Sangamo Therapeutics Announces Evidence of Clinical Benefit in Phase 1/2 STAAR Study 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 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 All five patients who began the dose escalation phase on ERT had been successfully withdrawn from ERT and continued to exhibit supraphysiological levels of α-Gal A activity following withdrawal.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.
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.
Read the press release here