Warren, N.J. – September 21, 2026 -- PTC Therapeutics, Inc. (NASDAQ: PTCT) has completed its acquisition of ST-920, a BLA-stage, one-time AAV gene therapy for Fabry disease, from Sangamo Therapeutics, Inc.
PTC targets Q4 2026 completion of rolling BLA submission for accelerated approval
The company confirmed that a rolling Biologics License Application submission to the FDA seeking accelerated approval of ST-920, also known as isaralgagene civaparvovec, is expected to be completed in the fourth quarter of 2026.
Gene therapy aims to replace chronic enzyme replacement therapy with single administration
ST-920 is designed to deliver long-term production of the deficient alpha-galactosidase A (α-Gal A) enzyme through a single administration, addressing the treatment burden associated with chronic Enzyme Replacement Therapy (ERT) for Fabry disease, a lysosomal storage disorder caused by GLA gene mutations. In clinical studies, the therapy demonstrated durable clinical benefit alongside significant reductions in globotriaosylceramide (Gb3) levels, the substance that accumulates in cells and damages the kidney, heart, nerves, eyes, gut and skin.
Regulatory agencies across three major markets have granted expedited designations
The FDA has awarded ST-920 Orphan Drug, Fast Track and Regenerative Medicine Advanced Therapy (RMAT) designations. The European Medicines Agency has granted Orphan Medicinal Product designation and PRIME eligibility, while the U.K. Medicines and Healthcare products Regulatory Agency has included the candidate in its Innovative Licensing and Access Pathway.
Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, said the team is focused on completing the BLA submission and potentially delivering a durable, one-time treatment option for the Fabry community.