RICHMOND, Calif., Aug. 25, 2017 /PRNewswire/ -- Sangamo Therapeutics, Inc. (Nasdaq: SGMO) and Pfizer Inc. (NYSE: PFE) today announced that the first patient received treatment in the Phase 1/2 clinical trial ("the Alta trial") evaluating SB-525, an investigational gene therapy for patients with Hemophilia A, a rare genetic blood disorder.
"We are excited to announce the first patient infusion in this initial clinical trial of SB-525. Gene therapy holds promise as a potential one-time treatment to control Hemophilia A and prevent spontaneous bleeding," said Dr. Sandy Macrae, Chief Executive Officer of Sangamo Therapeutics.
The Alta trial is an open-label, adaptive, dose-ranging clinical study designed to assess the safety and tolerability of SB-525 investigational gene therapy in up to 20 adult subjects with severe Hemophilia A. SB-525 is being developed as part of a global collaboration between Sangamo and Pfizer for the development and commercialization of gene therapy programs for Hemophilia A.
"We are excited to see progress in our partnership program for SB-525 with Sangamo," said Greg LaRosa, Chief Scientific Officer, Pfizer Rare Disease. "By combining Sangamo's scientific and technical expertise across multiple genomic platforms, with Pfizer's global reach, resources and expertise, we are advancing the promise of gene therapy with the hope of making a meaningful impact for people living with Hemophilia A."
SB-525 comprises a recombinant adeno-associated virus (rAAV) vector carrying a Factor VIII gene construct driven by a proprietary, synthetic, liver-specific promoter. The U.S. Food and Drug Administration has granted Orphan Drug and Fast Track designations to SB-525, which also received Orphan Medicinal Product designation from the European Medicines Agency. Hemophilia A is a rare blood disorder caused by a genetic mutation resulting in insufficient activity of Factor VIII, a blood clotting protein the body uses to stop bleeding. There are approximately 16,000 patients in the U.S. and more than 150,000 worldwide with Hemophilia A.
About Sangamo Therapeutics Sangamo Therapeutics, Inc. is focused on translating ground-breaking science into genomic therapies that transform patients' lives using the company's industry leading platform technologies in genome editing, gene therapy, gene regulation and cell therapy. The Company has open Phase 1/2 clinical trials in Hemophilia A and Hemophilia B, and lysosomal storage disorders MPS I and MPS II. Sangamo has an exclusive, global collaboration and license agreement with Pfizer Inc. for gene therapy programs for Hemophilia A, with Bioverativ Inc. for hemoglobinopathies, including beta thalassemia and sickle cell disease, and with Shire International GmbH to develop therapeutics for Huntington's disease. In addition, it has established strategic partnerships with companies in non-therapeutic applications of its technology, including Sigma-Aldrich Corporation and Dow AgroSciences. For more information about Sangamo, visit the Company's website at www.sangamo.com.
About Pfizer Inc.: Working together for a healthier worldTM At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world's best-known consumer health care products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.pfizer.com. In addition, to learn more, please visit us on www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
Forward Looking Statements This press release contains forward-looking statements, including, but not limited to, statements related to the therapeutic potential of gene therapy and SB-525, the number of patients to be treated in the Alta trial and other statements that are not historical facts. These forward-looking statements are based on Sangamo's current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with: gene therapy product candidate development and the uncertainty of clinical success, including whethertheAlta trial willvalidateandsupportthetolerabilityandefficacyofSB-525; Sangamo's substantial dependence on the clinical success of its lead therapeutic programs, including SB-525; the initiation, enrollment and completion of the stages of the Alta trial and its other clinical trials; technological challenges; the lengthy and uncertain regulatory approval process; Sangamo's ability to develop commercially viable products; technological developments by its competitors; and Sangamo's dependence on its collaboration with Pfizer and its ability to maintain its collaboration with Pfizer. A more detailed discussion of these and other risks and uncertainties may be found under the caption "Risk Factors" and elsewhere in Sangamo's SEC filings and reports, including Sangamo's Quarterly Report on Form 10-Q for the quarter ended June 30, 2017 and future filings and reports by Sangamo. Sangamo assumes no obligation to update the forward-looking information contained in this press release.