Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced 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. As of the September 17, 2021 cutoff date, results from the four patients treated in the first two dose cohorts (0.5e13 vg/kg and 1e13 vg/kg) showed that isaralgagene civaparvovec was generally well tolerated. All four patients exhibited above normal alpha-galactosidase A (α-Gal A) activity, which was maintained for up to one year for the first patient treated and through 14 weeks for the most recently treated patient. Activity of 2-fold to 15-fold above mean normal was observed at last measurement as of the cutoff date. Withdrawal from enzyme replacement therapy (ERT) has taken place for one patient and is planned for the other patient on ERT, based on the stability of their α-Gal A activity following treatment.

“There is significant unmet need in Fabry disease, a progressive and challenging condition that is currently treated with frequent burdensome infusions that do not adequately address the underlying disease,” said Rob Schott, M.D., M.P.H, F.A.C.C, Head of Development at Sangamo. “We are encouraged by these early yet promising results, particularly that the first three treated patients dosed all reported improvements in their ability to sweat, which is noteworthy as this limits a person’s capability to tolerate strenuous tasks and exercise. As we continue with this study, we hope to further understand the potential treatment effect over the longer-term and in more patients, while we initiate plans for a Phase 3 trial.”

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