Sangamo's Fabry Disease Gene Therapy Continues To Show Encouraging Safety Profile

Sangamo Therapeutics Inc SGMO has announced updated preliminary results from the Phase 1/2 STAAR study of isaralgagene civaparvovec, or ST-920, a gene therapy candidate for Fabry disease.
As of the February 14 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.
The sixth patient exhibited elevated α-Gal A activity within the normal range at two weeks post-dosing.
Related: Sangamo Therapeutics Shares Updated Interim Data From Fabry Disease Gene Therapy Trial
The first five patients treated across three dose cohorts showed sustained elevated α-Gal A activity ranging from nearly 3-fold to nearly 17-fold above mean normal.
Three patients have anecdotally reported improvements in their symptoms, including improvements in the ability to sweat, a primary and common Fabry disease symptom.
No progression of Fabry cardiomyopathy was observed in those patients who presented with signs of cardiomyopathy.
Sangamo expects to provide additional results from the STAAR study in 2H of 2022 and is currently planning a potential Phase 3 trial.
Price Action: SGMO shares closed at $5.25 on Monday.