Sangamo Gene Therapy Study for Hemophilia A now Live on ClinicalTrials.gov

SB-525 for Hemophilia A

Sangamo has developed an adeno-associated virus (AAV) carrying a clotting Factor 8 gene construct driven by Sangamo's proprietary synthetic liver specific promoter, which in preclinical studies is more potent than existing AAV-based cDNA constructs currently under evaluation for the treatment of hemophilia A. The Company’s investigational new drug (IND) application for this program has been cleared by the U.S. Federal Drug Administration (FDA) enabling the company to begin a Phase 1/2 clinical trial in adults with severe hemophilia A.

https://clinicaltrials.gov/ct2/show/NCT03061201?term=sangamo&rank=4