Sangamo Therapeutics Announces Positive Phase 1/2 STAAR Study Results for Fabry Gene Therapy

Sangamo Therapeutics Inc. (NASDAQ:SGMO) is one of the best penny stocks under $1 to buy now. On June 24, Sangamo Therapeutics announced positive topline results from its registrational Phase 1/2 STAAR study. This global, open-label, single-dose, dose-ranging, multicenter clinical study evaluated isaralgagene civaparvovec (ST-920), which is a wholly-owned investigational gene therapy for the treatment of adults with Fabry disease.

The study included male and female patients who were either on enzyme replacement therapy/ERT, ERT pseudo-naïve (off ERT for 6 or more months), or ERT-naïve. The median age of enrolled patients was 42, with a median follow-up duration of 24 months, and the longest-treated patient had achieved 4.5 years of follow-up.

A scientist peering into a microscope, researching the next gene therapy breakthrough.

The US FDA agreed that this eGFR slope will serve as an intermediate clinical endpoint for potential approval under the Accelerated Approval pathway. Key secondary endpoints in the study were also positive. All 18 patients who began the study on ERT have successfully discontinued it and remain off ERT as of the announcement date. Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene/GLA, leading to deficient alpha-galactosidase A (α-Gal A) enzyme activity.

Sangamo Therapeutics Inc. (NASDAQ:SGMO) is a clinical-stage genomic medicine company that translates science into medicines that transform the lives of patients and families afflicted with serious diseases in the US.

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Disclosure: None. This article is originally published at Insider Monkey.