Sangamo Presents Positive Phase 1/2 Gene Therapy Data for Fabry Disease

Sangamo Therapeutics, a genomic medicine firm, has reported positive interim results from its Phase 1/2 STAAR clinical trial for isaralgagene civaparvovec (ST-920), a gene therapy for Fabry disease. The treatment has shown sustained elevated α-Gal A activity for up to three years in the longest-treated patient, with all 12 patients withdrawn from enzyme replacement therapy (ERT) maintaining this elevated activity for up to 19 months. Antibodies against α-Gal A have significantly decreased in patients with ERT-associated antibodies at baseline, becoming undetectable in five patients. Among 13 patients monitored for over a year, renal function was stable, and improvements were noted in disease severity, quality of life, and gastrointestinal symptoms. The therapy demonstrated a favorable safety profile, with no post-treatment liver function test elevations requiring steroids. Enrollment in the Phase 1/2 study is complete, with dosing of remaining patients expected in the first half of 2024. Sangamo continues discussions with the U.S. FDA and other health authorities regarding registration pathways. The data will be presented at the 20th Annual WORLDSymposium in San Diego on February 7, 2024.

The Phase 1/2 STAAR study is an open-label, single-dose, dose-ranging, multicenter clinical trial assessing the safety and tolerability of ST-920 in Fabry disease patients. The study includes patients on ERT, ERT pseudo-naïve, or ERT-naïve. ST-920 has received Orphan Drug, Fast Track, and RMAT designations from the FDA and Orphan Medicinal Product designation from the European Medicines Agency.

Fabry disease is a lysosomal storage disorder caused by a deficiency in the α-Gal A enzyme, leading to a buildup of Gb3 in cells and damage to vital organs. Symptoms include decreased sweat production, heat intolerance, kidney disease, heart failure, gastrointestinal issues, and neuropathic pain.

Sangamo Therapeutics is focused on developing genomic medicines for serious neurological disorders without adequate treatment options. The company's zinc finger epigenetic regulators and capsid discovery platform aim to address these disorders and expand delivery beyond current intrathecal delivery capsids, including in the central nervous system.

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