PTC Completes Acquisition of Gene Therapy for Fabry Disease

WARREN, N.J., Sept. 21, 2026 /CourierPR/ -- PTC Therapeutics, Inc., a global biopharmaceutical company, has completed the acquisition of ST-920, a gene therapy for Fabry disease. This move marks a significant step in PTC's strategy to provide innovative treatments for rare disorders.

ST-920, developed by Sangamo Therapeutics, Inc., is a one-time administered AAV (adeno-associated virus) gene therapy designed to address the limitations of chronic enzyme replacement therapy (ERT) for Fabry disease. The therapy enables long-term production of the deficient alpha-galactosidase A (α-Gal A) enzyme and has shown significant reduction in globotriaosylceramide (Gb3) levels, leading to durable clinical benefits.

The FDA has granted Orphan Drug, Fast Track, and RMAT (Regenerative Medicine Advanced Therapy) designations to ST-920, highlighting its potential as a breakthrough treatment. Additionally, the therapy has received Orphan Medicinal Product designation and PRIME (Priority Medicines) eligibility from the European Medicines Agency and the U.K. Medicines and Healthcare products Regulatory Agency, respectively.

Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA), leading to deficient α-Gal A enzyme activity. This deficiency results in the buildup of Gb3 in cells, causing serious damage to vital organs such as the kidney, heart, nerves, eyes, gut, and skin. Symptoms include decreased or absent sweat production, heat intolerance, angiokeratoma (skin blemishes), vision problems, kidney disease, heart failure, gastrointestinal disturbance, mood disorders, neuropathic pain, and tingling in the extremities.

PTC Therapeutics is dedicated to the discovery, development, and commercialization of clinically differentiated medicines for children and adults living with rare disorders. The company's pipeline includes several promising therapies, including ST-920, which is expected to be submitted for regulatory approval in the fourth quarter of 2026.

The completion of the acquisition and the anticipated BLA submission underscore PTC's commitment to bringing innovative treatments to patients with unmet medical needs. The company is poised to potentially revolutionize the treatment landscape for Fabry disease with ST-920, offering a one-time, safe, and effective solution that addresses the limitations of current therapies.