AcquisitionsAug 13, 2026, 07:03 AM
PTC Therapeutics to Acquire ST-920 for Fabry Disease for $111M Upfront
AI Summary
PTC Therapeutics, Inc. announced it was selected as the winning bidder to acquire ST-920, a BLA-stage one-time administered AAV gene therapy for Fabry disease, from Sangamo Therapeutics. The acquisition involves a $111 million upfront payment and up to $100 million in contingent milestone payments based on regulatory approvals. The company expects to complete a rolling BLA submission to the FDA for accelerated approval in Q4 2026, with a potential commercial launch in 2027, leveraging its existing rare disease infrastructure.
Key Highlights
- PTC Therapeutics to acquire ST-920, a BLA-stage gene therapy for Fabry disease.
- Acquisition includes a $111 million upfront payment.
- Up to $100 million in contingent milestone payments, tied to regulatory approvals.
- ST-920 is a one-time administered AAV gene therapy for Fabry disease.
- Rolling BLA submission to FDA for accelerated approval expected to be completed in Q4 2026.
- Potential for commercial launch of ST-920 in 2027.
- Phase 1/2 STAAR study showed positive mean annualized eGFR slope at 52 weeks.
- ST-920 has RMAT, Orphan Drug, and Fast Track designations from FDA.
Price Impact
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