Sangamo Therapeutics Advances Rolling Submission of BLA to U.S. FDA for ST-920 in Fabry Disease

Core Insights - Sangamo Therapeutics is advancing the rolling submission of a Biologics License Application (BLA) to the FDA for isaralgagene civaparvovec, a gene therapy for Fabry disease, aiming for accelerated approval based on positive clinical data [1][3] Group 1: Clinical Study and Data - The STAAR study demonstrated a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all dosed patients, which the FDA has agreed will serve as an endpoint for accelerated approval [1][3] - The Phase 1/2 STAAR study is a global, open-label, single-dose, dose-ranging clinical study designed to evaluate isaralgagene civaparvovec in patients with Fabry disease [4] - Data from the STAAR study were presented at the 22nd Annual WORLDSymposium™, highlighting the potential of isaralgagene civaparvovec as a one-time, well-tolerated, and durable treatment option [3] Group 2: Regulatory Pathway - Following the initiation of the rolling submission in December 2025, Sangamo has submitted preclinical and clinical modules to the FDA for ongoing review [2] - The antibody assay companion diagnostic for screening patient eligibility has been submitted and accepted by the FDA's Center for Devices and Radiological Health for Premarket Approval [2] Group 3: Company Overview - Sangamo Therapeutics focuses on genomic medicine, aiming to develop treatments for serious neurological diseases with inadequate existing options [6] - The company’s pipeline includes multiple partnered programs and opportunities for further partnerships and investments [6]