Sangamo Therapeutics announced the U.S. Food and Drug Administration (FDA) has accepted the Investigational New Drug (IND) application for ST-920, a gene therapy candidate being evaluated for the treatment of adults with Fabry disease. Current standard of care for this rare, progressive condition involves regular lifelong infusions of enzyme replacement therapy (ERT).

"The FDA's acceptance of the IND to evaluate ST-920 in Fabry disease enables the initiation of our third clinical development program focused on rare metabolic diseases, and our sixth active clinical program," said Edward Conner, M.D., Chief Medical Officer at Sangamo. "We are eager to commence trials of ST-920 to evaluate our hypothesis that the one-time administration of a gene therapy can fundamentally change the clinical course for patients with 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.

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