Sangamo Therapeutics, Inc. (SGMO) Tuesday reported positive topline results from the registrational Phase 1/2 STAAR study of its gene therapy candidte isaralgagene civaparvovec for the treatment of Fabry disease.
Fabry disease, is a rare genetic disorder that can affect many parts of the body.
Following a single dose of isaralgagene civoparvovec, a positive mean annualized eGFR (estimated Glomerular Filtration Rate) slope of 1.965 mL/min/1.73m2/year at 52-weeks was observed across all 32 dosed patients in the study, which the U.S. Food and Drug Administration (FDA) has agreed will serve as primary basis of approval.
Sangamo intends to submit a Biologics License Application (BLA) in 2026.
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