Sangamo provides evidence of clinical benefit in Phase 1/2 STAAR study

Sangamo Therapeutics announced updated preliminary data as of the October 20, 2022 cutoff date 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. These data, which present new biomarker data and results from the first kidney biopsies in this study, indicate evidence of clinical benefit for isaralgagene civaparvovec in Fabry disease. As of the November 15, 2022 supplemental cutoff date, 13 patients across the dose escalation and expansion phases exhibited supraphysiological levels of alpha-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 All five patients who began the dose escalation phase on ERT had been successfully withdrawn from ERT and continued to exhibit supraphysiological levels of alpha-Gal A activity following withdrawal. No patient has required the resumption of ERT treatment to date. Importantly, there is evidence of significant Gb3 substrate reduction at six months in one of the first kidney biopsies taken from this study, along with a significant reduction in urine podocyte loss. In addition, the study observed a clinically meaningful and statistically significant increase in mean general health scores, as measured by the SF-36 General Health survey. These updated data will be shared at the 19th Annual WORLDSymposium on Friday, February 24, 2023 via an oral presentation in the session from 8:00-9:00am Eastern Time and a poster presentation from 3:00-4:00pm Eastern Time (Poster Ref: 169). These data will also be available on Sangamo’s website on the Presentations page.