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

Reuters2026/08/13 11:06

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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.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. PTC Therapeutics Inc. published the original content used to generate this news brief via EDGAR, the Electronic Data Gathering, Analysis, and Retrieval system operated by the U.S. Securities and Exchange Commission (Ref. ID: 0001104659-26-095424), on August 13, 2026, and is solely responsible for the information contained therein.

Disclaimer: The content of this article solely reflects the author's opinion and does not represent the platform in any capacity. This article is not intended to serve as a reference for making investment decisions.

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

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