Sangamo: Fabry Gene Therapy Update Shows Evidence Of Clinical Benefit
- Sangamo Therapeutics Inc 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- All five patients who began the dose escalation phase on enzyme replacement therapy (ERT) had been successfully withdrawn from ERT and continued to exhibit supraphysiological levels of α-Gal A activity following withdrawal. .
- 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Since the cutoff date, four additional patients have been dosed in the expansion phase, and two more patients have been withdrawn from ERT.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.
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