January 06, 2017
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FDA clears application for SB-525 gene therapy program for hemophilia A

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The FDA cleared Sangamo Biosciences’ investigational new drug application for its SB-525 gene therapy program, intended for the treatment of adults with hemophilia A.

The clearance allows for clinical development to assess the efficacy, safety and tolerability of the treatment.

The SB-525 gene therapy program uses an adeno-associated virus (AAV2/6) complementary DNA human Factor VIII construct driven by a proprietary synthetic liver-specific promoter, according to a company-issued press release.

Preclinical studies indicated the therapy was more potent than existing adeno-associated virus complementary DNA therapies under investigation for hemophilia A treatment.

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