PTC Therapeutics is buying a gene therapy for Fabry disease from Sangamo Therapeutics in a deal worth up to $211 million — picking up a promising asset after its onetime pioneer fell into bankruptcy.
Under the agreement (dated August 13, 2026), PTC will pay $111 million up front, plus $80 million tied to an accelerated FDA approval and $20 million for full approval. PTC won the asset at auction after Sangamo — once a genetic-medicine trailblazer, undone by a series of setbacks and ill-fated partnerships — filed for Chapter 11 bankruptcy. (Eli Lilly separately won other Sangamo assets, including its zinc-finger technology, for $50 million.)
The therapy
The drug, ST-920 (isaralgagene civaparvovec), is a one-time infusion that delivers a working copy of the GLA gene, which is faulty in Fabry disease — an inherited disorder that damages the heart, kidneys and other organs. The Fabry treatment market is already worth more than $2 billion, and a durable one-and-done gene therapy could address the shortcomings of current enzyme therapies that require regular infusions.
What’s next
A rolling FDA submission is underway; PTC plans to complete the application in the fourth quarter of 2026, with a potential launch in 2027. An analyst called it “a prudent deal with low risk and strong upside.” For PTC, it’s a chance to add a late-stage rare-disease asset cheaply; for patients, a possible new option for a lifelong condition.