Sangamo Therapeutics Begins Rolling FDA Submission For ST-920 Gene Therapy To Treat Adults With Fabry Disease

Sangamo Therapeutics, Inc., a genomic medicine company, has begun a rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of isaralgagene civaparvovec, also known as ST-920. ST-920 is Sangamo’s wholly owned investigational gene therapy for the treatment of adults with Fabry disease. A rolling submission allows completed sections of the BLA to be submitted and reviewed by the FDA on an ongoing basis, rather than waiting for the entire application to be complete.

Nathalie Dubois-Stringfellow, Ph.D., Chief Development Officer at Sangamo, stated that the initiation of the BLA submission is an important milestone for the company and for patients with Fabry disease. She highlighted that the data from the registrational STAAR study demonstrate the potential of ST-920 to provide safe, long-lasting clinical benefits to a broad population of Fabry patients. She added that Sangamo looks forward to collaborating with the FDA as the regulatory process continues.

The STAAR study data suggest that isaralgagene civaparvovec may serve as a one-time, durable therapy that addresses the underlying pathology of Fabry disease, offering meaningful clinical benefits across multiple organs beyond current standard-of-care treatments. The study also showed a positive mean annualized eGFR slope at 52 weeks across all dosed patients, which the FDA has agreed can serve as an endpoint to support accelerated approval.

Isaralgagene civaparvovec has received Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency, and eligibility for the Innovative Licensing and Access Pathway from the U.K. Medicines and Healthcare products Regulatory Agency. Sangamo expects to complete the BLA submission to the FDA under the accelerated approval pathway by the second quarter of 2026.