Sangamo Biosciences, Inc. Announces FDA Clearance of Investigational New Drug Application for SB-525 Gene Therapy Program for Hemophilia A
January 05, 2017 at 06:30 pm IST
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Sangamo BioSciences, Inc. announced that the U.S. Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug application (IND) for its SB-525 gene therapy program for the treatment of hemophilia A. The IND is now active and enables clinical development to assess the safety, tolerability and potential efficacy of SB-525 in adults with hemophilia A. SB-525 is one of four lead development programs for which Sangamo is planning to conduct clinical trials i 2017. Sangamo is evaluating SB-FIX, an in vivo genome editing therapy for hemophilia B, in a Phase 1/2 clinical trial, with sites currently screening patients for the study. This year Sangamo will also conduct two Phase 1/2 clinical trials evaluating in vivo genome editing therapies for lysosomal storage disorders MPS I (SB-318) and MPS II (SB-913).
Sangamo Therapeutics, Inc. is a genomic medicines company that is developing medicines for neurological diseases. The Company's neurology preclinical development is focused on two areas: development of epigenetic regulation therapies to treat serious neurological diseases, and development of novel engineered adeno-associated virus (AAV) capsids to deliver its therapies to the intended neurological targets. The Company's zinc finger epigenetic regulators are ideally suited to potentially address neurological disorders and its capsid discovery platform is expanding delivery beyond intrathecal delivery capsids, including in the central nervous system. Its clinical-stage product candidates are Isaralgagene civaparvovec, also known as ST-920, its wholly-owned gene therapy product candidate for the treatment of Fabry disease, and TX200, its wholly-owned CAR-Treg cell therapy product candidate for the prevention of immune-mediated rejection in HLA-A2 mismatched kidney transplantation.
SANGAMO THERAPEUTICS, INC. 
