PTC Therapeutics completes purchase of Sangamo's Fabry disease gene therapy
The company plans to finish a rolling FDA submission for the treatment, ST-920, in the fourth quarter of 2026
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PTC Therapeutics has completed its purchase of ST-920, a gene therapy for Fabry disease, from Sangamo Therapeutics. The deal had already been announced.
ST-920 is a one-time treatment given through a single infusion, using a viral vector called AAV to deliver its therapy. It is still awaiting approval. PTC Therapeutics expects to finish a rolling submission to the Food and Drug Administration in the fourth quarter of 2026, seeking accelerated approval.
"Our team looks forward to completing the ST-920 BLA submission and potentially bringing a one-time administered, safe and effective durable disease treatment," said Matthew B. Klein, the company's chief executive.
The acquisition gives PTC Therapeutics a clinical-stage gene therapy in a field where several rivals are already moving one-time treatments through development for other rare diseases. With the FDA submission not due to be completed until late 2026, any approval is roughly two years away, leaving the company without a near-term catalyst even as approvals in metabolic disease, oncology and other rare conditions have been picking up pace elsewhere in biotech.