Sangamo announces updated Phase 1/2 STAAR study data in fabry disease

Sangamo Therapeutics announced updated preliminary data from the Phase 1/2 STAAR clinical study evaluating isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for the treatment of Fabry disease. In the largest known clinical gene therapy program in Fabry disease to date, data from 24 patients continued to show durable safety and preliminary efficacy data as of the data cutoff date, which continue to underscore the potential of isaralgagene civaparvovec as a single-dose treatment option for Fabry disease. These data will be shared at the 20th Annual WORLDSymposium in San Diego, CA on Wednesday, February 7, 2024, via an oral presentation in the Clinical Applications session from 8:00-9:00 a.m. P.T. and a poster presentation from 3:00-5:00 p.m. P.T. These data will also be available on Sangamo’s website on the Presentations page. “Despite the availability of ERT and chaperone therapies, Fabry disease treatment is burdensome, with some patients still developing disease progression. To date, ST-920 has been well-tolerated, and the preliminary data showing sustained supraphysiologic alpha-Gal A activity and the ability to discontinue and remain off ERT are promising,” said Dr. Robert Hopkin, M.D., Cincinnati Children’s Hospital Medical Center, and investigator of the Phase 1/2 STAAR study. “The early improvements reported in disease severity, quality of life and gastrointestinal symptoms, together with evidence of reduced immunogenicity, illustrate the potential of ST-920 as a treatment option for adults with Fabry disease.”