PTC Therapeutics expects FDA BLA filing for ST-920 Fabry gene therapy in Q4 2026

• PTC Therapeutics won a bankruptcy auction to acquire Sangamo’s BLA-stage ST-920 gene therapy for Fabry disease.
• Phase 1/2 STAAR results were already presented, showing sustained clinical benefit and a tolerable safety profile that supported stopping chronic enzyme therapy.
• Rolling FDA filing targets accelerated approval, with submission expected to be completed in Q4 2026 and a potential 2027 launch.
• Deal terms include USD 111 million upfront, up to USD 100 million in regulatory milestones, with closing expected in late Q3 or early Q4 2026.

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