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.
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