March 09, 2026-
Sangamo Therapeutics has advanced the regulatory process for its investigational AAV gene therapy isaralgagene civaparvovec (ST-920), announcing progress in the rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration for the treatment of adults with Fabry disease.
The company reported that it has now submitted the preclinical and clinical modules of the BLA following the initiation of the rolling submission in December 2025. Rolling submissions allow companies developing AAV gene therapies and other advanced medicines to submit completed sections of an application for review as they become available rather than waiting to file the entire package at once. Sangamo also submitted an antibody assay companion diagnostic to the FDA’s Center for Devices and Radiological Health seeking Premarket Approval to help identify patients eligible for the AAV gene therapy.
The regulatory filing is supported by data from the Phase 1/2 STAAR study, which evaluated the single-dose AAV gene therapy ST-920 in patients with Fabry disease. According to Sangamo, the study demonstrated a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks across all dosed patients, an endpoint that the FDA has agreed may support the accelerated approval pathway for the AAV-based gene therapy.
Researchers believe the totality of STAAR data supports the potential of the AAV gene therapy to serve as a one-time treatment capable of delivering durable enzyme expression and multi-organ clinical benefits in Fabry disease. Unlike enzyme replacement therapies that require lifelong infusions, ST-920 is designed to provide sustained production of the α-galactosidase A enzyme after a single administration of the AAV vector gene therapy.
The STAAR study is a global open-label, dose-ranging clinical trial evaluating the safety and efficacy of the single-infusion AAV gene therapy in individuals with Fabry disease. The therapy does not require preconditioning prior to administration. ST-920 has received multiple regulatory designations, including Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) status from the FDA, along with Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency.
Fabry disease is a rare lysosomal storage disorder caused by mutations in the GLA gene that reduce production of the enzyme α-galactosidase A. The resulting buildup of globotriaosylceramide in cells can damage multiple organs, including the kidneys, heart, nervous system, and skin, leading to symptoms such as neuropathic pain, kidney disease, heart complications, gastrointestinal issues, and heat intolerance.
If approved, ST-920 could become one of the next AAV gene therapies targeting metabolic disorders, potentially transforming treatment for patients living with Fabry disease.
