ATSN-201 Gene Therapy in RS1-Associated X-linked Retinoschisis

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

NCT00875160 ·Status: TERMINATED ·Phase: PHASE1

Clinical Evaluation of Patients With X-linked Retinoschisis

NCT02331173 ·Status: COMPLETED

Extension Study of AGT-181-102 to Evaluate Long Term Safety and Activity of AGT-181

NCT02597114 ·Status: COMPLETED ·Phase: PHASE1

Gene Transfer Clinical Study in Crigler-Najjar Syndrome

NCT03223194 ·Status: TERMINATED ·Phase: PHASE1

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy

NCT02603562 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944 ·Status: RECRUITING ·Phase: PHASE3

Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201

NCT04999059 ·Status: TERMINATED

Long-Term Follow-Up of Subjects Treated With AXO-AAV-GM2 for Tay-Sachs or Sandhoff Disease

NCT06614569 ·Status: ACTIVE_NOT_RECRUITING

A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Aniridia

NCT04117880 ·Status: WITHDRAWN ·Phase: PHASE2

An Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2 Who Have Completed MEX-DM-302 Study.

NCT06549400 ·Status: ENROLLING_BY_INVITATION ·Phase: PHASE3

Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1)

NCT01175239 ·Status: UNKNOWN ·Phase: NA

Gene Therapy for Severe Crigler Najjar Syndrome

NCT03466463 ·Status: RECRUITING ·Phase: NA

An Initial Study of AZD7325 in Adults With Fragile X Syndrome

NCT03140813 ·Status: COMPLETED ·Phase: PHASE1

A Gene Therapy Study in Patients With Gaucher Disease Type 1

NCT05324943 ·Status: COMPLETED ·Phase: PHASE1

Safety and Tolerability of VGR-R01 for Patients With Bietti Crystalline Dystrophy

NCT05694598 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1

An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3

NCT05815004 ·Status: WITHDRAWN ·Phase: PHASE2/PHASE3

A Study of AT132 in Young Children With X-Linked Myotubular Myopathy (XLMTM)

NCT03199469 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2/PHASE3

To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria

NCT07318909 ·Status: NOT_YET_RECRUITING ·Phase: EARLY_PHASE1

Phase IA and IB Study of AAVrh.10hFXN Gene Therapy for the Cardiomyopathy of Friedreich's Ataxia

NCT05302271 ·Status: RECRUITING ·Phase: PHASE1

Long-term Follow-Up for RGX-121

NCT04597385 ·Status: UNKNOWN

A Study of Gene Therapy for Classic Congenital Adrenal Hyperplasia (CAH)

NCT04783181 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

4D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)

NCT04517149 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of Intravitreal Injections of FCFD4514S in Patients With Geographic Atrophy

NCT00973011 ·Status: COMPLETED ·Phase: PHASE1

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

NCT04145037 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency

NCT02168686 ·Status: COMPLETED ·Phase: PHASE1/PHASE2