- Sangamo BioSciences(NASDAQ:SGMO) announces that the FDA has granted orphan drug designation to SB-FIX, the company's zinc finger nuclease ("ZFN")-mediated genome editing product candidate for the treatment of hemophilia B.
- Sangamo expects to initiate a Phase 1/2 clinical study (SB-FIX-1501) in adult subjects with the disease in 2016.
- Management commentt: "By enabling targeted integration of a therapeutic factor 9 gene, our ZFN-mediated genome editing approach may offer hemophilia B patients a therapeutic option that has potential advantages over conventional gene therapy approaches. We will enroll adult hemophilia patients into our first clinical trial, however, our goal is to move into pediatric patients, a population we believe could particularly benefit from a treatment that has the potential to provide lifelong expression of therapeutic levels of Factor IX protein."
- Source: Press Release