Sangamo Therapeutics Announces Updated Preliminary Phase 1/2 Data in Fabry Disease Clinical Study Showing Continued Tolerability and Sustained Elevated α-gal A Enzyme Activity in Five Longest Treated Patients

Author's Avatar
Aug 30, 2022

Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced updated preliminary results 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 latest data show that, as of the February 14, 2022 cutoff date, the investigational treatment continued to be generally well tolerated, with no treatment-related adverse events above Grade 1 (mild). The five longest treated patients continued to exhibit elevated alpha-galactosidase A (α-Gal A) activity, sustained up to 15 months as of the last date of measurement. The sixth patient exhibited elevated α-Gal A activity to within normal range at two weeks post dosing.