Clinical Trials - Gene Therapy Trial Browser
The Gene Therapy Trial Browser represents a unique publicly accessible, free database for the benefit of users seeking information on gene therapy development. The information within integrates various sources, including clinicaltrials.gov, publications, sponsor press releases, patent applications, and more to give a comprehensive overview of the gene therapy clinical trial landscape.
Disclaimer: The information on this dashboard has been collected for the convenience of patients and researchers. The SCGE team are not medical doctors and cannot provide medical advice. Please discuss with your provider the risks/benefits of participating in a clinical trial, and do not send us your personal medical information. The information contained within this table does not make use of any confidential or privileged information-all data is collected from publicly available sources. The SCGE makes no comment as to the efficacy and safety of the items listed, as these are not known at the time of publication. For the most up to date information, or to inquire about enrollment, please refer to clinicaltrials.gov or the Sponsor's website for contact information.
Clinical Trials Daily Digest
Updates in the last 14 days
NCT05456880
2026-04-08
Protocol
null
LinkName:Clinical Trial Protocol;LinkUrl:https://w...
curator
Clinical publications
null
LinkName:Base Editing of HBG1 and HBG2 Promoters f...
curator
Recent updates
Beam expects to dose 30 patients by mid-2025, data...
clinical dose manufacturing is complete, BLA submi...
curator
Clinical publications
null
LinkName:(Poster) Robust HbF induction and improve...
curator
News and press releases
null
LinkName:Beam Therapeutics Reports Fourth Quarter ...
curator
Eligibility criteria
null
Key inclusion criteria include:
1. age ≥12 years ...
api
Sickle Cell Disease
Beam Therapeutics Inc.
Active not recruiting
Clinical dose manufacturing is complete, BLA submission expected as early as year-end 2026
NCT05073133
2026-04-08
Related nctid
LinkName:Phase 4: NCT05861986 (study subjects were...
LinkName:Phase 4: NCT05861986 (study subjects were...
curator
Related nctid
LinkName:Phase 4: NCT05861999 (study subjects were...
LinkName:Phase 4: NCT05861999 (study subjects were...
curator
Related nctid
null
LinkName:Phase 4: NCT04488133 (study subjects were...
curator
Related nctid
null
LinkName:Phase 3: NCT03505099;LinkUrl:https://www....
curator
Related nctid
LinkName:Phase 3: NCT03505099;LinkUrl:https://www....
null
curator
Related nctid
LinkName:Phase 4: NCT05089656;LinkUrl:https://www....
null
curator
Related nctid
LinkName:Long Term Follow-Up: NCT04174157;LinkUrl:...
LinkName:Patient Registry: NCT04174157;LinkUrl:htt...
curator
Related nctid
LinkName:Phase 1: NCT03381729;LinkUrl:https://www....
null
curator
Related nctid
null
LinkName:Phase 3: NCT03461289;LinkUrl:https://clin...
curator
Related nctid
null
LinkName:Long Term Follow-Up: NCT03421977;LinkUrl:...
curator
Related nctid
null
LinkName:Phase 3: NCT03306277;LinkUrl:https://clin...
curator
Related nctid
null
LinkName:Phase 3: NCT03837184;LinkUrl:https://clin...
curator
Related nctid
null
LinkName:Long Term Follow-Up: NCT04042025;LinkUrl:...
curator
Eligibility criteria
null
Inclusion criteria:
1. written informed consent/a...
api
Spinal Muscular Atrophy
Novartis Pharmaceuticals
Completed
FDA approved 5/24/19, price/treatment $2.1M
NCT05903794
2026-04-08
Last update post date
2025-04-17
2026-04-08
api
Estimated completion date
2026-02-28
2026-02-16
api
Overall status
Active not recruiting
Completed
api
Enrollment type
Estimated
Actual
api
Eligibility criteria
null
Inclusion criteria:
1. male or female, Age ≥ 50 y...
api
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Exegenesis Bio
Completed
FDA cleared IND 1/18/23
NCT03199469
2026-04-08
Last update post date
2026-01-14
2026-04-08
api
Preclinical publications
null
LinkName:AAV8 gene therapy and dietary insults tog...
curator
Development status
Active
Inactive
curator
Recent updates
On clinical hold since 2021
Astellas has restarted their XLMTM program by crea...
curator
Eligibility criteria
null
Inclusion criteria:
* subject has a diagnosis of ...
api
X-Linked Myotubular Myopathy
Astellas Gene Therapies
Active not recruiting
Astellas has restarted their XLMTM program by creating a new drug product ASP2957, which is based on this prior study, with substantial modifications ...
NCT05386680
2026-04-07
Related nctid
null
LinkName:Phase 4: NCT07448610;LinkUrl:https://clin...
curator
Related nctid
LinkName:Phase 3: NCT03505099;LinkUrl:https://clin...
null
curator
Eligibility criteria
null
Inclusion criteria
* sma diagnosis
* aged 2 to \<...
api
Record status
Draft
Active
curator
Recent updates
Same drug product as Zolgensma (dosing and route o...
curator
Protocol
null
LinkName:FDA Approval Documents;LinkUrl:https://ww...
curator
Protocol
null
LinkName:Clinical Trial Protocol;LinkUrl:https://c...
curator
Protocol
null
LinkName:Statistical Analysis Plan;LinkUrl:https:/...
curator
Clinical publications
null
LinkName:Intrathecal onasemnogene abeparvovec for ...
curator
Clinical publications
null
LinkName:Intrathecal onasemnogene abeparvovec in t...
curator
Clinical publications
null
LinkName:Intrathecal Onasemnogene Abeparvovec for ...
curator
Preclinical publications
null
LinkName:Single-Dose Intrathecal Dorsal Root Gangl...
curator
Indication doid
null
DOID:13137
curator
Development status
null
Approved
curator
Compound name
null
ITVISMA
curator
Dose 3
null
Approved Dose: 1.2E14 vg
curator
Dose 2
null
Phase 3: 1.2E14 vg
curator
Dose 1
null
Phase 1/2: 6.0E13 vg, 1.2E14 vg, 2.4E14 vg
curator
Vector type
null
AAV9
curator
Delivery system
null
viral transduction
curator
Drug product type
null
viral vector
curator
Route of administration
null
intrathecal
curator
Mechanism of action
null
functional gene replacement
curator
Therapy route
null
in-vivo
curator
Therapy type
null
gene transfer
curator
Target gene
null
SMN1
curator
Alias type
null
proper name, proprietary names
curator
Alias value
null
onasemnogene abeparvovec, OAV101, AVXS-101
curator
Fda designation
Breakthrough Therapy, Orphan Drug Designation
curator
Related nctid
null
LinkName:Patient Registry: NCT04174157;LinkUrl:htt...
curator
Related nctid
null
LinkName:Long Term Follow Up: NCT05335876;LinkUrl:...
curator
Related nctid
null
LinkName:Phase 3: NCT05089656;LinkUrl:https://clin...
curator
Related nctid
null
LinkName:Phase 1: NCT03381729;LinkUrl:https://clin...
curator
Spinal Muscular Atrophy
Novartis Pharmaceuticals
Completed
Same drug product as Zolgensma (dosing and route of administration differs), FDA approved 11/24/25 for list price $2.59M
All Recent Updates (55)
NCT05456880
Show Changes
2026-04-08
Protocol
null
LinkName:Clinical Trial Protocol;LinkUrl:https://w...
curator
Clinical publications
null
LinkName:Base Editing of HBG1 and HBG2 Promoters f...
curator
Recent updates
Beam expects to dose 30 patients by mid-2025, data...
clinical dose manufacturing is complete, BLA submi...
curator
Clinical publications
null
LinkName:(Poster) Robust HbF induction and improve...
curator
News and press releases
null
LinkName:Beam Therapeutics Reports Fourth Quarter ...
curator
Eligibility criteria
null
Key inclusion criteria include:
1. age ≥12 years ...
api
Sickle Cell Disease
Beam Therapeutics Inc.
Active not recruiting
Clinical dose manufacturing is complete, BLA submission expected as early as year-end 2026
NCT05073133
Show Changes
2026-04-08
Related nctid
LinkName:Phase 4: NCT05861986 (study subjects were...
LinkName:Phase 4: NCT05861986 (study subjects were...
curator
Related nctid
LinkName:Phase 4: NCT05861999 (study subjects were...
LinkName:Phase 4: NCT05861999 (study subjects were...
curator
Related nctid
null
LinkName:Phase 4: NCT04488133 (study subjects were...
curator
Related nctid
null
LinkName:Phase 3: NCT03505099;LinkUrl:https://www....
curator
Related nctid
LinkName:Phase 3: NCT03505099;LinkUrl:https://www....
null
curator
Related nctid
LinkName:Phase 4: NCT05089656;LinkUrl:https://www....
null
curator
Related nctid
LinkName:Long Term Follow-Up: NCT04174157;LinkUrl:...
LinkName:Patient Registry: NCT04174157;LinkUrl:htt...
curator
Related nctid
LinkName:Phase 1: NCT03381729;LinkUrl:https://www....
null
curator
Related nctid
null
LinkName:Phase 3: NCT03461289;LinkUrl:https://clin...
curator
Related nctid
null
LinkName:Long Term Follow-Up: NCT03421977;LinkUrl:...
curator
Related nctid
null
LinkName:Phase 3: NCT03306277;LinkUrl:https://clin...
curator
Related nctid
null
LinkName:Phase 3: NCT03837184;LinkUrl:https://clin...
curator
Related nctid
null
LinkName:Long Term Follow-Up: NCT04042025;LinkUrl:...
curator
Eligibility criteria
null
Inclusion criteria:
1. written informed consent/a...
api
Spinal Muscular Atrophy
Novartis Pharmaceuticals
Completed
FDA approved 5/24/19, price/treatment $2.1M
NCT05903794
Show Changes
2026-04-08
Last update post date
2025-04-17
2026-04-08
api
Estimated completion date
2026-02-28
2026-02-16
api
Overall status
Active not recruiting
Completed
api
Enrollment type
Estimated
Actual
api
Eligibility criteria
null
Inclusion criteria:
1. male or female, Age ≥ 50 y...
api
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Exegenesis Bio
Completed
FDA cleared IND 1/18/23
NCT03199469
Show Changes
2026-04-08
Last update post date
2026-01-14
2026-04-08
api
Preclinical publications
null
LinkName:AAV8 gene therapy and dietary insults tog...
curator
Development status
Active
Inactive
curator
Recent updates
On clinical hold since 2021
Astellas has restarted their XLMTM program by crea...
curator
Eligibility criteria
null
Inclusion criteria:
* subject has a diagnosis of ...
api
X-Linked Myotubular Myopathy
Astellas Gene Therapies
Active not recruiting
Astellas has restarted their XLMTM program by creating a new drug product ASP2957, which is based on this prior study, with substantial modifications to the capsid to detarget the liver
NCT05386680
Show Changes
2026-04-07
Related nctid
null
LinkName:Phase 4: NCT07448610;LinkUrl:https://clin...
curator
Related nctid
LinkName:Phase 3: NCT03505099;LinkUrl:https://clin...
null
curator
Eligibility criteria
null
Inclusion criteria
* sma diagnosis
* aged 2 to \<...
api
Record status
Draft
Active
curator
Recent updates
Same drug product as Zolgensma (dosing and route o...
curator
Protocol
null
LinkName:FDA Approval Documents;LinkUrl:https://ww...
curator
Protocol
null
LinkName:Clinical Trial Protocol;LinkUrl:https://c...
curator
Protocol
null
LinkName:Statistical Analysis Plan;LinkUrl:https:/...
curator
Clinical publications
null
LinkName:Intrathecal onasemnogene abeparvovec for ...
curator
Clinical publications
null
LinkName:Intrathecal onasemnogene abeparvovec in t...
curator
Clinical publications
null
LinkName:Intrathecal Onasemnogene Abeparvovec for ...
curator
Preclinical publications
null
LinkName:Single-Dose Intrathecal Dorsal Root Gangl...
curator
Indication doid
null
DOID:13137
curator
Development status
null
Approved
curator
Compound name
null
ITVISMA
curator
Dose 3
null
Approved Dose: 1.2E14 vg
curator
Dose 2
null
Phase 3: 1.2E14 vg
curator
Dose 1
null
Phase 1/2: 6.0E13 vg, 1.2E14 vg, 2.4E14 vg
curator
Vector type
null
AAV9
curator
Delivery system
null
viral transduction
curator
Drug product type
null
viral vector
curator
Route of administration
null
intrathecal
curator
Mechanism of action
null
functional gene replacement
curator
Therapy route
null
in-vivo
curator
Therapy type
null
gene transfer
curator
Target gene
null
SMN1
curator
Alias type
null
proper name, proprietary names
curator
Alias value
null
onasemnogene abeparvovec, OAV101, AVXS-101
curator
Fda designation
Breakthrough Therapy, Orphan Drug Designation
curator
Related nctid
null
LinkName:Patient Registry: NCT04174157;LinkUrl:htt...
curator
Related nctid
null
LinkName:Long Term Follow Up: NCT05335876;LinkUrl:...
curator
Related nctid
null
LinkName:Phase 3: NCT05089656;LinkUrl:https://clin...
curator
Related nctid
null
LinkName:Phase 1: NCT03381729;LinkUrl:https://clin...
curator
Spinal Muscular Atrophy
Novartis Pharmaceuticals
Completed
Same drug product as Zolgensma (dosing and route of administration differs), FDA approved 11/24/25 for list price $2.59M
NCT04850118
Show Changes
2026-04-07
Last update post date
2025-07-16
2026-04-07
api
Estimated completion date
2029-10
2031-10-31
api
Number of locations
24
25
api
Locations
United states, United kingdom, Australia
Canada, United states, United kingdom, Australia
api
Related nctid
null
LinkName:Phase 2: NCT07174726;LinkUrl:https://clin...
curator
Eligibility criteria
null
General inclusion criteria:
1. provide written in...
api
X-Linked Retinitis Pigmentosa (XLRP)
Beacon Therapeutics
Active not recruiting
First patient dosed in registrational trial 6/12/24, enrollment ongoing
NCT07052929
Show Changes
2026-04-07
Record status
Draft
Active
curator
Target tissue
muscle cells
curator
Recent updates
Astellas has restarted their XLMTM program by crea...
Astellas has restarted their XLMTM program by crea...
curator
Dose 2
2E12 vg/kg
curator
Dose 1
1E12 vg/kg
curator
Related nctid
null
LinkName:Phase 2/3: NCT03199469 (prior drug produc...
curator
Related nctid
null
LinkName:Epidemiological study: NCT06581146;LinkUr...
curator
News and press releases
null
LinkName:Astellas Takes Aim at XLMTM Again, Now wi...
curator
News and press releases
null
LinkName:VALOR Clinical Trial Patient Information;...
curator
Protocol
null
LinkName:(Abstract) VALOR study design: a phase 1/...
curator
Protocol
null
LinkName:(Abstract 74P) Design of a phase 1/2 stud...
curator
Preclinical publications
null
LinkName:(Abstract) Efficacy, Biodistribution, and...
curator
Compound description
MyoAAV3.8-MHCK7-hMTM1
curator
Indication doid
DOID:0111225
curator
Development status
Active
curator
Compound name
ASP2957
curator
Vector type
AAV3.8
curator
Delivery system
viral transduction
curator
Drug product type
viral vector
curator
Route of administration
intravenous
curator
Mechanism of action
functional gene replacement
curator
Therapy route
in-vivo
curator
Therapy type
gene transfer
curator
Target gene
MTM1
curator
Alias type
null
proprietary name
curator
Alias value
null
KT430
curator
Clinical publications
null
LinkName:Intrahepatic Cholestasis Is a Clinically ...
curator
Clinical publications
null
LinkName:Hepatobiliary disease in XLMTM: a common ...
curator
News and press releases
null
LinkName:Astellas Sees Path Forward for Gene Thera...
curator
Eligibility criteria
null
Inclusion criteria:
* participant is projected to...
api
Last update post date
2026-01-08
2026-03-13
api
Number of locations
2
3
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...
NCT03566043
Show Changes
2026-04-07
Related nctid
null
LinkName:Phase 3: NCT07236606;LinkUrl:https://clin...
curator
Recent updates
BLA Submitted, PDUFA date extended from 11/9/25 to...
FDA placed this study on clinical hold in January ...
curator
News and press releases
null
LinkName:REGENXBIO Announces Regulatory Update on ...
curator
News and press releases
null
LinkName:REGENXBIO Announces Regulatory Update on ...
curator
Eligibility criteria
null
Part 1 inclusion criteria:
* the subject's legal ...
api
Mucopolysaccharidosis Type II (Hunter Syndrome)
REGENXBIO Inc.
Active not recruiting
FDA placed this study on clinical hold in January 2026 following the incidence of a CNS tumor in a participant in another clinical trial for a different MPS Syndrome
CRL issued 2/7/26 (issues with st...
NCT05606614
Show Changes
2026-04-07
Related nctid
null
LinkName:Phase 3: NCT07480564 (Pediatric cohort);L...
curator
Recent updates
Clinical data expected H1 2025
First patient dosed in REVEAL pivotal trial (adole...
curator
Fda designation
Fast Track, Orphan Drug Designation, Rare Pediatri...
Breakthrough Therapy, Fast Track, Orphan Drug Desi...
curator
News and press releases
null
LinkName:Taysha Gene Therapies Announces Progress ...
curator
News and press releases
null
LinkName:SEC Form 10-K: Taysha Gene Therapies, Inc...
curator
News and press releases
null
LinkName:Taysha Gene Therapies Regains Full Rights...
curator
Record status
Inactive
Active
curator
Eligibility criteria
null
Inclusion criteria:
* females between the ages of...
api
Rett Syndrome
Taysha Gene Therapies, Inc.
Recruiting
First patient dosed in REVEAL pivotal trial (adolescent cohort), expect to complete dosing 2Q26
NCT05881408
Show Changes
2026-04-07
Recent updates
First approval was June 2023, expanded indication ...
First approval was June 2023, expanded indication ...
curator
News and press releases
null
LinkName:Sarepta Announces that Screening and Enro...
curator
Related nctid
null
LinkName:Phase 4: NCT06270719;LinkUrl:https://clin...
curator
Related nctid
null
LinkName:Phase 2: NCT06128564;LinkUrl:https://clin...
curator
Eligibility criteria
null
Inclusion criteria:
* definitive diagnosis of dmd...
api
Clinical publications
null
LinkName:Two-Year Outcomes Following Delandistroge...
curator
Duchenne Muscular Dystrophy (DMD)
Sarepta Therapeutics, Inc.
Active not recruiting
First approval was June 2023, expanded indication to all DMD patients regardless of ambulatory status or age on 6/20/24; new black box warning on risk of liver injury and liver failure added November ...
NCT07048808
Show Changes
2026-04-07
Indication doid
null
DOID:114; DOID:3393
curator
Development status
null
Active
curator
Record status
Provisional
Active
curator
Compound name
null
XC001
curator
Recent updates
null
First patient dosed in Phase 2b trial in July 2025
curator
Dose 4
null
1E11 vp (Expansion Dose)
curator
Dose 3
null
4E10 vp
curator
Dose 2
null
1E10 vp
curator
Dose 1
null
1E9 vp
curator
Editor type
null
none
curator
Vector type
null
Ad5
curator
Delivery system
null
Viral transduction
curator
Drug product type
null
Viral vector
curator
Route of administration
null
Intramyocardial
curator
Mechanism of action
null
Overexpression of protective allele/gene
curator
Therapy route
null
In-vivo
curator
Therapy type
null
Gene transfer
curator
Target gene
null
VEGF
curator
Alias type
null
proper name
curator
Alias value
null
Encoberminogene rezmadenovec
curator
Related nctid
null
LinkName:Phase 2: NCT07118449;LinkUrl:https://clin...
curator
Related nctid
null
LinkName:Phase 1/2: NCT04125732;LinkUrl:https://cl...
curator
Protocol
null
LinkName:Epicardial delivery of XC001 gene therapy...
curator
Preclinical publications
null
LinkName:Safety of direct cardiac administration o...
curator
Preclinical publications
null
LinkName:Alteration of splicing signals in a genom...
curator
Preclinical publications
null
LinkName:Adenovirus-mediated transfer of a minigen...
curator
Preclinical publications
null
LinkName:Safety and biodistribution of XC001 (enco...
curator
News and press releases
null
LinkName:https://www.xylocor.com/news.html;LinkUrl...
curator
News and press releases
null
LinkName:XyloCor doses first subject in Phase IIb ...
curator
Clinical publications
null
LinkName:EXACT Trial: Results of the Phase 1 Dose-...
curator
Clinical publications
null
LinkName:Angiogenic Gene Therapy for Refractory An...
curator
Eligibility criteria
null
Inclusion criteria:
1. males and females, Age 18 ...
api
Refractory Angina, Coronary Artery Disease
XyloCor Therapeutics, Inc.
Recruiting
First patient dosed in Phase 2b trial in July 2025
NCT05835895
Show Changes
2026-04-07
Recent updates
Initial data for Phase 1b trial expected Q4 2024
the last patient exited the study in March 2025, a...
curator
Preclinical publications
null
LinkName:Intra-articular gene delivery and express...
curator
Preclinical publications
null
LinkName:Safety and biodistribution assessment of ...
curator
Clinical publications
null
LinkName:(Review) Osteoarthritis Gene Therapy: A 3...
curator
Related nctid
null
LinkName:Phase 1: NCT02790723;LinkUrl:https://clin...
curator
Eligibility criteria
null
Key inclusion criteria:
* oa in at least one knee...
api
Osteoarthritis, Knee
Genascence Corporation
Active not recruiting
The last patient exited the study in March 2025, and data are being analyzed
NCT05432310
Show Changes
2026-04-07
Last update post date
2025-04-03
2026-04-07
api
Estimated completion date
2026-12-31
2027-12-31
api
Eligibility criteria
null
Inclusion criteria:
all subjects must fulfill the...
api
Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID)
University of California, Los Angeles
Recruiting
Orchard terminated this program, returned the program to UCLA which administers the therapy under Compassionate Use
NCT07226206
Show Changes
2026-04-07
Compound description
AAV LK03 capsid + TTRmut-hFVIII-X07
AAV LK03 capsid + TTRmut + APC-resistant FVIII var...
curator
Preclinical publications
null
LinkName:Activated protein C has a regulatory role...
curator
Indication doid
null
DOID:12134
curator
Development status
null
Active
curator
Record status
Provisional
Active
curator
Compound name
null
SPK-8011QQ
curator
Recent updates
null
Study was withdrawn by Sponsor (no participants we...
curator
Dose 4
null
2E12 vg/kg
curator
Dose 3
null
1.5E12 vg/kg
curator
Dose 2
null
1E12 vg/kg
curator
Dose 1
null
5E11 vg/kg
curator
Editor type
null
none
curator
Vector type
null
LK03
curator
Delivery system
null
Viral transduction
curator
Drug product type
null
Viral vector
curator
Route of administration
null
Intravenous
curator
Mechanism of action
null
Functional gene replacement
curator
Therapy route
null
In-vivo
curator
Therapy type
null
Gene transfer
curator
Target gene
null
F8
curator
Alias type
null
proper name
curator
Alias value
null
Dirloctogene samoparvovec
curator
Related nctid
null
LinkName:Phase 3: NCT06297486 (Withdrawn);LinkUrl:...
curator
Related nctid
null
LinkName:Phase 1/2: NCT03003533;LinkUrl:https://ww...
curator
Related nctid
null
LinkName:Long Term Follow-Up: NCT03432520;LinkUrl:...
curator
Preclinical publications
null
LinkName:Preclinical assessment of an optimized AA...
curator
Preclinical publications
null
LinkName:(Abstract) Preclinical evaluation of SPK-...
curator
News and press releases
null
LinkName:Roche mothballs another hemophilia A gene...
curator
News and press releases
null
LinkName:Integration of Spark into Roche Group Set...
curator
Clinical publications
null
LinkName:Multiyear Factor VIII Expression after AA...
curator
Last update post date
2026-03-12
2026-03-31
api
Overall status
Recruiting
Not yet recruiting
api
Eligibility criteria
null
Inclusion criteria:
* signed informed consent for...
api
Number of locations
0
1
api
Locations
United states
api
Hemophilia A
Hoffmann-La Roche
Not yet recruiting
Study was withdrawn by Sponsor (no participants were enrolled), Sponsor is extensively modifying the product before clinical trials resume
NCT06884865
Show Changes
2026-04-07
Compound description
null
AdV-NFKB-IL1RA
curator
Indication doid
null
DOID:8398
curator
Development status
null
Active
curator
Record status
Provisional
Active
curator
Compound name
null
PCRX-201
curator
Recent updates
null
Pacira announced Phase 2 enrollment was complete i...
curator
Dose 3
null
1.4E12 gc/knee
curator
Dose 2
null
1.4E11 gc/knee
curator
Dose 1
null
1.4E10 gc/knee
curator
Editor type
null
none
curator
Vector type
null
Ad5
curator
Delivery system
null
Viral transduction
curator
Drug product type
null
Viral vector
curator
Route of administration
null
Intraarticular
curator
Mechanism of action
null
Overexpression of protective allele/gene
curator
Therapy route
null
In-vivo
curator
Therapy type
null
gene transfer
curator
Target gene
null
IL1RA
curator
Alias type
null
proper name, proprietary name
curator
Alias value
null
Enekinragene inzadenovec, FX-201
curator
Fda designation
Regenerative Medicine Advanced Therapy
curator
Preclinical publications
null
LinkName:PCRX-201, a novel IL-1Ra gene therapy tre...
curator
Preclinical publications
null
LinkName:Efficacy and Safety of FX201, a Novel Int...
curator
News and press releases
null
LinkName:Pacira BioSciences Announces PCRX-201 Gra...
curator
News and press releases
null
LinkName:(Corporate Presentation) An Introduction ...
curator
News and press releases
null
LinkName:Pacira BioSciences Concludes Patient Enro...
curator
Clinical publications
null
LinkName:(Abstract POS0492) A SINGLE INTRA-ARTICUL...
curator
Clinical publications
null
LinkName:(Abstract #594) Interim Data from the Fir...
curator
Last update post date
2025-06-26
2026-03-24
api
Estimated completion date
2032-03
2032-07
api
Overall status
Recruiting
Active not recruiting
api
Eligibility criteria
null
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...
api
Osteoarthritis (OA) of the Knee
Pacira Pharmaceuticals, Inc
Active not recruiting
Pacira announced Phase 2 enrollment was complete in November 2025
NCT06325709
Show Changes
2026-04-06
Last update post date
2026-04-01
2026-04-06
api
Description
Background:
chronic granulomatous disease (cgd) i...
Background:
chronic granulomatous disease (cgd) i...
api
Eligibility criteria
null
* inclusion criteria:
-\>= 18 years of age.
* c...
api
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
NCT05739643
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow-Up: NCT06308718;LinkUrl:...
curator
Eligibility criteria
null
Inclusion criteria:
1. group infantile krabbe: su...
api
Krabbe Disease
Forge Biologics, Inc
Active not recruiting
Program discontinuation was announced January 2025
NCT06388200
Show Changes
2026-04-06
Related nctid
null
LinkName:Phase 1/2: NCT05203939;LinkUrl:https://cl...
curator
News and press releases
LinkType:Protocol;LinkName:SEC Form 10-Q: Ocugen, ...
LinkType:News and Press Releases;LinkName:SEC Form...
curator
News and press releases
LinkName:Ocugen Announces Positive Opinion of EMAâ...
LinkName:Ocugen Announces Positive Opinion of EMA'...
curator
Recent updates
Potential BLA/MAA filings by mid-2026
completed enrollment in Phase 3 trial, on track to...
curator
News and press releases
null
LinkName:Ocugen Provides Business Update with Four...
curator
Eligibility criteria
null
Inclusion criteria:
1. males or females ≥ 3 years...
api
Retinitis Pigmentosa
Ocugen
Active not recruiting
Completed enrollment in Phase 3 trial, on track to file rolling BLA in 3Q2026
NCT05419492
Show Changes
2026-04-06
Last update post date
2026-04-02
2026-04-06
api
Number of locations
8
9
api
Eligibility criteria
null
Inclusion criteria:
* participant must be aged be...
api
Recent updates
Enrollment in dose escalation studies expected to ...
received Breakthrough Designation from FDA in Janu...
curator
Fda designation
Fast Track, Orphan Drug Designation, Rare Pediatri...
Breakthrough Therapy, Fast Track, Orphan Drug Desi...
curator
Dravet Syndrome
Encoded Therapeutics
Recruiting
Received Breakthrough Designation from FDA in January 2026
NCT05345171
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow-Up: NCT03636438;LinkUrl:...
curator
Recent updates
Enrollment of Phase 3 study complete in February 2...
Interim results announced March 2026, data are exp...
curator
Dose 4
Phase 3: 1.0E13 GC/kg
Phase 3: 1.7E13 GC/kg
curator
News and press releases
null
LinkName:Ultragenyx Announces Positive 36-Week Dat...
curator
Eligibility criteria
null
Key inclusion criteria:
* confirmed clinical diag...
api
Ornithine Transcarbamylase (OTC) Deficiency
Ultragenyx Pharmaceutical Inc
Active not recruiting
Interim results announced March 2026, data are expected first half of 2027
NCT05139316
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow-Up: NCT03970278;LinkUrl:...
curator
Last update post date
2026-03-11
2026-03-25
api
Estimated completion date
2026-02-05
2026-02-20
api
Eligibility criteria
null
Key inclusion criteria:
* documented gsdia with c...
api
Recent updates
BLA Submission completed 12/30/25
BLA Submission completed 12/30/25, PDUFA Action Da...
curator
Fda designation
Fast Track, Orphan Drug Designation, Rare Pediatri...
Fast Track, Orphan Drug Designation, Priority Revi...
curator
Clinical publications
null
LinkName:Trial Interviews to Explore Glycogen Stor...
curator
News and press releases
null
LinkName:Ultragenyx Reports Fourth Quarter and Ful...
curator
News and press releases
null
LinkName:Ultragenyx Announces U.S. FDA Acceptance ...
curator
Overall status
Active not recruiting
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
NCT02651675
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow-Up: NCT04080050;LinkUrl:...
curator
Eligibility criteria
null
Inclusion criteria:
* male or female ≥ 18 years o...
api
Homozygous Familial Hypercholesterolemia (HoFH)
REGENXBIO Inc.
Terminated
Terminated by Sponsor for business reasons
NCT04293185
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow-Up: NCT04628585;LinkUrl:...
curator
Eligibility criteria
null
Inclusion criteria:
* have a diagnosis of scd, Wi...
api
Sickle Cell Disease
Genetix Biotherapeutics Inc.
Active not recruiting
FDA approved 12/8/23, price/treatment $3.1M
NCT07007065
Show Changes
2026-04-06
Last update post date
2026-04-03
2026-04-06
api
Number of locations
95
96
api
Locations
Puerto rico, Hungary, United states, United kingdo...
Puerto rico, Hungary, United states, Czechia, Unit...
api
Eligibility criteria
null
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
NCT04680065
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow Up: NCT07081841;LinkUrl:...
curator
Eligibility criteria
null
Inclusion criteria:
* male and female 35-75 years...
api
Multiple System Atrophy
Brain Neurotherapy Bio, Inc.
Active not recruiting
Phase 1 enrollment completed September 2025
NCT04671433
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow-Up: NCT04671433;LinkUrl:...
curator
Last update post date
2025-09-29
2026-03-16
api
Eligibility criteria
null
Inclusion criteria:
* male or female
* 3 years of...
api
Number of locations
27
28
api
Enrollment count
97
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
NCT05956626
Show Changes
2026-04-06
Last update post date
2025-10-16
2026-04-06
api
Overall status
Recruiting
Active not recruiting
api
Eligibility criteria
Phase 2/3 inclusion criteria (applicable for both ...
Phase 2/3 inclusion criteria (applicable for both ...
api
Description
Phase 2/3 pivotal confirmatory clinical trial is a...
Phase 2/3 pivotal confirmatory clinical trial is a...
api
Recent updates
First patient dosed in Phase 2/3 pivotal trial, BL...
Phase 2/3 pivotal confirmatory trial nearing enrol...
curator
News and press releases
null
LinkName:Ocugen Provides Business Update with Four...
curator
Clinical publications
null
LinkName:A novel modifier gene therapy to treat St...
curator
Stargardt Disease
Ocugen
Active not recruiting
Phase 2/3 pivotal confirmatory trial nearing enrollment completion. Interim data expected in the third quarter 2026, followed by topline Phase 2/3 data in the second quarter 2027 in advance of the BLA...
NCT06517888
Show Changes
2026-04-06
Last update post date
2026-03-31
2026-04-06
api
Eligibility criteria
null
Criteria for inclusion:
1. presence of unilateral...
api
Vestibular Schwannoma
Akouos, Inc.
Recruiting
Eli Lilly acquired this company in 2022
NCT06063850
Show Changes
2026-04-06
Last update post date
2025-12-10
2026-04-06
api
Eligibility criteria
null
Inclusion criteria:
* diagnosis of unilateral ref...
api
Mesial Temporal Lobe Epilepsy
UniQure Biopharma B.V.
Recruiting
First 3 patients in first cohort enrolled September 2025
NCT06856577
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow Up: NCT04645212;LinkUrl:...
curator
Related nctid
null
LinkName:Phase 3: NCT07482176;LinkUrl:https://clin...
curator
Related nctid
LinkName:Phase 2: NCT04418427 (for Diabetic Macula...
null
curator
Related nctid
LinkName: Phase 1: NCT03748784;LinkUrl:https://www...
LinkName:Phase 1: NCT03748784;LinkUrl:https://www....
curator
Eligibility criteria
null
Inclusion criteria:
1. able and willing to provid...
api
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Adverum Biotechnologies, Inc.
Recruiting
Screening completed September 2025, company acquired by Lilly in October 2025
NCT05725018
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow-Up: NCT05708677;LinkUrl:...
curator
Eligibility criteria
null
Inclusion criteria:
1. clinical diagnosis of deb....
api
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Abeona Therapeutics, Inc
Active not recruiting
FDA approved on 4/29/25, list price $3.1M USD
NCT06559176
Show Changes
2026-04-06
Last update post date
2025-05-25
2026-04-06
api
Overall status
Active not recruiting
Enrolling by invitation
api
Eligibility criteria
null
Inclusion criteria:
* autosomal recessive chronic...
api
Development status
Inactive
Active
curator
Recent updates
Prime is shelving this program due to market consi...
Prime intends to submit BLA on the basis of the 2 ...
curator
News and press releases
null
LinkName:Prime Medicine Reports Full Year 2025 Fin...
curator
Chronic Granulomatous Disease
Prime Medicine, Inc.
Enrolling by invitation
Prime intends to submit BLA on the basis of the 2 patients treated, working on alignment with FDA
NCT03580083
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow-Up: NCT06103487;LinkUrl:...
curator
Recent updates
REGENXBIO is partnering with Nippon Shinyaku to co...
FDA placed clinical hold in January 2026 following...
curator
News and press releases
null
LinkName:REGENXBIO Announces Regulatory Update on ...
curator
Eligibility criteria
null
Inclusion criteria:
1. a male or female ≥ 4 month...
api
Last update post date
2023-12-06
2026-03-12
api
Estimated completion date
2024-10
2029-03-06
api
Overall status
Unknown
Suspended
api
Official title
A phase i/ii multicenter, Open-label study to eval...
A phase i/ii multicenter, Open-label study to eval...
api
Number of locations
4
2
api
Locations
United states, Brazil, Israel
United states, Brazil
api
Enrollment type
Actual
Estimated
api
Enrollment count
8
21
api
Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
REGENXBIO Inc.
Suspended
FDA placed clinical hold in January 2026 following preliminary analysis of a CNS tumor in a five-year-old patient treated in this study four years ago
NCT06680232
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow-Up: NCT07254208;LinkUrl:...
curator
Eligibility criteria
null
Key inclusion criteria:
* male or women of non-ch...
api
Recent updates
Cohort 3 dosing started, additional data readouts ...
Cohort 5 dosing started at 0.65mg/kg, additional d...
curator
Dose 4
0.65mg/kg
curator
News and press releases
null
LinkName:Precision BioSciences Sets Strategic Prio...
curator
Chronic Hepatitis B
Precision BioSciences, Inc.
Recruiting
Cohort 5 dosing started at 0.65mg/kg, additional data readouts planned in early 2026
NCT04145037
Show Changes
2026-04-06
Related nctid
null
LinkName:Phase 2/3: NCT05815004 (Gaucher's Disease...
curator
Related nctid
null
LinkName:Long Term Follow-Up: NCT04836377;LinkUrl:...
curator
Eligibility criteria
null
Inclusion criteria for all enrolled (switch-stable...
api
Gaucher Disease Type 1
AVROBIO
Terminated
Program development was halted in July 2023
NCT04046224
Show Changes
2026-04-06
Related nctid
null
LinkName:Long Term Follow-Up: NCT05039866;LinkUrl:...
curator
Eligibility criteria
null
Inclusion criteria:
* ≥ 18 years of age
* documen...
api
Recent updates
Recent Type B meeting with FDA allowed ongoing Pha...
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
NCT03861273
Show Changes
2026-04-03
Last update post date
2026-03-16
2026-04-03
api
Eligibility criteria
null
Inclusion criteria
* males who completed 6 months...
api
Number of locations
60
65
api
Hemophilia B
Pfizer
Active not recruiting
FDA approved 4/25/24, Price/treatment $3.5M; Pfizer will no longer market Beqvez
NCT03496012
Show Changes
2026-04-03
Related nctid
null
LinkName:Phase 2: NCT03507686;LinkUrl:https://clin...
curator
Eligibility criteria
null
Key inclusion criteria:
* are willing and able to...
api
Choroideremia
Biogen
Completed
Phase III study did not meet its primary endpoint or demonstrate efficacy on key secondary endpoints
NCT06432140
Show Changes
2026-04-03
Last update post date
2024-06-03
2026-04-03
curator
Estimated completion date
2030-09
2030-09-20
curator
Overall status
Not yet recruiting
Active not recruiting
curator
Number of locations
0
1
curator
Locations
China
curator
Enrollment type
Estimated
Actual
curator
Enrollment count
16
13
curator
Phases
Phase1
Phase3
curator
Eligibility criteria
null
Inclusion criteria:
1. the child patient has to b...
api
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Shanghai Vitalgen BioPharma Co., Ltd.
Active not recruiting
FDA IND accepted 7/26/24
NCT02652767
Show Changes
2026-04-03
Related nctid
null
LinkName:Phase 3: NCT03293524;LinkUrl:https://clin...
curator
Related nctid
null
LinkName:Phase 3: NCT02652780;LinkUrl:https://clin...
curator
Eligibility criteria
null
Inclusion criteria:
selection criteria:
particip...
api
Leber Hereditary Optic Neuropathy (LHON)
GenSight Biologics
Completed
Granted compassionate use authorization for named patient in France, early access use authorized in Israel; MAA in UK goal in 2026
NCT00999609
Show Changes
2026-04-03
Related nctid
null
LinkName:Long Term Follow-Up: NCT03602820;LinkUrl:...
curator
Related nctid
null
LinkName:Long Term Follow-Up: NCT03597399;LinkUrl:...
curator
Eligibility criteria
null
Inclusion criteria:
* willingness to adhere to pr...
api
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Spark Therapeutics, Inc.
Active not recruiting
FDA approved 12/19/17, Price/treatment $850K/eye
NCT00979238
Show Changes
2026-04-02
Last update post date
2026-02-04
2026-04-02
api
Estimated completion date
2026-03
2026-03-26
api
Overall status
Active not recruiting
Completed
api
Number of locations
8
4
api
Eligibility criteria
null
1) or
* received an aav vector or any other gene transfer agent in the previous 6 months
* presence of lung nodule(s) suspicious of malignancy on screening chest tomography
* presence of liver abnormalities suspicious of malignancy on screening liver ultrasound">Inclusion criteria:
* males ≥ 18 years of age wit...
api
Hemophilia B
St. Jude Children's Research Hospital
Completed
Was the first Hemophilia B trial to show sustained efficacy
NCT06458595
Show Changes
2026-04-01
Recent updates
First patient dosed in May 2025 at a US site (tria...
curator
Related nctid
LinkName:Phase 1: NCT06825858;LinkUrl:https://www....
LinkName:Phase 1: NCT06825858 (US sites);LinkUrl:h...
curator
News and press releases
null
LinkName:Chengdu Origen and Vanotech Announce Firs...
curator
Eligibility criteria
null
Inclusion criteria:
1. are willing and able to si...
api
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Chengdu Origen Biotechnology Co., Ltd.
Recruiting
First patient dosed in May 2025 at a US site (trial ID: NCT06825858)
NCT06662188
Show Changes
2026-04-01
Recent updates
Clinical sites are now open
Cohort 1 (3 patients) dosing is complete, first tw...
curator
News and press releases
null
LinkName:Completion of Dosing of First Patient Coh...
curator
Eligibility criteria
null
Key inclusion criteria:
1. is male or female, And...
api
SHANK3 Haploinsufficiency, Phelan-McDermid Syndrome
Jaguar Gene Therapy, LLC
Recruiting
Cohort 1 (3 patients) dosing is complete, first two patients in dose-escalation cohort 2 have been dosed, completion expected in Q2 2026
NCT05203679
Show Changes
2026-04-01
Last update post date
2025-07-01
2026-04-01
curator
Eligibility criteria
null
Inclusion criteria of phase 1/2/3:
1. males ≥ 18 ...
api
Hemophilia B
Shanghai Xinzhi BioMed Co., Ltd.
Active not recruiting
In April 2025, Product was approved by National Medical Products Administration (NMPA) in China
NCT04611503
Show Changes
2026-04-01
Development status
Active
Inactive
curator
Recent updates
no functional improvements seen with this product
curator
Clinical publications
null
LinkName:Safety and vision outcomes of subretinal ...
curator
Eligibility criteria
null
Inclusion criteria:
* clinical diagnosis of retin...
api
Retinitis Pigmentosa
STZ eyetrial
Active not recruiting
No functional improvements seen with this product
NCT06164730
Show Changes
2026-04-01
Last update post date
2026-03-25
2026-04-01
api
Number of locations
21
22
api
Eligibility criteria
null
Inclusion criteria:
* diagnosis of hefh or premat...
api
Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Verve Therapeutics, Inc.
Recruiting
Verve was acquired by Lilly in June 2025
NCT02716246
Show Changes
2026-04-01
Recent updates
BLA filed under Accelerated Approval, CRL issued 7...
BLA resubmitted 1/30/26, in February 2026 the Spon...
curator
News and press releases
null
LinkName:Ultragenyx Resubmits Biologics License Ap...
curator
News and press releases
null
LinkName:Ultragenyx Reports Fourth Quarter and Ful...
curator
Last update post date
2025-12-17
2026-03-25
api
Eligibility criteria
Inclusion criteria:
* diagnosis of mps iiia confi...
Inclusion criteria:
* diagnosis of mps iiia confi...
api
Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Ultragenyx Pharmaceutical Inc
Recruiting
BLA resubmitted 1/30/26, in February 2026 the Sponsor received an Incomplete Response Letter requesting additional supportive documentation related to CRL CMC responses
NCT06255782
Show Changes
2026-04-01
Recent updates
Sponsor reports complete clinical response in firs...
Sponsor reports complete clinical response in firs...
curator
Fda designation
Fast Track
Fast Track, Regenerative Medicine Advanced Therapy
curator
News and press releases
null
LinkName:iECURE Selected for U.S. FDA Chemistry, M...
curator
News and press releases
null
LinkName:iECURE Receives FDA Regenerative Medicine...
curator
Eligibility criteria
null
Key inclusion criteria:
1. male sex
2. gestationa...
api
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...
NCT06185673
Show Changes
2026-04-01
Recent updates
Interim study results posted January 2026
first Patient in Cohort 2 was safely treated with ...
curator
News and press releases
null
LinkName:Benitec Biopharma Releases Second Quarter...
curator
Eligibility criteria
null
Inclusion criteria:
* subject was previously enro...
api
News and press releases
LinkName:Benitec Biopharma Provides Positive Inter...
null
curator
Compound description
modified AAV9 capsid that expresses a bifunctional...
modified AAV9 capsid that expresses a bifunctional...
curator
Preclinical publications
null
LinkName:(Presentation) BB-301: A SINGLE “SILENCE ...
curator
Vector type
AAV9
AAV9-PL
curator
News and press releases
null
LinkName:Benitec Biopharma Provides Positive Long-...
curator
Last update post date
2025-02-05
2025-12-31
curator
Intervention description
Bb-301 is composed of an aav9 capsid, Aav9pl, Whic...
Bb-301 is composed of an aav9 capsid, Aav9pl, Whic...
curator
Oculopharyngeal Muscular Dystrophy
Benitec Biopharma, Inc.
Recruiting
First Patient in Cohort 2 was safely treated with the higher-dose of BB-301 in 4Q of 2025 and an update on the interim clinical results of Cohort 2 is planned for mid-2026
NCT04411654
Show Changes
2026-04-01
Last update post date
2025-12-15
2026-04-01
api
Eligibility criteria
null
Inclusion criteria:
* bi-allelic gba1 mutations c...
api
Gaucher Disease Type 2
Prevail Therapeutics
Active not recruiting
NCT06480461
Show Changes
2026-03-31
Last update post date
2024-06-28
2026-03-31
curator
Estimated completion date
2031-07-01
2030-09-10
curator
Overall status
Not yet recruiting
Active not recruiting
curator
Number of locations
0
1
curator
Locations
null
China
curator
Eligibility criteria
null
1 cm3 infarct, Which, In the opinion of the investigator, Are contraindications to intraparenchymal injection.
17. clinically significant abnormalities in laboratory test values at screening are in the opinion of the investiga-tor, Unsuitable for enrollment immediately.
subject under any of the following general conditions
18. contraindications to mri/pet and/or agents used in pet.
19. contraindications to general anesthesia or deep sedation.
20. woman who is pregnant or breastfeeding.
21. participation within 3 months prior to screening in another therapeutic investigational drug or device study, Unless it can be documented that the patient received a placebo.
22. presence of substance (drug, Alcohol) abuse within 2 years prior to screening.
23. the patient is generally frail or has any condition for which, In view of the investigator, Participation in the study would not be in the best interest of the patient or is likely to prohibit further participation during the study.">Inclusion criteria:
* subjects must meet all the ...
api
Parkinson's Disease
Shanghai Vitalgen BioPharma Co., Ltd.
Active not recruiting
IND cleared in July 2024
NCT07282847
Show Changes
2026-03-31
Last update post date
2026-02-24
2026-03-31
api
Eligibility criteria
null
Inclusion criteria:
1. participant must be ≥18 ye...
api
Record status
Draft
Active
curator
Preclinical publications
LinkName:Int J Mol Sci
. 2022 Jun 4;23(11):6298. ...
LinkName:Isogenic GAA-KO Murine Muscle Cell Lines ...
curator
Indication doid
null
DOID:2752
curator
Development status
null
Active
curator
Compound name
null
AB-1009
curator
Recent updates
null
FDA accepted IND in January 2026
curator
Dose 2
null
1.5E13 vg/kg
curator
Dose 1
null
1E13 vg/kg
curator
Vector type
null
AAV
curator
Delivery system
null
viral transduction
curator
Drug product type
null
viral vector
curator
Route of administration
null
intravenous
curator
Mechanism of action
null
functional gene replacement
curator
Therapy route
null
in-vivo
curator
Therapy type
null
gene transfer
curator
Target gene
null
GAA
curator
Fda designation
Fast Track, Orphan Drug Designation
curator
Preclinical publications
null
LinkName:Rescue of Pompe disease in mice by AAV-me...
curator
Preclinical publications
null
LinkName:Low-Dose Liver-Targeted Gene Therapy for ...
curator
News and press releases
null
LinkName:Genethon Signs Licensing Agreement with A...
curator
News and press releases
null
LinkName:AskBio Announces FDA Acceptance of Invest...
curator
Pompe Disease (Late-onset), Pompe Disease Late-Onset, LOPD
AskBio Inc
Recruiting
FDA accepted IND in January 2026
NCT06292650
Show Changes
2026-03-31
Last update post date
2025-12-04
2026-03-31
curator
Eligibility criteria
null
Inclusion criteria:
patients who meet all of the ...
api
Retinitis Pigmentosa
Zhongmou Therapeutics
Recruiting
Two patients treated early 2024, IND cleared December 2025
NCT06066008
Show Changes
2026-03-31
Last update post date
2024-02-21
2026-03-31
curator
Estimated completion date
2027-10
2026-01-31
curator
Overall status
Recruiting
Completed
curator
Enrollment type
Estimated
Actual
curator
Enrollment count
9
2
curator
Eligibility criteria
null
Inclusion criteria:
subjects who meet all of the ...
api
X-Linked Retinoschisis
Zhongmou Therapeutics
Completed
Showing 1-54 of 54 results in ClinicalTrials
SCGE Platform Gene Therapy Clinical Trials downloaded on: 2026/04/14 06:58:14; 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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X-Linked Myotubular Myopathy
Sponsor
Astellas Gene Therapies (Industry)
Compound
AT132
Therapy Type
Gene transfer
Target Gene
MTM1
Vector Type
AAV8
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2017-06-21
Last Modified Date: 2026-04-08
Enrollment: 27
Canada, France, Germany + 1 more
Spinal Muscular Atrophy
Sponsor
Novartis Pharmaceuticals (Industry)
Compound
ITVISMA
Therapy Type
Gene transfer
Target Gene
SMN1
Vector Type
AAV9
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: 2026-04-07
Enrollment: 27
Australia, Belgium, Canada + 6 more
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor
Beacon Therapeutics (Industry)
Compound
AGTC-501
Therapy Type
Gene transfer
Target Gene
RPGR
Vector Type
AAV2tYF
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2021-04-05
Last Modified Date: 2026-04-07
Enrollment: 85
Australia, Canada, United Kingdom + 1 more
Mucopolysaccharidosis Type II (Hunter Syndrome)
Sponsor
REGENXBIO Inc. (Industry)
Compound
RGX-121
Therapy Type
Gene transfer
Target Gene
IDS
Vector Type
AAV9
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2018-05-01
Last Modified Date: 2026-04-07
Enrollment: 48
Brazil, United States
Rett Syndrome
Sponsor
Taysha Gene Therapies, Inc. (Industry)
Compound
TSHA-102
Therapy Type
Gene transfer
Target Gene
MiniMECP2
Vector Type
scAAV9
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2022-10-28
Last Modified Date: 2026-04-07
Enrollment: 15
Canada, United States
Duchenne Muscular Dystrophy (DMD)
Sponsor
Sarepta Therapeutics, Inc. (Industry)
Compound
ELEVIDYS
Therapy Type
Gene transfer
Target Gene
Micro-dystrophin
Vector Type
AAVrh74
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Priority Review, Accelerated Approval, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-05-19
Last Modified Date: 2026-04-07
Enrollment: 148
Australia, Belgium, Canada + 11 more
Retinitis Pigmentosa
Sponsor
Ocugen (Industry)
Compound
OCU400
Therapy Type
Gene transfer
Target Gene
NR2E3
Vector Type
AAV5
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2024-04-09
Last Modified Date: 2026-04-06
Enrollment: 140
Canada, United States
Ornithine Transcarbamylase (OTC) Deficiency
Sponsor
Ultragenyx Pharmaceutical Inc (Industry)
Compound
DTX301
Therapy Type
Gene transfer
Target Gene
OTC
Vector Type
AAV8
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation
First Submitted: 2022-04-18
Last Modified Date: 2026-04-06
Enrollment: 32
Argentina, Brazil, Canada + 7 more
Glycogen Storage Disease Type Ia
Sponsor
Ultragenyx Pharmaceutical Inc (Industry)
Compound
DTX401
Therapy Type
Gene transfer
Target Gene
G6PC
Vector Type
AAV8
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2021-07-14
Last Modified Date: 2026-04-06
Enrollment: 49
Brazil, Canada, Denmark + 6 more
Sickle Cell Disease
Sponsor
Genetix Biotherapeutics Inc. (Industry)
Compound
LYFGENIA
Therapy Type
Gene transfer
Target Gene
HBB
Vector Type
BB305 LV
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2020-02-12
Last Modified Date: 2026-04-06
Enrollment: 35
United States
Neovascular Age-related Macular Degeneration
Sponsor
AbbVie (Industry)
Compound
ABBV-RGX-314
Therapy Type
Gene transfer
Target Gene
Anti-VEGF
Vector Type
AAV8
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
First Submitted: 2025-05-28
Last Modified Date: 2026-04-06
Enrollment: 561
Austria, Belgium, Bulgaria + 14 more
X-Linked Retinitis Pigmentosa (XLRP)
Sponsor
Janssen Research & Development, LLC (Industry)
Compound
Botaretigene sparoparvovec
Therapy Type
Gene transfer
Target Gene
RPGR
Vector Type
AAV2/5
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: 2026-04-06
Enrollment: 105
Belgium, Canada, Denmark + 8 more
Stargardt Disease
Sponsor
Ocugen (Industry)
Compound
OCU410ST
Therapy Type
Gene transfer
Target Gene
RORA
Vector Type
AAV5
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Orphan Drug Designation
First Submitted: 2023-06-30
Last Modified Date: 2026-04-06
Enrollment: 51
United States
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor
Adverum Biotechnologies, Inc. (Industry)
Compound
ADVM-022
Therapy Type
Gene transfer
Target Gene
Codon optimized aflibercept
Vector Type
AAV.7m8
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
FDA Designations
Fast Track, Regenerative Medicine Advanced Therapy
First Submitted: 2025-02-26
Last Modified Date: 2026-04-06
Enrollment: 284
United States
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor
Abeona Therapeutics, Inc (Industry)
Compound
ZEVASKYN
Therapy Type
Gene transfer
Target Gene
COL7A1
Vector Type
LZRSE
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-01-23
Last Modified Date: 2026-04-06
Enrollment: 12
United States
Hemophilia B
Sponsor
Pfizer (Industry)
Compound
BEQVEZ
Therapy Type
Gene transfer
Target Gene
F9-R338L
Vector Type
AAVrh74
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2019-03-01
Last Modified Date: 2026-04-03
Enrollment: 51
Australia, Brazil, Canada + 13 more
Choroideremia
Sponsor
Biogen (Industry)
Compound
BIIB111
Therapy Type
Gene transfer
Target Gene
CHM
Vector Type
AAV2
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Regenerative Medicine Advanced Therapy
First Submitted: 2018-03-07
Last Modified Date: 2026-04-03
Enrollment: 169
Canada, Denmark, Finland + 5 more
Aromatic L-amino Acid Decarboxylase (AADC) Deficiency
Sponsor
Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound
VGN-R09b
Therapy Type
Gene transfer
Target Gene
DDC
Vector Type
AAV9
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2024-05-17
Last Modified Date: 2026-04-03
Enrollment: 13
China
Leber Hereditary Optic Neuropathy (LHON)
Sponsor
GenSight Biologics (Industry)
Compound
LUMEVOQ
Therapy Type
Gene transfer
Target Gene
ND4G11778A
Vector Type
AAV2
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2016-01-07
Last Modified Date: 2026-04-03
Enrollment: 39
France, Germany, Italy + 2 more
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Sponsor
Spark Therapeutics, Inc. (Industry)
Compound
LUXTURNA
Therapy Type
Gene transfer
Target Gene
RPE65
Vector Type
AAV2
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Priority Review, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2009-10-21
Last Modified Date: 2026-04-03
Enrollment: 31
United States
Hemophilia B
Sponsor
Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound
BBM-H901
Therapy Type
Gene transfer
Target Gene
F9
Vector Type
AAV843
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2021-12-29
Last Modified Date: 2026-04-01
Enrollment: 32
China
Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor
Ultragenyx Pharmaceutical Inc (Industry)
Compound
UX111
Therapy Type
Gene transfer
Target Gene
SGSH
Vector Type
AAV9
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2016-03-17
Last Modified Date: 2026-04-01
Enrollment: 36
Australia, Spain, United States
Transthyretin Amyloidosis (ATTR) with Cardiomyopathy
Sponsor
Intellia Therapeutics (Industry)
Compound
NTLA-2001
Therapy Type
Gene editing
Target Gene
TTR
Vector Type
LNP
Delivery System
Lipid encapsulation
Mechanism of Action
Gene inactivation
FDA Designations
Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-11-08
Last Modified Date: 2026-03-27
Enrollment: 1200
Argentina, Australia, Austria + 23 more
Hemophilia B
Sponsor
CSL Behring (Industry)
Compound
HEMGENIX
Therapy Type
Gene transfer
Target Gene
F9-R338L
Vector Type
AAV5
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: 2026-03-27
Enrollment: 67
Belgium, Denmark, Germany + 5 more
Beta-Thalassemia, Sickle Cell Disease
Sponsor
Vertex Pharmaceuticals Incorporated (Industry)
Compound
CASGEVY
Therapy Type
Gene editing
Target Gene
BCL11A
Vector Type
RNP
Delivery System
Electroporation
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2022-07-26
Last Modified Date: 2026-03-23
Enrollment: 26
Germany, Italy, Saudi Arabia + 1 more
Rett Syndrome
Sponsor
Neurogene Inc. (Industry)
Compound
NGN-401
Therapy Type
Gene transfer
Target Gene
MECP2
Vector Type
AAV9
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Support for Clinical Trials Advancing Rare Disease Therapeutics, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2023-06-01
Last Modified Date: 2026-03-18
Enrollment: 33
Australia, United Kingdom, United States
Neovascular Age-Related Macular Degeneration (nAMD)
Sponsor
4D Molecular Therapeutics (Industry)
Compound
4D-150
Therapy Type
Gene transfer
Target Gene
Anti-VEGF
Vector Type
AAV
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
FDA Designations
Regenerative Medicine Advanced Therapy
First Submitted: 2025-07-07
Last Modified Date: 2026-03-17
Enrollment: 480
Argentina, Bulgaria, Germany + 9 more
Diabetic Foot Ulcer
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapy Type
Gene transfer
Target Gene
HGF
Delivery System
None (naked plasmid)
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2015-09-28
Last Modified Date: 2026-03-17
Enrollment: 44
United States
Critical Limb Ischemia
Sponsor
Beijing Northland Biotech. Co., Ltd. (Industry)
Compound
NL003
Therapy Type
Gene transfer
Target Gene
HGF
Delivery System
None (naked plasmid)
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2020-01-18
Last Modified Date: 2026-03-17
Enrollment: 302
China
Beta-Thalassemia Major
Sponsor
Genetix Biotherapeutics Inc. (Industry)
Compound
ZYNTEGLO
Therapy Type
Gene transfer
Target Gene
HBB
Vector Type
BB305 LV
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: 2026-03-17
Enrollment: 19
France, Germany, Greece + 3 more
Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome)
Sponsor
LYSOGENE (Industry)
Compound
LYS-SAF301
Therapy Type
Gene transfer
Target Gene
SGSH
Vector Type
AAV2rh.10
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2018-07-04
Last Modified Date: 2026-03-17
Enrollment: 20
France, Germany, Netherlands + 2 more
Duchenne Muscular Dystrophy (DMD)
Sponsor
Solid Biosciences Inc. (Industry)
Compound
SGT-003
Therapy Type
Gene transfer
Target Gene
Micro-dystrophin
Vector Type
AAV-SLB101
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Fast Track, Rare Pediatric Disease Designation
First Submitted: 2025-08-29
Last Modified Date: 2026-03-17
Enrollment: 80
Australia, Canada
Duchenne Muscular Dystrophy (DMD)
Sponsor
REGENXBIO Inc. (Industry)
Compound
RGX-202
Therapy Type
Gene transfer
Target Gene
Micro-dystrophin
Vector Type
AAV8
Delivery System
Viral transduction
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2023-01-04
Last Modified Date: 2026-03-17
Enrollment: 65
Canada, United States
Familial Lipoprotein Lipase Deficiency
Sponsor
Amsterdam Molecular Therapeutics (Industry)
Compound
GLYBERA
Therapy Type
Gene transfer
Target Gene
LPL
Vector Type
AAV1
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation
First Submitted: 2009-04-30
Last Modified Date: 2026-03-17
Enrollment: 5
Canada
Metachromatic Leukodystrophy
Sponsor
Orchard Therapeutics (Industry)
Compound
LENMELDY
Therapy Type
Gene transfer
Target Gene
ARSA
Vector Type
VSV-G
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2020-02-13
Last Modified Date: 2026-03-17
Enrollment: 6
Italy
Bietti Crystalline Dystrophy
Sponsor
Chigenovo Co., Ltd (Network)
Compound
ZVS101e
Therapy Type
Gene transfer
Target Gene
CYP4V2
Vector Type
AAV2/8
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2024-12-17
Last Modified Date: 2026-03-17
Enrollment: 62
China
Gaucher Disease Type 1
Sponsor
Spur Therapeutics (Industry)
Compound
FLT201
Therapy Type
Gene transfer
Target Gene
GBA1
Vector Type
AAVS3
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Regenerative Medicine Advanced Therapy
First Submitted: 2025-10-30
Last Modified Date: 2026-03-17
Enrollment: 45
Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Sponsor
AbbVie (Industry)
Compound
ABBV-RGX-314
Therapy Type
Gene transfer
Target Gene
Anti-VEGF
Vector Type
AAV8
Delivery System
Viral transduction
Mechanism of Action
Genetic delivery of therapeutic protein
First Submitted: 2021-01-08
Last Modified Date: 2026-03-17
Enrollment: 630
United States
Hemophilia A
Sponsor
Shanghai Xinzhi BioMed Co., Ltd. (Industry)
Compound
BBM-H803
Therapy Type
Gene transfer
Target Gene
F8
Vector Type
AAV
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation
First Submitted: 2023-10-27
Last Modified Date: 2026-03-17
Enrollment: 55
China
Diabetic Neuropathy
Sponsor
Helixmith Co., Ltd. (Industry)
Compound
ENGENSIS
Therapy Type
Gene transfer
Target Gene
HGF
Delivery System
None (naked plasmid)
Mechanism of Action
Overexpression of protective allele/gene
FDA Designations
Regenerative Medicine Advanced Therapy
First Submitted: 2020-06-25
Last Modified Date: 2026-03-17
Enrollment: 162
United States
Duchenne Muscular Dystrophy (DMD)
Sponsor
Pfizer (Industry)
Compound
PF-06939926
Therapy Type
Gene transfer
Target Gene
Mini-dystrophin
Vector Type
AAV9
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2020-02-11
Last Modified Date: 2026-03-17
Enrollment: 114
Australia, Belgium, Canada + 12 more
Peripheral Artery Disease
Sponsor
Beijing Northland Biotech. Co., Ltd. (Industry)
Compound
NL003
Therapy Type
Gene transfer
Target Gene
HGF
Delivery System
None (naked plasmid)
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2023-01-04
Last Modified Date: 2026-03-17
Enrollment: 36
China
Cerebral Adrenoleukodystrophy (CALD)
Sponsor
Genetix Biotherapeutics Inc. (Industry)
Compound
SKYSONA
Therapy Type
Gene transfer
Target Gene
ABCD1
Vector Type
LV
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: 2026-03-17
Enrollment: 35
France, Germany, Italy + 3 more
Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor
Krystal Biotech, Inc. (Industry)
Compound
VYJUVEK
Therapy Type
Gene transfer
Target Gene
COL7A1
Vector Type
HSV1
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Priority Review, Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2021-06-02
Last Modified Date: 2026-03-17
Enrollment: 47
United States
Bietti Crystalline Dystrophy
Sponsor
Shanghai Vitalgen BioPharma Co., Ltd. (Industry)
Compound
VGR-R01
Therapy Type
Gene transfer
Target Gene
CYP4V2
Vector Type
AAV2/8
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation
First Submitted: 2024-11-19
Last Modified Date: 2026-03-17
Enrollment: 45
China
Hemophilia A
Sponsor
Pfizer (Industry)
Compound
Giroctogogene fitelparvovec
Therapy Type
Gene transfer
Target Gene
F8
Vector Type
AAV2/6
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2020-04-21
Last Modified Date: 2026-03-17
Enrollment: 77
Australia, Brazil, Canada + 13 more
Wiskott-Aldrich Syndrome
Sponsor
Fondazione Telethon (Other)
Compound
WASKYRA
Therapy Type
Gene transfer
Target Gene
WAS
Vector Type
LV
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2019-02-08
Last Modified Date: 2026-03-17
Enrollment: 10
Italy, United States
Mucopolysaccharidosis Type I (Hurler Syndrome)
Sponsor
Orchard Therapeutics (Industry)
Compound
OTL-203
Therapy Type
Gene transfer
Target Gene
IDUA
Vector Type
LV
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2023-11-17
Last Modified Date: 2026-03-17
Enrollment: 41
Italy, Netherlands, United Kingdom + 1 more
Limb-Girdle Muscular Dystrophy, Type 2E/R4
Sponsor
Sarepta Therapeutics, Inc. (Industry)
Compound
SRP-9003
Therapy Type
Gene transfer
Target Gene
SGCB
Vector Type
AAVrh74
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Fast Track, Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-01-30
Last Modified Date: 2026-03-17
Enrollment: 17
Belgium, Germany, Italy + 3 more
Hemophilia A
Sponsor
Gritgen Therapeutics Co., Ltd. (Industry)
Compound
GS1191-0445
Therapy Type
Gene transfer
Target Gene
F8
Vector Type
AAV8
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
First Submitted: 2025-01-28
Last Modified Date: 2026-03-17
Enrollment: 50
China
Critical Limb Ischemia
Sponsor
AnGes USA, Inc. (Industry)
Compound
COLLATEGENE
Therapy Type
Gene transfer
Target Gene
HGF
Delivery System
None (naked plasmid)
Mechanism of Action
Overexpression of protective allele/gene
First Submitted: 2014-05-20
Last Modified Date: 2026-03-17
Enrollment: 46
Belgium, Canada, Denmark + 8 more
Hereditary Spastic Paraplegia Type 50
Sponsor
Elpida Therapeutics SPC (Industry)
Compound
MELPIDA
Therapy Type
Gene transfer
Target Gene
AP4M1
Vector Type
AAV9
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Orphan Drug Designation, Rare Pediatric Disease Designation
First Submitted: 2024-11-14
Last Modified Date: 2026-03-17
Enrollment: 24
Spain, United States
Hemophilia A
Sponsor
BioMarin Pharmaceutical (Industry)
Compound
ROCTAVIAN
Therapy Type
Gene transfer
Target Gene
F8
Vector Type
AAV5
Delivery System
Viral transduction
Mechanism of Action
Functional gene replacement
FDA Designations
Breakthrough Therapy, Fast Track, Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2020-03-24
Last Modified Date: 2026-03-17
Enrollment: 22
Australia, Brazil, Taiwan + 1 more
Hereditary Angioedema
Sponsor
Intellia Therapeutics (Industry)
Compound
NTLA-2002
Therapy Type
Gene editing
Target Gene
KLKB1
Vector Type
LDLR
Delivery System
Lipid encapsulation
Mechanism of Action
Gene inactivation
FDA Designations
Orphan Drug Designation, Regenerative Medicine Advanced Therapy
First Submitted: 2024-10-07
Last Modified Date: 2026-03-17
Enrollment: 60
Australia, Canada, France + 6 more