FDA approves Sangamo's drug application for hemophilia A treatment

The U.S. Food and Drug Administration has approved Sangamo BioSciences Inc.'s Investigational New Drug Application for the SB-525 gene therapy program for patients with hemophilia A.
The program is now active, which enables the company’s clinical development team to research the safety, tolerability and potential efficacy of the product on patients with hemophilia A.
"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," Sangamo CEO Dr. Sandy Macrae said in a statement. "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."
Hemophilia, caused by genetic mutations, is a rare disorder in which a person’s blood does not clot normally. The mutation occurs in roughly one in 5,000 live male births, according to statistics the U.S. Centers for Disease Control and Prevention. Estimates show that about 16,000 people are living with hemophilia A in the U.S.