"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
"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
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
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.
About Sangamo's IVPRP
The IVPRP approach makes use of the albumin gene locus, a highly expressing and liver-specific genomic "safe-harbor site," that can be edited with zinc finger nucleases (ZFNs) to accept and express any therapeutic gene. The platform is designed to enable the patient's liver to permanently produce circulating therapeutic levels of a corrective protein product such as Factor VIII or IX to treat hemophilia, or replacement enzymes to treat lysosomal storage disorders. The ability to permanently integrate the therapeutic gene in a highly specific targeted fashion significantly differentiates Sangamo's IVPRP approach from conventional AAV gene therapy approaches, which are non-integrating, and may "wash out" of the liver as cells divide and turn over. Ultimately, the target population for IVPRP programs will be pediatric patients and it will be important in this population to be able to produce stable levels of therapeutic protein for the lifetime of the patient. With such a large capacity for protein production (approximately 15g/day of albumin), targeting and co-opting only a very small percentage of the albumin gene's capacity is sufficient to produce the needed replacement protein at therapeutically relevant levels with no significant effect on albumin production.
About Hemophilia B
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. According to the
ZFP Therapeutic® is a registered trademark of
This press release may contain forward-looking statements based on Sangamo's current expectations. These forward-looking statements include, without limitation, references relating to research and development of novel ZFP TFs and ZFNs and therapeutic applications of Sangamo's ZFP technology platform, the potential of Sangamo's ZFP technology to treat hemophilia B, hemophilia A and lysosomal storage disorders, including MPS I, MPS II, Gaucher disease, the expected timing of trial enrollment for SB-FIX-1501 and filing of IND applications for hemophilia A, MPS I MPS II and Gaucher disease, the impact of the SB-FIX clinical trial on the field of genetic medicine, and the safety and efficacy of the approach of using ZFN-mediated genome editing. Actual results may differ materially from these forward-looking statements due to a number of factors, including uncertainties relating to the initiation and completion of stages of our clinical trials, whether the clinical trials will validate and support the safety, tolerability and efficacy of ZFNs and ZFP TFs, technological challenges, Sangamo's ability to develop commercially viable products and technological developments by our competitors. For a more detailed discussion of these and other risks, please see Sangamo's public filings with the
To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/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-300185804.html
News Provided by Acquire Media
Copyright 2016 Sangamo BioSciences, Inc.