Indication
Gene Therapy
5 clinical trials
5 products
Product
SPK-8016Clinical trial
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII InhibitorsStatus: Completed, Estimated PCD: 2020-10-14
Clinical trial
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan ConditioningStatus: Active (not recruiting), Estimated PCD: 2025-10-01
Clinical trial
A Phase 1/2 Safety, Tolerability, and Efficacy Study of Valoctocogene Roxaparvovec, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Hemophilia A Patients With Residual FVIII Levels ≤ 1 IU/dL and Pre-existing Antibodies Against AAV5Status: Active (not recruiting), Estimated PCD: 2024-11-01
Product
Valoctocogene RoxaparvovecClinical trial
Growth and Development, Health-related Quality of Life of Children With Transfusion-dependent Beta-thalassemia After Gene TherapyStatus: Recruiting, Estimated PCD: 2028-12-31
Product
Gene therapyClinical trial
Clinical Exploration of Clinical Exploration Adeno-associated Virus Vector Expression of Human Coagulation Factor VIII Gene Therapy for Hemophilia AStatus: Recruiting, Estimated PCD: 2028-07-31
Product
GS001