PTC Therapeutics has agreed to acquire Sangamo Therapeutics’ ST-920 Fabry disease gene therapy program, gaining control of a treatment candidate that is already undergoing a rolling regulatory submission for accelerated approval in the United States. The program, also known as isaralgagene civaparvovec, is being developed for Fabry disease and is moving toward a potential FDA review after Sangamo submitted the clinical and nonclinical portions of its biologics license application.
The acquisition comes as Sangamo sells assets through Chapter 11 bankruptcy proceedings. PTC said the program fits within its existing rare disease operations and plans to continue advancing the regulatory process.
Regulatory Submission for ST-920 Continues Under New Ownership
Sangamo initiated a rolling submission seeking accelerated approval of ST-920 in December. According to PTC, the remaining chemistry, manufacturing, and controls package is expected to be submitted to the FDA in the fourth quarter, completing the filing.
The company also plans to seek approvals outside the United States. Under the current regulatory strategy, 52-week clinical data are intended to support accelerated approval, while results collected at 104 weeks are expected to support a later application for full approval.
PTC Chief Executive Officer Matthew Klein said the company believes it can integrate the program into its existing operations.
“This is what made this a pretty special opportunity for us,” Klein said during a conference call with investors.
Klein also said PTC has the capacity and expertise needed to add the program without significant expansion of its current infrastructure.
Auction Process Draws Multiple Interested Bidders
The Fabry disease program was initially slated to be sold to Astellas, which had been selected as the stalking horse bidder in June. Under that proposed agreement, Astellas would have paid $25 million upfront and up to $25 million in milestone payments.
Sangamo later conducted an auction on Aug. 10 to allow competing offers. According to Klein, the bidding process lasted more than two days, reflecting substantial interest in the asset. PTC ultimately submitted the winning offer, while TerSera Therapeutics was named the backup bidder.
The transaction is expected to close by early in the fourth quarter.
Financial Terms of the Agreement
Under the agreement, PTC will pay $111 million in upfront cash for the ST-920 program. The deal also includes up to $100 million in milestone payments.
Of that amount, $80 million would become payable if ST-920 receives accelerated FDA approval, while the remaining $20 million would be tied to full approval of the therapy.
Lilly Purchases Additional Sangamo Programs and Platforms
Separate from the ST-920 transaction, Eli Lilly acquired several other Sangamo assets. Lilly purchased the company’s capsid delivery platform, zinc finger technology platform, MINT platform and the prion disease program ST-506.
Lilly had been designated as the stalking horse bidder for those assets and, after no competing qualified bids emerged, acquired them for the previously agreed purchase price of $50 million.
The asset sales represent a key part of Sangamo’s bankruptcy process, which has involved the divestiture of major programs and technology platforms while the company restructures its operations.
PTC Therapeutics has acquired the ST-920 gene therapy program from Sangamo Therapeutics, strengthening its focus on rare diseases and expanding its gene therapy portfolio.
The transaction gives PTC Therapeutics control of an investigational treatment designed for Fabry disease, a rare genetic disorder caused by mutations affecting the GLA gene and the production of the enzyme alpha-galactosidase A.
PTC Therapeutics Takes Over ST-920 Development
ST-920 is an investigational gene therapy that uses an adeno-associated virus vector to deliver a functional copy of the GLA gene. The approach is designed to enable patients’ cells to produce alpha-galactosidase A, potentially addressing an underlying cause of Fabry disease.
PTC Therapeutics Advances Gene Therapy Strategy
The acquisition strengthens PTC Therapeutics’ broader interest in genetic medicine and advanced therapeutic platforms. Gene therapy programs can offer the potential for longer-lasting treatment effects by delivering functional genetic material to target cells.
For PTC Therapeutics, ST-920 adds another rare disease asset to its development pipeline and could complement its existing focus on genetically driven conditions.
PTC Therapeutics and Future Clinical Development
Further development of ST-920 will depend on clinical data, regulatory interactions, and the company’s ability to demonstrate an acceptable safety and efficacy profile.
As PTC Therapeutics takes responsibility for the program, investors and the rare disease community will be watching for additional clinical updates and regulatory milestones.
PTC Therapeutics Strengthens Rare Disease Focus
The acquisition highlights the continued interest in gene therapies for rare genetic disorders. For PTC Therapeutics, bringing ST-920 into its portfolio represents another step toward expanding treatment options for patients with serious rare diseases.
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