Sangamo BioSciences Announces FDA Clearance Of Investigational New Drug Application For SB-FIX, First In Vivo Protein Replacement Platform Program For Treatment Of Hemophilia B

Sangamo BioSciences, Inc.

, the leader in therapeutic genome editing, announced that the U.S. Food and Drug Administration (FDA) has cleared the Company's Investigational New Drug (IND) application for SB-FIX, a potentially curative, single treatment therapy for hemophilia B. SB-FIX is the first in vivo genome editing application to enter the clinic and is based on Sangamo's proprietary In Vivo Protein Replacement Platform™ (IVPRP™). The IND is now active and enables Sangamo to initiate a Phase 1/2 clinical study (SB-FIX-1501) designed to assess safety, tolerability and potential efficacy of SB-FIX in adults with hemophilia B. Sangamo BioSciences, Inc. "We are very pleased to be in a position to initiate a clinical trial of our hemophilia B program which will not only be the first clinical application of our IVPRP but the first human study of any in vivo genome editing application," said Edward Lanphier, Sangamo's president and chief executive officer. "The FDA's determination that our planned clinical trial may proceed is not only an important milestone for Sangamo's hemophilia program, but is a major de-risking event for our entire IVPRP as we are able to leverage this same approach to develop ZFP Therapeutics® for hemophilia A and numerous lysosomal storage disorders (LSDs). This is a significant achievement for the fields of genome editing and gene therapy, and marks a new era in genetic medicine." "A single treatment with SB-FIX has the potential to provide life-long, stable expression of clinically relevant levels of Factor IX (FIX) protein and eliminate patients' need for chronic transfusions of recombinant clotting factor," said Geoff Nichol, M.B., Ch.B., Sangamo's executive vice president of research and development. "Our proprietary IVPRP genome editing approach allows us to precisely target and edit the albumin "safe harbor" locus in the DNA of liver cells which we expect to result in therapeutic levels of clotting factor that are maintained for the life of the patient. In contrast, conventional AAV gene therapy approaches, which are non-integrating, have the potential to "wash out" over time as the patient's liver cells divide and turn over. Ultimately, our target population for this approach will be young children with hemophilia B who will benefit most from a permanent treatment." SB-FIX-1501 is a Phase 1/2 open-label, dose-escalation study in male subjects over eighteen years of age, with severe hemophilia B, who do not have inhibitors to FIX and have no hypersensitivity to recombinant Factor IX (rFIX) protein. The study, which will begin enrolling up to nine subjects in 2016, will evaluate the safety and efficacy of a single administration of SB-FIX. The principal investigators are Nadia Ewing, M.D., Director, Comprehensive Hemophilia Treatment Center and Sickle Cell Program, and John Zaia, M.D., Director, Center for Gene Therapy at City of Hope in Duarte, California. The ZFP Therapeutic includes the therapeutic FIX replacement gene and zinc finger nucleases (ZFNs) formulated as adeno-associated virus (AAV) vector preparations and will be administered as a single intravenous dose. Sangamo remains on track to file an IND for MPS I (Hurler syndrome), the first of the Company's LSD programs employing its IVPRP approach, by the end of 2015, and an IND application for MPS II (Hunter syndrome) in the first half of 2016. The Company also expects to file three more IND applications in the second half of 2016 for hemophilia A, Gaucher disease, and one other LSD target.