Gene Therapy

A Study to Evaluate a Novel Gene Therapy in Patients With Relapsed and Refractory Multiple Myeloma

Gene Replacement Therapy Clinical Trial for Participants With Spinal Muscular Atrophy Type 1

Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning

Needs Assessment of Knowledge, Beliefs, and Attitudes of Patients With Hemophilia B About Gene Therapy

Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5

CIBMTR Research Database

Gene Therapy for Chinese Hemophilia A

Perceptual Evaluation and Rehabilitation System Development for Congenital Hearing Loss

A Clinical Study of the Safety and Efficacy of Chemogenetics Therapy in the Treatment of Parkinson's Disease

Long-Term Follow-Up Study of RGX-314 Administered in the Suprachoroidal Space for Participants With nAMD

A Prospective, Single-center, Open-label Clinical Study to Evaluate the Safety and Efficacy of Inhaled BMD003(CFTR mRNA) in Chinese Adult Patients With Cystic Fibrosis

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

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

Growth and Development-related Outcomes in Children With Transfusion-dependent Beta-thalassemia After Gene Therapy

Intravitreal AAVCAGsCD59 for Advanced Dry Age-related Macular Degeneration (AMD) With Geographic Atrophy (GA)

Gene Therapy for X Linked Severe Combined Immunodeficiency

Safety and Tolerability Study of AAV2-sFLT01 in Patients With Neovascular Age-Related Macular Degeneration (AMD)