PTC Therapeutics, Inc. has completed the acquisition of ST-920, a gene therapy product candidate for Fabry disease, from Sangamo Therapeutics, Inc. The transaction was finalized on September 21, 2026, as disclosed in a press release furnished with the company's Form 8-K.
ST-920, also known as isaralgagene civaparvovec, is a one-time administered AAV gene therapy. The company stated that a rolling biologics license application (BLA) submission to the U.S. Food and Drug Administration for accelerated approval is expected to be completed in the fourth quarter of 2026.
The BLA is based on data from the Phase 1/2 STAAR study, which demonstrated a meaningful favorable clinical effect on renal function and a positive safety and tolerability profile over 52 weeks. Clinical data indicated that the therapy enables long-term production of the alpha-galactosidase A enzyme and leads to a significant reduction in globotriaosylceramide (Gb3) levels.
PTC Therapeutics noted that the therapy addresses the limitations of chronic Enzyme Replacement Therapy (ERT). The product has received various designations, including Orphan Drug, Fast Track, and Regenerative Medicine Advanced Therapy (RMAT) status from the FDA. It has also been granted Orphan Medicinal Product designation and PRIME eligibility by the European Medicines Agency.
