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

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

NCT07318909 ·Status: NOT_YET_RECRUITING ·Phase: EARLY_PHASE1

A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria

NCT05972629 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema

NCT05121376 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

NCT06948019 ·Status: NOT_YET_RECRUITING ·Phase: PHASE1/PHASE2

Gene Transfer Study in Patients With Late Onset Pompe Disease

NCT04174105 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE1/PHASE2

Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug

NCT06391736 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

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

NCT05878860 ·Status: RECRUITING ·Phase: PHASE3

A Study of AAV9 Gene Therapy in Participants With Canavan Disease (CANaspire Clinical Trial)

NCT04998396 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

A Study to Evaluate Safety, Tolerability, and Efficacy of AB-1009 Gene Therapy (GAA Gene) in Adult Participants With Late Onset Pompe Disease (PROGRESS-GT LOPD)

NCT07282847 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

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

NCT02603562 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Clinical Exploration of Adeno-associated Virus (AAV) Expressing Human Acid Alpha- Glucosidase (GAA) Gene Therapy for Patients With Infantile-onset Pompe Disease

NCT05567627 ·Status: RECRUITING ·Phase: NA

Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia

NCT04117711 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

NCT05222178 ·Status: TERMINATED ·Phase: PHASE1

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

NCT00635427 ·Status: COMPLETED ·Phase: PHASE3

Gene Replacement Therapy for Treatment of Paediatric Patients With CTNNB1 Neurodevelopmental Syndrome

NCT07270549 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Gene Therapy for Severe Crigler Najjar Syndrome

NCT03466463 ·Status: RECRUITING ·Phase: NA

A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

NCT00433147 ·Status: COMPLETED ·Phase: PHASE2

A Dose-finding Study to Evaluate mRNA-3210 in Participants With Phenylketonuria

NCT06147856 ·Status: WITHDRAWN ·Phase: PHASE1/PHASE2

A Phase 1/2/3 Study of TSHA-102 Gene Therapy in Females With Rett Syndrome (REVEAL Pivotal Study)

NCT05606614 ·Status: RECRUITING ·Phase: PHASE3

Study of Infigratinib in Children With Achondroplasia

NCT04265651 ·Status: COMPLETED ·Phase: PHASE2

A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy

NCT06907875 ·Status: RECRUITING ·Phase: PHASE1/PHASE2

Study of BH4, a New and Simple Treatment of Mild PKU

NCT00260000 ·Status: COMPLETED ·Phase: PHASE2

Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

NCT02725580 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

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

NCT02597114 ·Status: COMPLETED ·Phase: PHASE1

First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221

NCT02675465 ·Status: COMPLETED ·Phase: PHASE1/PHASE2