FDA Grants Orphan Drug Designation for SB-FIX for Hemophilia B

The FDA granted orphan drug designation to SB-FIX – a zinc finger nuclease–mediated in vivo genome-editing product – for patients with hemophilia B. SB-FIX is designed to be a one-time intravenous treatment that provides stable, continuous production of factor IX proteins throughout a patient’s life. A phase I/II, non-randomized, open-label clinical trial to assess the safety, tolerability, and efficacy of SB-FIX in adult patients with hemophilia B is scheduled to take place later this year at five sites in the United States.

Source: Sangamo BioSciences press release, September 6, 2016