Sangamo BioSciences, Inc. Announce that the U.S. Food and Drug Administration (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.
"We are pleased with the FDA's decision to grant orphan drug designation to SB-FIX for hemophilia B," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "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."
"Our mission is to translate our ground-breaking science into genetic therapies that transform patients' lives," said Sandy Macrae, M.B., Ch.B., Ph.D., Sangamo's president and chief executive officer. "This is another positive step forward for the first therapeutic in vivo genome editing application cleared for clinical evaluation in humans. I am pleased with the progress that we have made to advance this program and we look forward to initiating a Phase 1/2 clinical trial by the end of 2016."
SB-FIX is designed as a single treatment strategy intended to provide stable, continuous production of Factor IX clotting protein (FIX) for the lifetime of the patient. Sangamo's ZFN-mediated in vivo genome editing approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic "safe-harbor site," that can be edited with ZFNs to accept and express therapeutic genes. The approach is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein product.
Hemophilia, a rare bleeding disorder in which the blood does not clot normally, is caused by mutations in genes that encode factors which help the blood clot and stop bleeding when blood vessels are injured. Hemophilia B is caused by a defect in the gene encoding clotting Factor IX protein and individuals with this mutation experience bleeding episodes after injuries and spontaneous bleeding episodes that often lead to joint disease such as arthritis.
