Sangamo Therapeutics Shares Updated Data From Fabry Gene Therapy Study, Says It Shows Evidence Of Clinical Benefit

  • Sangamo Therapeutics Inc SGMO announced updated preliminary data as of October 20, 2022, the cutoff date from the Phase 1/2 STAAR study of isaralgagene civaparvovec, or ST-920, a wholly owned gene therapy product candidate for Fabry disease. 
  • As of the November 15, 2022, supplemental cutoff date, 13 patients exhibited supraphysiological levels of α-Gal A activity, sustained for over two years for the patient with the longest follow-up. 
  • All five patients who began the dose escalation phase on enzyme replacement therapy (ERT) had been successfully withdrawn from ERT and continued to exhibit supraphysiological levels of α-Gal A activity following withdrawal. 
  • Related: Sangamo Touts Additional Positive Data For Fabry Gene Therapy Candidate.
  • No patient has required the resumption of ERT treatment to date.
  • One patient achieved 78% globotriaosylceramide (Gb3) substrate clearance at six months and a 77% reduction in urine podocyte loss in one of the first kidney biopsies.
  • Gb3 is a fatty substrate that accumulates in the cells of Fabry disease patients and can damage multiple organs.
  • Since the cutoff date, four additional patients have been dosed in the expansion phase, and two more patients have been withdrawn from ERT.
  • The Phase 1/2 STAAR study expansion phase is ongoing, and preparations for a potential Phase 3 trial are underway, anticipated to start by the end of 2023. Dosing could begin as early as the first part of 2024.
  • Price Action: SGMO shares traded 9.97% higher at $3.20 on the last check Thursday.
Market News and Data brought to you by Benzinga APIs
Comments
Loading...
Posted In: BiotechNewsPenny StocksHealth CareSmall CapMoversGeneralBriefswhy it's moving
Benzinga simplifies the market for smarter investing

Trade confidently with insights and alerts from analyst ratings, free reports and breaking news that affects the stocks you care about.

Join Now: Free!