Sangamo Announces FDA Acceptance of IND Application for ST-920 Gene Therapy Candidate for Fabry Disease
Fabry disease is an inherited metabolic disease caused by mutations in the GLA gene, which result in a deficiency of the enzyme alpha-galactosidase A (α-Gal A). In the absence of functional enzyme, fatty compounds called ganglioside globotriaosylceramides (Gb3) accumulate and can cause serious harm to the skin, kidneys, heart, and the nervous system.
ST-920 comprises an AAV vector carrying a GLA gene construct driven by a proprietary liver-specific promoter. ST-920 gene therapy is designed to enable a patient's liver to produce a long-lasting and continuous supply of the α-Gal A enzyme.
The active IND enables Sangamo to initiate a Phase 1/2 clinical trial designed to assess the safety, tolerability and efficacy of ST-920 in adults with Fabry disease. Sangamo expects to open several clinical sites later this year.
About Sangamo Therapeutics
This press release contains forward-looking statements regarding Sangamo's current expectations. These forward-looking statements include, without limitation, Sangamo's plans to initiate the Phase 1/2 clinical trial for ST-920 in 2019; the potential for these trials to evaluate our hypothesis that the one-time administration of a gene therapy can fundamentally change the clinical course for these patients; the design of ST-920 gene therapy to enable a patient's liver to produce a long-lasting and continuous supply of the α-Gal A enzyme; and Sangamo's expectations to open several clinical sites later this year. These statements are not guarantees of future performance and are subject to certain risks, uncertainties and assumptions that are difficult to predict. Factors that could cause actual results to differ include, but are not limited to, the dependence on the success of clinical trials of programs, the lengthy and uncertain regulatory approval process, uncertainties related to the initiation, enrollment and completion of clinical trials, Sangamo's reliance on partners and other third-parties to meet their clinical and manufacturing obligations, and the ability to maintain strategic partnerships. Further, there can be no assurance that the necessary regulatory approvals will be obtained or that Sangamo and its partners will be able to develop commercially viable product candidates. Actual results may differ from those projected in forward-looking statements due to risks and uncertainties that exist in Sangamo's operations and business environments. These risks and uncertainties are described more fully in Sangamo's Quarterly Report on Form 10-Q for the quarter ended
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