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Sangamo Receives FDA Approval for Hemophilia A Gene Therapy Trial

January 10, 2017
Sangamo Receives FDA Approval for Hemophilia A Gene Therapy Trial

Sangamo recently announced that the US Food and Drug Administration (FDA) has cleared the Investigational New Drug Application (IND) for SB-525, the company’s gene therapy program for people with hemophilia A, or factor VIII (FVIII) deficiency. The clearance allows Sangamo to move forward with clinical development of SB-525 to evaluate its safety, tolerability and efficacy in adults with hemophilia A.

Sangamo is developing new gene therapies using adeno-associated viruses (AAVs) as delivery vehicles, or vectors, to carry the genetic codes that initiate the production of the deficient factor protein—in this case, FVIII in those with hemophilia A. The AAVs deliver this genetic material into liver cells, without causing disease or triggering significant immune responses.

“We are very pleased to begin 2017 with the announcement of an open IND for our SB-525 cDNA gene therapy and intend to initiate a clinical trial evaluating SB-525 as soon as possible,” said Sandy Macrae, MB, ChB, PhD, CEO of Sangamo. “We are committed to developing the best therapeutic options for patients, and based on non-human primate studies, SB-525 has demonstrated the potential to be the best-in-class treatment for hemophilia A.”

Sangamo is also investigating a therapy for hemophilia B, SB-FIX, using a proprietary “genome editing” technology. SB-FIX was granted orphan drug designation from the FDA in September 2016.

Source: Sangamo press release dated January 5, 2017