiting
Current:
Recruiting
api
Brief title
Previous:
A phase i/ii open-label safety and efficacy study ...
Current:
Study of lnp.ucd.abe in patients with urea cycle d...
api
Eligibility criteria
Previous:
Inclusion criteria:
1. diagnosis of a severe urea...
Current:
Inclusion criteria:
1. diagnosis of a severe urea...
api
Record status
Previous:
Draft
Current:
Active
curator
Compound description
Previous:
Current:
personalized variant-specific LNP.UCD.ABE
curator
Indication doid
Previous:
Current:
DOID:9267
curator
Development status
Previous:
Current:
Active
curator
Compound name
Previous:
Current:
LNP.UCD.ABE
curator
Recent updates
Previous:
Current:
This clinical trial protocol is a master protocol ...
curator
Dose 1
Previous:
Current:
undisclosed dose based on body weight with potenti...
curator
Editor type
Previous:
Current:
ABE mRNA
curator
Delivery system
Previous:
Current:
Endocytosis
curator
Drug product type
Previous:
Current:
Encapsulated mRNA
curator
Route of administration
Previous:
Current:
Intravenous
curator
Mechanism of action
Previous:
Current:
Mutation correction
curator
Therapy route
Previous:
Current:
in vivo
curator
Therapy type
Previous:
Current:
Gene editing
curator
Target gene
Previous:
Current:
CPS1,OTC,ASS1,ASL,ARG,NAGS,SLC25A15
curator
Preclinical publications
Previous:
null
Current:
LinkName:Pre-IND Official Responses/Meeting Summar...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Pre-IND Briefing Book;LinkUrl:https://scg...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Patient-Specific In Vivo Gene Editing to ...
curator
Preclinical publications
Previous:
null
Current:
LinkName:How to create personalized gene editing p...
curator
Urea Cycle Disorders, Carbamoyl-Phosphate Synthase I Deficiency
Rebecca Ahrens-Nicklas
Recruiting
This clinical trial protocol is a master protocol for participants with a mutation in one of the 7 genes that cause a urea cycle disorder. Participants with a variant that is amenable to correction by...
NCT05898620
Show Changes
1786424400000
News and press releases
Previous:
LinkName:Corporate Presentation - June 2024;LinkUr...
Current:
LinkName:Corporate Presentation - August 2026;Link...
curator
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Intracerebroventricular
Current:
Intraventricular
curator
Last update post date
Previous:
2026-03-13
Current:
2026-06-10
api
Overall status
Previous:
Recruiting
Current:
Active not recruiting
api
Recent updates
Previous:
Dosing completed in 2Q2026; Interim safety and eff...
Current:
Dosing completed in June 2026; Interim safety and ...
curator
News and press releases
Previous:
null
Current:
LinkName:Neurogene Announces Successful Completion...
curator
Fda designation
Previous:
Fast Track, Orphan Drug Designation, Rare Pediatri...
Current:
Breakthrough Therapy, Fast Track, Orphan Drug Desi...
curator
News and press releases
Previous:
null
Current:
LinkName:Neurogene Announces Recent Achievements a...
curator
News and press releases
Previous:
null
Current:
LinkName:Neurogene Announces FDA Breakthrough Ther...
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* females who are between the...
api
Rett Syndrome
Neurogene Inc.
Active not recruiting
Dosing completed in June 2026; Interim safety and efficacy data in both age cohorts expected mid-2026, topline data expected 2H27
NCT05821959
Show Changes
1786424400000
News and press releases
Previous:
LinkName:SEC Form 10-K: 2024 Annual Report;LinkUrl...
Current:
LinkName:SEC Form 10-K: 2024 Annual Report;LinkUrl...
curator
Indication
Previous:
Sensorineural Hearing Loss, Bilateral
Current:
Bilateral Sensorineural Hearing Loss
curator
Vector type
Previous:
dual Anc80L65
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Criteria for inclusion:
1. participants may be of...
api
Bilateral Sensorineural Hearing Loss
Akouos, Inc.
Recruiting
NCT03566043
Show Changes
1786424400000
Clinical publications
Previous:
LinkName:(Corporate Presentation) Rare Program Upd...
Current:
LinkName:(Corporate Presentation) Delivering the p...
curator
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Compound name
Previous:
RGX-121
Current:
NAVSUNLI
curator
Recent updates
Previous:
FDA placed this study on clinical hold in January ...
Current:
CRL issued 2/7/26 (issues with study eligibility c...
curator
Alias type
Previous:
proper name
Current:
proper name, proprietary name
curator
Alias value
Previous:
clemidosogene lanparvovec
Current:
clemidosogene lanparvovec, RGX-121
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Announces Alignment with FDA on...
curator
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Intracisterna magna
Current:
Intracisternal
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 3: NCT07236606;LinkUrl:https://clin...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Announces Regulatory Update on ...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Announces Regulatory Update on ...
curator
Eligibility criteria
Previous:
null
Current:
Part 1 inclusion criteria:
* the subject's legal ...
api
Mucopolysaccharidosis Type II (Hunter Syndrome)
REGENXBIO Inc.
Active not recruiting
CRL issued 2/7/26 (issues with study eligibility criteria, comparability of natural history control arm, appropriateness of surrogate endpoint), Company appealed this decision and reached alignment wi...
NCT06611436
Show Changes
1786424400000
Recent updates
Previous:
Program terminated due to strategic business decis...
Current:
Program terminated due to strategic business decis...
curator
Protocol
Previous:
LinkName:(Poster) BeCoMe-9: A Phase 1/2 Dose Escal...
Current:
LinkName:(Abstract) BeCoMe-9: A Phase 1/2 Dose Esc...
curator
Preclinical publications
Previous:
LinkName:(Presentation) Development of an Ex Vivo ...
Current:
LinkName:(Abstract) Development of an Ex Vivo Prec...
curator
News and press releases
Previous:
LinkName:FDA Grants Orphan Drug Designation for BE...
Current:
LinkName:FDA Grants Orphan Drug Designation for BE...
curator
Development status
Previous:
Active
Current:
Inactive
curator
News and press releases
Previous:
null
Current:
LinkName:Be Bio bins hemophilia B cell therapy tri...
curator
News and press releases
Previous:
LinkName:Be Bio Announces First Participant Dosed ...
Current:
null
curator
Vector type
Previous:
AAV6
Current:
Adeno-associated virus
curator
Dose 3
Previous:
Undisclosed dose 3
Current:
curator
Dose 2
Previous:
Undisclosed dose 2
Current:
curator
Dose 1
Previous:
Undisclosed dose 1
Current:
Undisclosed dose escalation, 3 levels
curator
Last update post date
Previous:
2025-10-20
Current:
2026-06-15
api
Estimated completion date
Previous:
2027-07
Current:
2026-05-29
api
Overall status
Previous:
Recruiting
Current:
Terminated
api
Number of locations
Previous:
4
Current:
5
api
Enrollment type
Previous:
Estimated
Current:
Actual
api
Enrollment count
Previous:
24
Current:
5
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* adult males (≥18) with mode...
api
Hemophilia B
Be Biopharma
Terminated
Program terminated due to strategic business decisions, website is no longer available
NCT05248230
Show Changes
1786424400000
Clinical publications
Previous:
LinkName:(Corporate Presentation) Aerosolized 4D-7...
Current:
LinkName:(Corporate Presentation) 4D-710 for Cysti...
curator
Dose 4
Previous:
2E15 vg (discontinued due to high transduction to ...
Current:
curator
Dose 3
Previous:
1E15 vg (maximum tolerated dose)
Current:
curator
Dose 2
Previous:
5E14 vg
Current:
Phase 2 doses: 2.5E14 vg (anticipated pivotal/comm...
curator
Dose 1
Previous:
2.5E14 vg (anticipated pivotal/commercial dose)
Current:
Phase 1 doses: 1E15 vg (n=3), 2E15 vg (n=4, was di...
curator
News and press releases
Previous:
LinkName:Corporate Presentation - November 2025;Li...
Current:
LinkName:Corporate Presentation - July 2026;LinkUr...
curator
Vector type
Previous:
AAV A101
Current:
Adeno-associated virus
curator
Last update post date
Previous:
2026-03-11
Current:
2026-07-06
api
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Inhalational
Current:
Respiratory (inhalation)
curator
Eligibility criteria
Previous:
null
Current:
Key inclusion criteria (primary study):
1. 18 yea...
api
Estimated completion date
Previous:
2030-01
Current:
2031-06
api
Number of locations
Previous:
19
Current:
20
api
Enrollment count
Previous:
40
Current:
30
api
Cystic Fibrosis
4D Molecular Therapeutics
Recruiting
Interim data announced December 2025
NCT05878860
Show Changes
1786424400000
Last update post date
Previous:
2026-08-04
Current:
2026-08-10
api
Number of locations
Previous:
18
Current:
19
api
Locations
Previous:
United states
Current:
United states, United kingdom
api
Vector type
Previous:
AAV.SPR
Current:
Adeno-associated virus
curator
News and press releases
Previous:
LinkName:
Atsena Therapeutics Doses First Patient...
Current:
LinkName:Atsena Therapeutics Doses First Patient a...
curator
Recent updates
Previous:
Enrollment in pivotal cohort is expected to comple...
Current:
First patient in pivotal cohort has been dosed. En...
curator
Editor type
Previous:
none
Current:
curator
Dose 5
Previous:
2.3E10 vg/eye (225ul)
Current:
curator
Dose 4
Previous:
1.5E10 vg/eye (150ul)
Current:
curator
Dose 3
Previous:
5.0E10 vg/eye
Current:
Phase 3: 1.1E10 vg/eye; n=39
curator
Dose 2
Previous:
3.0E10 vg/eye
Current:
Phase 1/2 Part B: 1.1E10 vg/eye, 1.7 vg/eye; n=3/c...
curator
Dose 1
Previous:
1.5E10 vg/eye
Current:
Phase 1/2 Part A: 1.1E10 vg/eye, 3.8E10 vg/eye, 2....
curator
Clinical publications
Previous:
LinkName:Safety and efficacy of ATSN-101 in patien...
Current:
null
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Safety and efficacy of ATS...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Safety and efficacy of ATS...
curator
News and press releases
Previous:
null
Current:
LinkName:Atsena Presents Positive Clinical Data fr...
curator
News and press releases
Previous:
LinkName:
Atsena Presents Positive Interim Six-Mo...
Current:
LinkName:Atsena Presents Positive Interim Six-Mont...
curator
News and press releases
Previous:
LinkName:
Atsena Therapeutics Receives Data Monit...
Current:
LinkName:Atsena Therapeutics Receives Data Monitor...
curator
Estimated completion date
Previous:
2029-10
Current:
2033-04
api
Official title
Previous:
A phase 1/2, Open-label, Dose escalation and dose ...
Current:
A phase 1/2/3, Open-label, Dose escalation, Dose e...
api
Eligibility criteria
Previous:
Inclusion criteria:
1. age ≥ 18 for cohorts 1 thr...
Current:
Part a and b:
inclusion criteria:
1. age ≥ 18 fo...
api
Eligibility sex
Previous:
Male
Current:
All
api
Enrollment count
Previous:
21
Current:
97
api
Phases
Previous:
Phase1, Phase2
Current:
Phase3
api
Description
Previous:
This study will evaluate the safety and tolerabili...
Current:
This study will evaluate the safety and efficacy o...
api
X-Linked Retinoschisis
Atsena Therapeutics Inc.
Recruiting
First patient in pivotal cohort has been dosed. Enrollment in pivotal cohort is expected to complete by end of Q1 2027, topline results expected 1H2028, BLA filing targeted for 2H2028
NCT04853576
Show Changes
1786424400000
News and press releases
Previous:
LinkName:Corporate Presentation - June 2024;LinkUr...
Current:
LinkName:Editas Medicine Announces Fourth Quarter ...
curator
News and press releases
Previous:
LinkName:(Corporate Presentation) Strategic Update...
Current:
LinkName:Editas Medicine Announces Third Quarter 2...
curator
Vector type
Previous:
LV
Current:
Lentivirus
curator
Editor type
Previous:
ASCas12a
Current:
ASCas12a mRNA
curator
Delivery system
Previous:
Current:
Viral transduction
curator
Clinical publications
Previous:
null
Current:
LinkName:CRISPR-Cas12a Gene Editing of HBG1 and HB...
curator
Eligibility criteria
Previous:
null
Current:
Key inclusion criteria:
diagnosis of severe sickl...
api
Sickle Cell Disease
Editas Medicine, Inc.
Active not recruiting
Ended development of this program due to inability to locate financial partner
NCT03634007
Show Changes
1786424400000
Recent updates
Previous:
Originally developed by Cornell University, licens...
Current:
Originally developed by Cornell University, licens...
curator
News and press releases
Previous:
LinkName:Corporate Overview - November 2025;LinkUr...
Current:
LinkName:SEC Form 10-K: Lexeo Therapeutics, Inc FY...
curator
Vector type
Previous:
AAVrh10
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* apoe4 homozygotes
* willing...
api
Alzheimer's Disease, Early Onset Alzheimer's Disease
Lexeo Therapeutics
Completed
Originally developed by Cornell University, licensed to Lexeo in May 2020, Lexeo announced they were pursuing partnership opportunities for continued development of LX1001 in January 2025
NCT05139316
Show Changes
1786424400000
Clinical publications
Previous:
LinkName:(Corporate Presentation) Top-Line Phase 3...
Current:
LinkName:(Corporate Presentation) August 2026;Link...
curator
Vector type
Previous:
AAV8
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Related nctid
Previous:
null
Current:
LinkName:Long Term Follow-Up: NCT03970278;LinkUrl:...
curator
Last update post date
Previous:
2026-03-11
Current:
2026-03-25
api
Estimated completion date
Previous:
2026-02-05
Current:
2026-02-20
api
Eligibility criteria
Previous:
null
Current:
Key inclusion criteria:
* documented gsdia with c...
api
Recent updates
Previous:
BLA Submission completed 12/30/25
Current:
BLA Submission completed 12/30/25, PDUFA Action Da...
curator
Fda designation
Previous:
Fast Track, Orphan Drug Designation, Rare Pediatri...
Current:
Fast Track, Orphan Drug Designation, Priority Revi...
curator
Clinical publications
Previous:
null
Current:
LinkName:Trial Interviews to Explore Glycogen Stor...
curator
News and press releases
Previous:
null
Current:
LinkName:Ultragenyx Reports Fourth Quarter and Ful...
curator
News and press releases
Previous:
null
Current:
LinkName:Ultragenyx Announces U.S. FDA Acceptance ...
curator
Overall status
Previous:
Active not recruiting
Current:
Completed
api
Glycogen Storage Disease Type Ia
Ultragenyx Pharmaceutical Inc
Completed
BLA Submission completed 12/30/25, PDUFA Action Date set for 8/23/26
NCT03368742
Show Changes
1786424400000
Last update post date
Previous:
2026-07-07
Current:
2026-08-10
api
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* established clinical diagno...
api
Duchenne Muscular Dystrophy (DMD)
Solid Biosciences Inc.
Active not recruiting
In September 2022, Solid Biosciences announced they would be pausing activities for SGT-001; "No longer developing" as of 2024 Annual Report
NCT05606614
Show Changes
1786424400000
Compound description
Previous:
Current:
scAAV-MeP426-miniMECP2-miRARE
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster) rAAV9 Vector Biodistribution in ...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster) Every Gain, Expanding Possibilit...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster) REVEAL Adolescent/Adult and Pedi...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster) Achieving Developmental Mileston...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Poster #54) Superior expression of self-...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster #7) Establishing the Rett Syndrom...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster #39) The Developmental Plateau in...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster #59) Safety and Efficacy Results ...
curator
Clinical publications
Previous:
LinkName:(Corporate Presentation) November 2024;Li...
Current:
LinkName:(Corporate Presentation) July 2026;LinkUr...
curator
Clinical publications
Previous:
LinkName:(Corporate Presentation) TSHA-102 in clin...
Current:
LinkName:(Corporate Presentation) TSHA-102 Rett Sy...
curator
Vector type
Previous:
scAAV9
Current:
Adeno-associated virus
curator
Last update post date
Previous:
2025-12-30
Current:
2026-07-07
api
Overall status
Previous:
Recruiting
Current:
Active not recruiting
api
Number of locations
Previous:
6
Current:
9
api
Enrollment type
Previous:
Estimated
Current:
Actual
api
Enrollment count
Previous:
15
Current:
17
api
Recent updates
Previous:
First patient dosed in REVEAL pivotal trial (adole...
Current:
Dosing completed in pivotal trial, topline data an...
curator
News and press releases
Previous:
null
Current:
LinkName:Taysha Gene Therapies Announces Completio...
curator
Editor type
Previous:
none
Current:
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 3: NCT07480564 (Pediatric cohort);L...
curator
Fda designation
Previous:
Fast Track, Orphan Drug Designation, Rare Pediatri...
Current:
Breakthrough Therapy, Fast Track, Orphan Drug Desi...
curator
News and press releases
Previous:
null
Current:
LinkName:Taysha Gene Therapies Announces Progress ...
curator
News and press releases
Previous:
null
Current:
LinkName:SEC Form 10-K: Taysha Gene Therapies, Inc...
curator
News and press releases
Previous:
null
Current:
LinkName:Taysha Gene Therapies Regains Full Rights...
curator
Record status
Previous:
Inactive
Current:
Active
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* females between the ages of...
api
Rett Syndrome
Taysha Gene Therapies, Inc.
Active not recruiting
Dosing completed in pivotal trial, topline data and FDA feedback on BLA submission pathway expected 1H 2027, BLA submission expected 1H 2027
NCT05228145
Show Changes
1786424400000
News and press releases
Previous:
LinkName:Corporate Presentation - June 2024;LinkUr...
Current:
LinkName:SEC Form 10-Q: Neurogene Inc., 1Q25;LinkU...
curator
Vector type
Previous:
AAV9
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Intracerebroventricular, intravitreal
Current:
Intraventricular, Intravitreal
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria
* age from 3 to 9 years (child...
api
Neuronal Ceroid Lipofuscinosis CLN5
Neurogene Inc.
Active not recruiting
RMAT application was denied by FDA, company discontinued the program
NCT06699108
Show Changes
1786424400000
Vector type
Previous:
AAV2/8
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Key inclusion criteria:
1. able to provide inform...
api
Bietti Crystalline Dystrophy
Shanghai Vitalgen BioPharma Co., Ltd.
Active not recruiting
Phase 3 enrollment complete, BLA submission target 2H 2026
NCT07592273
Show Changes
1786424400000
Last update post date
Previous:
2026-07-29
Current:
2026-08-05
api
Number of locations
Previous:
18
Current:
23
api
Vector type
Previous:
AAV8
Current:
Adeno-associated virus
curator
Recent updates
Previous:
First patient has been dosed in the Phase IIb/III ...
Current:
First patient has been dosed in the Phase IIb/III ...
curator
Dose 1
Previous:
Phase 2 doses: 2.5E11 GC/eye, 5.0E11 GC/eye,1.0E12...
Current:
Phase 2 doses: 2.5E11 GC/eye, 5.0E11 GC/eye, 1.0E1...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Suprachoroidal Delivery of...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Presents Positive Long-Term Dat...
curator
Related nctid
Previous:
LinkName:Phase 2: NCT06942520 (uses subretinal inj...
Current:
LinkName:Phase 2: NCT06942520 (using subretinal in...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Announces First Patient Dosed i...
curator
Locations
Previous:
United states
Current:
Puerto rico, United states
api
Indication doid
Previous:
DOID:9191, DOID:8947
Current:
DOID:8947
curator
Record status
Previous:
Draft
Current:
Active
curator
Dose 3
Previous:
1.0E12 GC/eye
Current:
curator
Dose 2
Previous:
5.0E11 GC/eye
Current:
Phase 3 dose: 1.0E12 GC/eye
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Announces Pivotal Program for S...
curator
Development status
Previous:
Current:
Active
curator
Compound name
Previous:
Current:
Surabgene lomparvovec
curator
Delivery system
Previous:
Current:
Viral transduction
curator
Drug product type
Previous:
Current:
Viral vector
curator
Route of administration
Previous:
Current:
Suprachoroidal
curator
Mechanism of action
Previous:
Current:
Genetic delivery of therapeutic protein
curator
Therapy route
Previous:
Current:
In-vivo
curator
Therapy type
Previous:
Current:
Gene transfer
curator
Target gene
Previous:
Current:
Anti-VEGF
curator
Alias type
Previous:
null
Current:
proprietary name
curator
Alias value
Previous:
null
Current:
ABBV-RGX-314
curator
Related nctid
Previous:
null
Current:
LinkName:Long Term Follow-Up: NCT05296447;LinkUrl:...
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 2: NCT04567550;LinkUrl:https://clin...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Reports Third Quarter 2025 Fina...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Suprachoroidal Surabgene L...
curator
Estimated completion date
Previous:
2036-02
Current:
2036-01
api
Overall status
Previous:
Not yet recruiting
Current:
Recruiting
api
Diabetic Retinopathy
AbbVie
Recruiting
First patient has been dosed in the Phase IIb/III trial using suprachoroidal delivery. Phase IIb data was presented at the ASRS annual meeting in July 2026. This product is also under evaluation for w...
NCT05441553
Show Changes
1786424400000
Vector type
Previous:
AAV8
Current:
Adeno-associated virus
curator
Therapy route
Previous:
In-vivo
Current:
in vivo
curator
Dose 1
Previous:
Undisclosed dose (ranging from 4E11 - 2E12 vg/kg)
Current:
Undisclosed dose
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. male ≥18 years and ≤65year...
api
Hemophilia B
Shanghai Vitalgen BioPharma Co., Ltd.
Unknown
Granted ODD in December 2021
NCT05603312
Show Changes
1786424400000
News and press releases
Previous:
LinkName:Corporate Presentation November 2025;Link...
Current:
LinkName:MeiraGTx Reports First Quarter 2026 Finan...
curator
Target tissue
Previous:
Current:
Subthalamic nucleus
curator
Route of administration
Previous:
Intraparenchymal (subthalamic nucleus)
Current:
Intraparenchymal
curator
Vector type
Previous:
AAV2
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* confirmed parkinson's disea...
api
Parkinson's Disease
MeiraGTx, LLC
Completed
Initiation of Phase III design discussions in H2 2024
NCT06325709
Show Changes
1786078800000
Last update post date
Previous:
2026-08-04
Current:
2026-08-07
api
Description
Previous:
Background:
chronic granulomatous disease (cgd) i...
Current:
Background:
chronic granulomatous disease (cgd) i...
api
Enrollment count
Previous:
10
Current:
15
api
Eligibility criteria
Previous:
* inclusion criteria:
-\>= 18 years of age.
* c...
Current:
* inclusion criteria:
-\>= 18 years of age.
* c...
api
Intervention description
Previous:
Stem cell mobilizing agent: subcutaneous administr...
Current:
Immunomodulating agent: daily subcutaneous adminis...
api
Intervention name
Previous:
Plerixafor, Filgrastim, Palifermin, Busulfan, Base...
Current:
Campath, Sirolimus, Base-edited hematopoietic stem...
api
Vector type
Previous:
none
Current:
curator
X-Linked Chronic Granulomatous Disease
National Institute of Allergy and Infectious Diseases (NIAID)
Recruiting
Trial stoppage rules triggered due to a serious adverse event in early 2025, study allowed to resume in July 2025
NCT06884865
Show Changes
1786078800000
Last update post date
Previous:
2026-03-24
Current:
2026-08-07
api
Overall status
Previous:
Active not recruiting
Current:
Recruiting
api
Eligibility criteria
Previous:
50 years ii. morning stiffness \<30 minutes iii. crepitus on knee motion c. osteophytes
* subjects must have k-l grade 2, 3, Or 4 in the index knee based on x-rays performed during screening and confirmed by trained radiographers at a central facility before enrollment
* subjects need to show the presence of moderate or severe synovitis based on 11-point synovitis score using contrast-enhanced mri
exclusion criteria:
* subjects have any current or prior diagnosis of autoimmune connective tissue disorders, Secondary oa conditions, Benign synovial tumors, Gout/pseudogout, Reactive arthritis, Ra, Psoriatic arthritis, Ankylosing spondylitis, Or arthritis associated with inflammatory bowel disease.
* subjects have any active systemic or local infection, Including infection of the index knee
* subjects are unable to undergo mri with contrast mri
* subjects with x-ray or mri exclusionary events
* subjects have an unstable index knee joint (eg, Torn anterior cruciate ligament) within 12 months of screening
* subjects have used any approved or investigational ia drug/biologic in index knee within 6 months of screening (eg, Hyaluronic acid, Platelet rich plasma, Stem cells, Prolotherapy, And amniotic fluid injection)
* subjects are receiving or have received any gene therapy treatment (eg, Il-1ra) in the past 3 years
* subjects have used ia steroids ≤3 months before screening
other protocol-defined criteria apply">Inclusion criteria:
* subjects must be willing an...
Current:
50 years ii. morning stiffness \<30 minutes iii. crepitus on knee motion c. osteophytes
* subjects must have k-l grade 2, 3, Or 4 (part a) or k-l grade 2 or 3 (part b) in the index knee based on x-rays performed during screening and confirmed by trained radiographers at a central facility before enrollment
* subjects need to show the presence of moderate or severe synovitis based on 11-point synovitis score using contrast-enhanced mri (part a). for part b, Participants need to show the presence of synovitis ≥5 on the guermazi synovitis scoring scale using contrast-enhanced mri to be enrolled.
exclusion criteria:
* subjects have any current or prior diagnosis of autoimmune connective tissue disorders, Secondary oa conditions, Benign synovial tumors, Gout/pseudogout, Reactive arthritis, Ra, Psoriatic arthritis, Ankylosing spondylitis, Or arthritis associated with inflammatory bowel disease.
* subjects have any active systemic or local infection, Including infection of the index knee
* subjects are unable to undergo mri with contrast mri
* subjects with x-ray or mri exclusionary events
* subjects have an unstable index knee joint (eg, Torn anterior cruciate ligament) within 12 months of screening
* subjects have used any approved or investigational ia drug/biologic in index knee within 6 months of screening (eg, Hyaluronic acid, Platelet rich plasma, Stem cells, Prolotherapy, And amniotic fluid injection)
* subjects are receiving or have received any gene therapy treatment (eg, Il-1ra) in the past 3 years
* subjects have used ia steroids ≤3 months before screening
* for part b, Subjects with any surgery related tot he index knee.
other protocol-defined criteria apply">Inclusion criteria:
* subjects must be willing an...
api
Number of locations
Previous:
17
Current:
40
api
Vector type
Previous:
Ad5
Current:
Adenovirus
curator
Editor type
Previous:
none
Current:
curator
Route of administration
Previous:
Intraarticular
Current:
Intra-articular
curator
Compound description
Previous:
null
Current:
AdV-NFKB-IL1RA
curator
Indication doid
Previous:
null
Current:
DOID:8398
curator
Development status
Previous:
null
Current:
Active
curator
Record status
Previous:
Provisional
Current:
Active
curator
Compound name
Previous:
null
Current:
PCRX-201
curator
Recent updates
Previous:
null
Current:
Pacira announced Phase 2 enrollment was complete i...
curator
Dose 3
Previous:
null
Current:
1.4E12 gc/knee
curator
Dose 2
Previous:
null
Current:
1.4E11 gc/knee
curator
Dose 1
Previous:
null
Current:
1.4E10 gc/knee
curator
Delivery system
Previous:
null
Current:
Viral transduction
curator
Drug product type
Previous:
null
Current:
Viral vector
curator
Mechanism of action
Previous:
null
Current:
Overexpression of protective allele/gene
curator
Therapy route
Previous:
null
Current:
In-vivo
curator
Therapy type
Previous:
null
Current:
gene transfer
curator
Target gene
Previous:
null
Current:
IL1RA
curator
Alias type
Previous:
null
Current:
proper name, proprietary name
curator
Alias value
Previous:
null
Current:
Enekinragene inzadenovec, FX-201
curator
Fda designation
Previous:
Current:
Regenerative Medicine Advanced Therapy
curator
Preclinical publications
Previous:
null
Current:
LinkName:PCRX-201, a novel IL-1Ra gene therapy tre...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Efficacy and Safety of FX201, a Novel Int...
curator
News and press releases
Previous:
null
Current:
LinkName:Pacira BioSciences Announces PCRX-201 Gra...
curator
News and press releases
Previous:
null
Current:
LinkName:(Corporate Presentation) An Introduction ...
curator
News and press releases
Previous:
null
Current:
LinkName:Pacira BioSciences Concludes Patient Enro...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Abstract POS0492) A SINGLE INTRA-ARTICUL...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Abstract #594) Interim Data from the Fir...
curator
Estimated completion date
Previous:
2032-03
Current:
2032-07
api
Osteoarthritis (OA) of the Knee
Pacira Pharmaceuticals, Inc
Recruiting
Pacira announced Phase 2 enrollment was complete in November 2025
NCT07052929
Show Changes
1786078800000
Last update post date
Previous:
2026-07-15
Current:
2026-08-07
api
Number of locations
Previous:
4
Current:
5
api
Vector type
Previous:
AAV3.8
Current:
Adeno-associated virus
curator
Locations
Previous:
United states
Current:
Canada, United states
api
Record status
Previous:
Draft
Current:
Active
curator
Target tissue
Previous:
Current:
muscle cells
curator
Recent updates
Previous:
Astellas has restarted their XLMTM program by crea...
Current:
Astellas has restarted their XLMTM program by crea...
curator
Dose 2
Previous:
Current:
2E12 vg/kg
curator
Dose 1
Previous:
Current:
1E12 vg/kg
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 2/3: NCT03199469 (prior drug produc...
curator
Related nctid
Previous:
null
Current:
LinkName:Epidemiological study: NCT06581146;LinkUr...
curator
News and press releases
Previous:
null
Current:
LinkName:Astellas Takes Aim at XLMTM Again, Now wi...
curator
News and press releases
Previous:
null
Current:
LinkName:VALOR Clinical Trial Patient Information;...
curator
Protocol
Previous:
null
Current:
LinkName:(Abstract) VALOR study design: a phase 1/...
curator
Protocol
Previous:
null
Current:
LinkName:(Abstract 74P) Design of a phase 1/2 stud...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Abstract) Efficacy, Biodistribution, and...
curator
Compound description
Previous:
Current:
MyoAAV3.8-MHCK7-hMTM1
curator
Indication doid
Previous:
Current:
DOID:0111225
curator
Development status
Previous:
Current:
Active
curator
Compound name
Previous:
Current:
ASP2957
curator
Delivery system
Previous:
Current:
viral transduction
curator
Drug product type
Previous:
Current:
viral vector
curator
Route of administration
Previous:
Current:
intravenous
curator
Mechanism of action
Previous:
Current:
functional gene replacement
curator
Therapy route
Previous:
Current:
in-vivo
curator
Therapy type
Previous:
Current:
gene transfer
curator
Target gene
Previous:
Current:
MTM1
curator
Alias type
Previous:
null
Current:
proprietary name
curator
Alias value
Previous:
null
Current:
KT430
curator
Clinical publications
Previous:
null
Current:
LinkName:Intrahepatic Cholestasis Is a Clinically ...
curator
Clinical publications
Previous:
null
Current:
LinkName:Hepatobiliary disease in XLMTM: a common ...
curator
News and press releases
Previous:
null
Current:
LinkName:Astellas Sees Path Forward for Gene Thera...
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* participant is projected to...
api
X-Linked Myotubular Myopathy
Astellas Gene Therapies
Recruiting
Astellas has restarted their XLMTM program by creating a new drug product ASP2957, with substantial modifications to the capsid to detarget the liver, and includes a muscle-specific promotor MHCK7, IN...
NCT04040049
Show Changes
1785992400000
With has results
Previous:
False
Current:
True
api
Last update post date
Previous:
2023-06-05
Current:
2026-08-06
api
Vector type
Previous:
AAVS3
Current:
Adeno-associated virus
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. adult males, ≥ 18 years of...
api
Fabry Disease
Spur Therapeutics
Terminated
Development cancelled to prioritize other programs
NCT05788536
Show Changes
1785906000000
Last update post date
Previous:
2026-07-01
Current:
2026-08-05
api
Number of locations
Previous:
16
Current:
17
api
Vector type
Previous:
dual AAV1
Current:
Adeno-associated virus
curator
Browse condition terms
Previous:
Db-oto, Gene therapy, Congenital hearing loss, Sen...
Current:
Db-oto, Gene therapy, Congenital hearing loss, Sen...
api
Estimated completion date
Previous:
2032-02-28
Current:
2032-02-25
api
Brief title
Previous:
A study of db-oto, An adeno-associated virus (aav)...
Current:
A study of db-oto, An adeno-associated virus (aav)...
api
Eligibility std age
Previous:
Child
Current:
Child, Adult, Older adult
api
Eligibility max age
Previous:
17 years
Current:
null
api
Eligibility criteria
Previous:
Key inclusion criteria:
1. willingness of at leas...
Current:
Key inclusion criteria:
1. willingness to provide...
api
Enrollment count
Previous:
30
Current:
36
api
Intervention description
Previous:
Db-oto will be administered as a single intracochl...
Current:
Administered per the protocol
api
Intervention name
Previous:
Db-oto, Db-oto
Current:
Db-oto
api
Description
Previous:
Regeneron is conducting a study of an investigatio...
Current:
Regeneron is conducting a study of an investigatio...
api
Editor type
Previous:
none
Current:
curator
Recent updates
Previous:
Otarmeni was approved 4/23/26 under the National P...
Current:
Otarmeni was approved 4/23/26 under the National P...
curator
News and press releases
Previous:
null
Current:
LinkName:Otarmeni™ (lunsotogene parvec) Receives E...
curator
Locations
Previous:
United states, United kingdom, Germany, Spain
Current:
United states, Japan, United kingdom, Germany, Spa...
api
Development status
Previous:
Active
Current:
Approved
curator
Compound name
Previous:
DB-OTO
Current:
OTARMENI
curator
Alias type
Previous:
null
Current:
proprietary name, proper name
curator
Alias value
Previous:
null
Current:
DB-OTO, lunsotogene parvec
curator
Fda designation
Previous:
Fast Track, Orphan Drug Designation, Rare Pediatri...
Current:
Accelerated Approval, Fast Track, Orphan Drug Desi...
curator
News and press releases
Previous:
null
Current:
LinkName:FDA Approval Documents;LinkUrl:https://ww...
curator
News and press releases
Previous:
null
Current:
LinkName:FDA Approves First-Ever Gene Therapy for ...
curator
Congenital Hearing Loss Secondary to Biallelic Mutations of the Otoferlin Gene (OTOF)
Regeneron Pharmaceuticals
Recruiting
Otarmeni was approved 4/23/26 under the National Priority Voucher Program, Marketing Authorization Application was accepted for review by the EMA
NCT07226206
Show Changes
1785819600000
Last update post date
Previous:
2026-07-14
Current:
2026-08-04
api
Number of locations
Previous:
2
Current:
3
api
Vector type
Previous:
LK03
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
Overall status
Previous:
Not yet recruiting
Current:
Recruiting
api
Compound description
Previous:
AAV LK03 capsid + TTRmut-hFVIII-X07
Current:
AAV LK03 capsid + TTRmut + APC-resistant FVIII var...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Activated protein C has a regulatory role...
curator
Indication doid
Previous:
null
Current:
DOID:12134
curator
Development status
Previous:
null
Current:
Active
curator
Record status
Previous:
Provisional
Current:
Active
curator
Compound name
Previous:
null
Current:
SPK-8011QQ
curator
Recent updates
Previous:
null
Current:
Study was withdrawn by Sponsor (no participants we...
curator
Dose 4
Previous:
null
Current:
2E12 vg/kg
curator
Dose 3
Previous:
null
Current:
1.5E12 vg/kg
curator
Dose 2
Previous:
null
Current:
1E12 vg/kg
curator
Dose 1
Previous:
null
Current:
5E11 vg/kg
curator
Delivery system
Previous:
null
Current:
Viral transduction
curator
Drug product type
Previous:
null
Current:
Viral vector
curator
Route of administration
Previous:
null
Current:
Intravenous
curator
Mechanism of action
Previous:
null
Current:
Functional gene replacement
curator
Therapy route
Previous:
null
Current:
In-vivo
curator
Therapy type
Previous:
null
Current:
Gene transfer
curator
Target gene
Previous:
null
Current:
F8
curator
Alias type
Previous:
null
Current:
proper name
curator
Alias value
Previous:
null
Current:
Dirloctogene samoparvovec
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 3: NCT06297486 (Withdrawn);LinkUrl:...
curator
Related nctid
Previous:
null
Current:
LinkName:Phase 1/2: NCT03003533;LinkUrl:https://ww...
curator
Related nctid
Previous:
null
Current:
LinkName:Long Term Follow-Up: NCT03432520;LinkUrl:...
curator
Preclinical publications
Previous:
null
Current:
LinkName:Preclinical assessment of an optimized AA...
curator
Preclinical publications
Previous:
null
Current:
LinkName:(Abstract) Preclinical evaluation of SPK-...
curator
News and press releases
Previous:
null
Current:
LinkName:Roche mothballs another hemophilia A gene...
curator
News and press releases
Previous:
null
Current:
LinkName:Integration of Spark into Roche Group Set...
curator
Clinical publications
Previous:
null
Current:
LinkName:Multiyear Factor VIII Expression after AA...
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* signed informed consent for...
api
Locations
Previous:
Current:
United states
api
Hemophilia A
Hoffmann-La Roche
Recruiting
Study was withdrawn by Sponsor (no participants were enrolled), Sponsor is extensively modifying the product before clinical trials resume
NCT07195825
Show Changes
1785819600000
Last update post date
Previous:
2026-01-28
Current:
2026-08-04
api
Vector type
Previous:
AAVT42
Current:
Adeno-associated virus
curator
Compound description
Previous:
Current:
AAVT42-hSyn-TH90del-T2A-AADC-WPRE
curator
Recent updates
Previous:
Current:
New results published June 2026, safety and tolera...
curator
Dose 1
Previous:
Undisclosed
Current:
Dose escalation phase: 4.0E11 vg (Cohort 1, n=1), ...
curator
Target tissue
Previous:
Current:
Putamen
curator
Mechanism of action
Previous:
Undisclosed
Current:
Overexpression of protective allele/gene
curator
Therapy type
Previous:
Undisclosed
Current:
Gene transfer
curator
Target gene
Previous:
Undisclosed
Current:
TH90del-AADC
curator
Clinical publications
Previous:
null
Current:
LinkName:Dual-target gene therapy in Parkinson’s d...
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
1. participants with a diagno...
api
Parkinson's Disease
Shanghai Xinzhi BioMed Co., Ltd.
Recruiting
New results published June 2026, safety and tolerability, no treatment-related SAEs or DLTs
NCT02599922
Show Changes
1785733200000
Overall status
Previous:
Active not recruiting
Current:
Unknown
curator
Vector type
Previous:
AAV2tYF
Current:
Adeno-associated virus
curator
Dose 5
Previous:
1.1E12 vg/ml; Dose 6: 3.2E12
Current:
curator
Dose 4
Previous:
3.6E11 vg/ml
Current:
curator
Dose 3
Previous:
1.2E11 vg/ml
Current:
curator
Dose 2
Previous:
4E10 vg/ml
Current:
curator
Dose 1
Previous:
2E11 vg/ml
Current:
Phase 1/2 study: 2E11 vg/ml, 4E10 vg/ml, 1.2E11 vg...
curator
Editor type
Previous:
none
Current:
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria include:
1. male or female sub...
api
Achromatopsia
Beacon Therapeutics
Unknown
Development discontinued by Sponsor
NCT03920007
Show Changes
1785733200000
With has results
Previous:
False
Current:
True
curator
Last update post date
Previous:
2024-02-20
Current:
2026-08-03
curator
Dose 1
Previous:
Dose escalation phase: 1.0E10 vg/eye (300ul),3.0E1...
Current:
Dose escalation phase: 1.0E10 vg/eye (300ul), 3.0E...
curator
Vector type
Previous:
AAV2/5
Current:
Adeno-associated virus
curator
Editor type
Previous:
none
Current:
curator
News and press releases
Previous:
LinkName:
Atsena Presents Positive Clinical Data ...
Current:
LinkName:Atsena Presents Positive Clinical Data fr...
curator
Recent updates
Previous:
Current:
36 month safety and efficacy data presented in May...
curator
Dose 3
Previous:
1.0E11 vg/eye (300ul), n=9
Current:
curator
Dose 2
Previous:
3.0E10 vg/eye (300ul), n=3
Current:
Dose expansion phase: 1.0E11 vg/eye (n=6)
curator
Clinical publications
Previous:
null
Current:
LinkName:(Poster) Modified Multi-Luminance Mobilit...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Safety and efficacy of ATS...
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria :
* male or female participant...
api
Leber Congenital Amaurosis, LCA, LCA1
Atsena Therapeutics Inc.
Active not recruiting
36 month safety and efficacy data presented in May 2026, Atsena plans to initiate pivotal Phase 3 trial in 2H26
NCT06594094
Show Changes
1785733200000
Last update post date
Previous:
2026-02-17
Current:
2026-08-03
curator
Estimated completion date
Previous:
2025-12-02
Current:
2027-06-02
curator
Overall status
Previous:
Completed
Current:
Active not recruiting
curator
Intervention description
Previous:
Once intravenous injection; the duration of the st...
Current:
Once intravenous injection; the duration of the st...
curator
Vector type
Previous:
AAV
Current:
Adeno-associated virus
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* males ≥ 4 and ≤8 years at t...
api
Duchenne Muscular Dystrophy (DMD)
HuidaGene Therapeutics Co., Ltd.
Active not recruiting
First patient dosed announced in December 2024
NCT07007065
Show Changes
1785733200000
Last update post date
Previous:
2026-07-29
Current:
2026-08-03
api
Number of locations
Previous:
129
Current:
130
api
Vector type
Previous:
AAV8
Current:
Adeno-associated virus
curator
Dose 4
Previous:
1.5E12 GC/eye (suprachoroidal delivery)
Current:
curator
Dose 3
Previous:
1.0E12 GC/eye
Current:
curator
Dose 2
Previous:
5.0E11 GC/eye
Current:
Phase 2 doses (suprachoroidal delivery): 2.5E11 GC...
curator
Dose 1
Previous:
2.5E11 GC/eye
Current:
Phase 1/2 doses: 3E9 GC/eye (n=5); 1E10 GC/eye (n=...
curator
Recent updates
Previous:
Another Phase 3 study (NCT05407636) is active, enr...
Current:
Another Phase 3 study (NCT05407636) is active, enr...
curator
Clinical publications
Previous:
null
Current:
LinkName:(Presentation) Subretinal Delivery of Inv...
curator
News and press releases
Previous:
null
Current:
LinkName:REGENXBIO Presents Positive Long-Term Dat...
curator
Editor type
Previous:
none
Current:
curator
Locations
Previous:
Puerto rico, Hungary, United states, United kingdo...
Current:
Puerto rico, Hungary, United states, Czechia, Unit...
api
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* pseudophakic (at least 12 w...
api
Neovascular Age-related Macular Degeneration
AbbVie
Recruiting
Another Phase 3 study (NCT05407636) is active, enrollment is completed; REGENXBIO expects to announce topline data with AbbVie from the ATMOSPHERE and ASCENT pivotal trials of sura-vec in wet AMD usin...
NCT06164730
Show Changes
1785733200000
Last update post date
Previous:
2026-07-13
Current:
2026-08-03
api
Number of locations
Previous:
24
Current:
27
api
Vector type
Previous:
LDLR + GalNAc
Current:
curator
Drug product type
Previous:
Encapsulated RNP
Current:
Encapsulated mRNA
curator
Recent updates
Previous:
Verve was acquired by Lilly in June 2025
Current:
Phase 1 study results published May 2026, Lilly pl...
curator
Dose 4
Previous:
Undisclosed dose 4
Current:
curator
Dose 3
Previous:
0.6mg/kg
Current:
curator
Dose 2
Previous:
0.45mg/kg
Current:
curator
Dose 1
Previous:
0.3mg/kg
Current:
Phase 1 trial: 0.3 mg/kg (n=4); 0.45 mg/kg (n=6); ...
curator
Editor type
Previous:
base editor
Current:
ABE8.8 mRNA
curator
News and press releases
Previous:
null
Current:
LinkName:A single dose of Lilly's PCSK9 base edito...
curator
Clinical publications
Previous:
null
Current:
LinkName:In Vivo Base Editing of PCSK9 with VERVE-...
curator
Delivery system
Previous:
Lipid encapsulation
Current:
Endocytosis
curator
Eligibility criteria
Previous:
null
Current:
Inclusion criteria:
* diagnosis of hefh or premat...
api
Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Verve Therapeutics, Inc.
Recruiting
Phase 1 study results published May 2026, Lilly plans to initiate Phase 2 study by the end of 2026
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.
Disclaimer
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.