--- title: "PTC named winning bidder for Sangamo's Fabry gene therapy ST-920" url: "https://aibio.space/news/ptc-named-winning-bidder-sangamos-fabry-gene-therapy-st-920/" publisher: "aibio.space (Biotech Investor Relations News)" date: "2026-08-12" section: "Distress" tickers: "NASDAQ:PTCT" source: "https://www.sec.gov/Archives/edgar/data/1070081/000110465926095424/tmb-20260812xex99d1.htm" summary: "PTC says the ST-920 rolling BLA will be completed in the fourth quarter of 2026, with a potential commercial launch in 2027 if the acquisition closes." --- By the aibio.space news desk. Sources - [PTC Therapeutics, Inc. 8-K (2026-08-13)](https://www.sec.gov/Archives/edgar/data/1070081/000110465926095424/tmb-20260812xex99d1.htm) - [PTC Therapeutics, Inc. 10-K (2026-02-19): business](https://www.sec.gov/Archives/edgar/data/1070081/000110465926017575/tmb-20251231x10k.htm) - [PTC Therapeutics, Inc. financial data (SEC XBRL)](https://www.sec.gov/Archives/edgar/data/1070081/000110465926017575/) [PTCT](https://aibio.space/companies/ptc-therapeutics/) 2026-08-12 # PTC named winning bidder for Sangamo's Fabry gene therapy ST-920 Tickers [PTCT](https://aibio.space/companies/ptc-therapeutics/) PTC says the ST-920 rolling BLA will be completed in the fourth quarter of 2026, with a potential commercial launch in 2027 if the acquisition closes. ## Key facts - **Company**: PTC Therapeutics, Inc. (NASDAQ: PTCT) - **News type**: Distress - **Primary source**: [PTC Therapeutics, Inc. 8-K (2026-08-13)](https://www.sec.gov/Archives/edgar/data/1070081/000110465926095424/tmb-20260812xex99d1.htm) *A supreme court building with lit columns. CC0, via rawpixel. The picture shows the field, not the companies named.* On August 12, 2026, PTC Therapeutics, Inc. said it was selected as the winning bidder to acquire ST-920 from Sangamo Therapeutics in a competitive bankruptcy auction. The terms include $111 million upfront and up to $100 million in contingent milestone payments based on certain regulatory approvals. The announcement was made in a [press release](https://www.sec.gov/Archives/edgar/data/1070081/000110465926095424/tmb-20260812xex99d1.htm) furnished on Form 8-K. ## The deal and the ST-920 program ST-920 is a one-time administered AAV gene therapy for Fabry disease, a rare, inherited lysosomal storage disorder caused by mutations in the GLA gene. The disorder leads to deficiency of the alpha-galactosidase A enzyme. It is estimated that there are 11,000 people living with Fabry disease in the United States. The company says ST-920 enables long-term production of the deficient enzyme and a significant reduction in globotriaosylceramide (Gb3) levels. PTC chief executive Matthew B. Klein, M.D., said the deal "advances our strategy of leveraging our accomplished existing rare disease global commercial infrastructure to accelerate short- and intermediate-term revenue growth." The company said the transaction did not need any development or commercial build, and that it would not affect its objective of reaching cashflow break even in 2026. ST-920 has Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug and Fast Track designations from the FDA. The company says the Phase 1/2 STAAR study showed a positive mean annualized estimated glomerular filtration rate (eGFR) slope at 52 weeks after administration, along with evidence of favorable effects on cardiac function and quality of life. The release gives no patient numbers, slope values or safety events, and it does not describe a control group. All study participants who were on enzyme replacement therapy at the start were withdrawn from it. The company reports durability of up to 4.5 years for the earliest treated participant, and says there is no requirement for routine prophylactic or post-infusion systemic immunosuppressive agents. ## Regulatory path The BLA for accelerated approval is based on the intermediate clinical endpoint of annualized eGFR at Week 52, which the company says is aligned with the FDA. That is the company's statement; the FDA has not approved anything, and accelerated approval rests on a surrogate endpoint with confirmatory data still to come. The 104-week results from the STAAR study are planned to provide confirmatory evidence for traditional approval. The nonclinical and clinical modules of the BLA have already been submitted as part of a rolling submission, and the CMC package is expected in Q4 2026. The company will also pursue regulatory approval outside the United States. The acquisition remains subject to definitive documentation, bankruptcy court approval, antitrust review and other customary closing conditions. PTC expects the deal to close in late Q3 or early Q4 2026. ## Financial position The financial figures in this story come from the annual report filed on February 19, 2026. PTC reported research and development expense of $455.2 million for 2025, compared with $534.5 million for 2024. It reported net income of $682.6 million for 2025, compared with a net loss of $363.3 million for 2024. Cash from operations was $711.2 million for 2025, compared with negative $107.7 million for 2024. Year-end 2024 cash and cash equivalents were $779.7 million, with short-term investments of $360.0 million. ## The business and what comes next PTC describes itself as a global biopharmaceutical company focused on rare disorders. Its marketed products include Sephience for phenylketonuria, Translarna and Emflaza for Duchenne muscular dystrophy, Kebilidi for AADC deficiency, and Evrysdi for spinal muscular atrophy, which is developed with Roche. The acquisition would add a Fabry program to that portfolio. Before closing, the definitive agreement must be signed, the bankruptcy court must approve the deal and antitrust review must be completed. The CMC package is expected in Q4 2026, and the 104-week STAAR results are planned as confirmatory evidence. [Report an error](https://aibio.space/reputation/#correction) ## Companies in this story PTCT PTC Therapeutics, Inc. NASDAQ ยท United States Open the starter desk ## More in Distress Distress ### [Mineralys takes ownership of lorundrostat patents but keeps Tanabe milestone debts](https://aibio.space/news/mineralys-takes-ownership-lorundrostat-patents-but-keeps-tanabe/) Oct 29, 2026 Distress ### [Bristol Myers Squibb drops Prothena's PRX019, ending up to $617.5 million in milestones](https://aibio.space/news/bristol-myers-squibb-drops-prothenas-prx019-ending-up-617-5-million/) Oct 29, 2026 Distress ### [LENZ Therapeutics reorganizes to cut 2027 operating expenses by more than 50%](https://aibio.space/news/lenz-therapeutics-reorganizes-cut-2027-operating-expenses-more-50/) Oct 28, 2026 --- Source page: https://aibio.space/news/ptc-named-winning-bidder-sangamos-fabry-gene-therapy-st-920/. Facts in aibio.space stories are checked against company filings and releases; opinion is labelled. Cite as: aibio.space, "PTC named winning bidder for Sangamo's Fabry gene therapy ST-920", 2026-08-12, https://aibio.space/news/ptc-named-winning-bidder-sangamos-fabry-gene-therapy-st-920/