Sangamo Stock Gains on FDA Submission for Fabry Disease Gene Therapy
The biotech company has initiated a rolling Biologics License Application for ST-920, a potential one-time treatment for the rare genetic disorder, signaling a major regulatory milestone.
Sangamo Therapeutics (NASDAQ: SGMO) saw its shares climb in trading after announcing it has begun a rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for its investigational gene therapy, ST-920, aimed at treating Fabry disease.
The initiation of the BLA is a pivotal step for the clinical-stage biotechnology company, moving its lead gene therapy candidate closer to potential commercialization. The rolling submission process allows Sangamo to submit completed sections of its application for FDA review on an ongoing basis, a designation often granted to therapies for serious conditions that can expedite the review timeline. Sangamo expects to complete the BLA submission in the second quarter of 2026.
"The initiation of our BLA submission marks an important milestone for Sangamo and for Fabry patients in need," said Nathalie Dubois-Stringfellow, Ph.D., Sangamo’s Chief Development Officer, in a statement released by the company. "The compelling data from our STAAR study shows the potential of ST-920 to provide safe and long-lasting clinical benefits to a wide range of Fabry disease patients."
Fabry disease is a rare and progressive genetic disorder caused by a deficiency of the alpha-galactosidase A enzyme. This deficiency leads to the harmful buildup of a fatty substance known as globotriaosylceramide (Gb3) in cells throughout the body, resulting in potential damage to critical organs, including the kidneys, heart, and brain. The current standard of care primarily involves lifelong intravenous enzyme replacement therapy (ERT) every two weeks, a market dominated by major pharmaceutical players like Sanofi with its drug Fabrazyme.
The global treatment market for Fabry disease was valued at over $2 billion in 2023 and is projected to more than double in the coming years, according to market research firms. Sangamo’s ST-920 (isaralgagene civaparvovec) aims to disrupt this paradigm by offering a potential one-time treatment. The gene therapy is designed to deliver a functional copy of the GLA gene to the liver, enabling the patient's own body to produce the deficient enzyme continuously.
Data from Sangamo's registrational STAAR clinical trial has been encouraging. The company highlighted that the study met an endpoint agreed upon with the FDA for accelerated approval: a positive mean annualized estimated glomerular filtration rate (eGFR) slope, which measures change in kidney function. The therapy has also demonstrated a favorable safety profile to date.
The potential of ST-920 has been recognized by regulators, with the therapy receiving Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA, as well as PRIME eligibility from the European Medicines Agency.
For Sangamo, a company with a market capitalization of approximately $148 million, a successful path to market for ST-920 would be transformative. While the company's stock has struggled over the past year, trading near its 52-week low, the average 12-month price target from analysts sits at $3.25, suggesting significant upside potential if it can successfully execute on its regulatory and commercial strategy. The initiation of the BLA is the first major step in realizing that potential, offering a glimmer of hope for both patients seeking a more durable treatment and investors awaiting a catalyst.