PTC Therapeutics Inc. (NASDAQ: PTCT) completed its acquisition of the gene therapy asset ST-920 from Sangamo Therapeutics, the company said on September 21. The deal gives PTC rights to develop the therapy for Fabry disease.
ST-920, also known as isaralgagene civaparvovec, is a one-time administered adeno-associated virus (AAV) gene therapy. According to PTC, the treatment is designed to enable production of the deficient alpha-galactosidase A enzyme and reduce levels of globotriaosylceramide — a substance that accumulates in cells due to mutations in the galactosidase alpha gene.
Fabry disease is a lysosomal storage disorder that causes damage to organs including the kidney, heart, nerves, eyes, gut and skin.
PTC, based in Warren, New Jersey, expects to complete a rolling Biologics License Application submission to the U.S. Food and Drug Administration for accelerated approval of ST-920 in the fourth quarter of 2026.
The asset has received several regulatory designations. The FDA granted orphan drug, fast track and regenerative medicine advanced therapy status. The European Medicines Agency issued orphan medicinal product designation and confirmed PRIME eligibility. In the United Kingdom, the Medicines and Healthcare products Regulatory Agency approved the asset for the Innovative Licensing and Access Pathway.
Matthew B. Klein, chief executive officer of PTC Therapeutics, said the company looks forward to completing the ST-920 BLA submission and potentially bringing a "one-time administered, safe and effective durable disease treatment" that addresses the limitations of chronic enzyme replacement therapy to the Fabry community.
