PTC Therapeutics agrees to pay up to $211 million for Sangamo’s experimental Fabry disease gene therapy

PTC Therapeutics announced it will invest up to $211 million to acquire rights to an advanced gene therapy for Fabry disease being developed by Sangamo Therapeutics.

The therapy, called ST-920 (isaralgagene civaparvovec), delivers a functional copy of the GLA gene via a single infusion, aiming to address the enzyme deficiency that causes fatty deposits in patients.

Sangamo has begun a rolling FDA submission and expects PTC to complete the filing in the fourth quarter, with a potential launch in 2027. Analysts view the transaction as a high‑risk, high‑reward play given Sangamo’s recent bankruptcy filing.

The deal reflects PTC’s strategy to expand its pipeline with gene‑editing assets, while the therapy could offer advantages over existing Fabry treatments if regulatory approval is secured.