--- title: "PTC completes acquisition of ST-920 Fabry gene therapy from Sangamo" url: "https://aibio.space/news/ptc-completes-acquisition-st-920-fabry-gene-therapy-sangamo/" publisher: "aibio.space (Biotech Investor Relations News)" date: "2026-09-21" section: "Company Updates" tickers: "NASDAQ:PTCT" source: "https://www.sec.gov/Archives/edgar/data/1070081/000107008126000023/tmb-20260921xex99d1.htm" summary: "PTC says the rolling BLA for ST-920 is expected to be completed in the fourth quarter of 2026, with the goal of accelerated approval." --- By the aibio.space news desk. Sources - [PTC Therapeutics, Inc. Exhibit 99.1 (2026-09-21)](https://www.sec.gov/Archives/edgar/data/1070081/000107008126000023/tmb-20260921xex99d1.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/) 2026-09-21 # PTC completes acquisition of ST-920 Fabry gene therapy from Sangamo Tickers [PTCT](https://aibio.space/companies/ptc-therapeutics/) PTC says the rolling BLA for ST-920 is expected to be completed in the fourth quarter of 2026, with the goal of accelerated approval. ## Key facts - **Company**: PTC Therapeutics, Inc. (NASDAQ: PTCT) - **News type**: Company Updates - **Primary source**: [PTC Therapeutics, Inc. Exhibit 99.1 (2026-09-21)](https://www.sec.gov/Archives/edgar/data/1070081/000107008126000023/tmb-20260921xex99d1.htm) *A compound microscope with its objective lenses. CC0, via rawpixel. The picture shows the field, not the companies named.* PTC Therapeutics, Inc. (NASDAQ: [PTCT](https://aibio.space/companies/ptc-therapeutics/)) said on September 21, 2026 that it completed its previously announced agreement with Sangamo Therapeutics, Inc. to acquire ST-920. ST-920 is a BLA-stage, one-time administered AAV gene therapy for Fabry disease. The company's release is datelined Warren, N.J. A rolling BLA submission to the FDA for accelerated approval of ST-920 is expected to be completed in the fourth quarter of 2026. Chief executive Matthew B. Klein, M.D., said the team looks forward to completing the BLA and to potentially bringing a one-time administered, durable treatment to the Fabry community, one that addresses the limits of chronic enzyme replacement therapy. ## What ST-920 is ST-920, also called isaralgagene civaparvovec, is a one-time administered AAV gene therapy. The release says that in clinical studies it enabled long-term production of the deficient alpha-galactosidase A (α-Gal A) enzyme and a significant reduction in globotriaosylceramide (Gb3) levels, with durable clinical benefit and a reduced burden of chronic enzyme replacement therapy (ERT). The FDA has granted Orphan Drug, Fast Track and RMAT designations to isaralgagene civaparvovec. It has also received Orphan Medicinal Product designation and PRIME eligibility from the European Medicines Agency, and the Innovative Licensing and Access Pathway from the U.K. Medicines and Healthcare products Regulatory Agency. ## About Fabry disease Fabry disease is a lysosomal storage disorder caused by mutations in the galactosidase alpha gene (GLA). The mutations lead to deficient alpha-galactosidase A activity, which is needed to metabolize globotriaosylceramide. Gb3 can build up in cells and cause serious damage to vital organs, including the kidney, heart, nerves, eyes, gut and skin. ## About PTC PTC describes itself as a global biopharmaceutical company focused on children and adults living with rare disorders. Its annual report says its commercial products include Sephience (sepiapterin) for phenylketonuria, which is approved in the United States, the European Economic Area, Japan and additional geographies. The company's Duchenne muscular dystrophy products are Translarna (ataluren) and Emflaza (deflazacort). Emflaza is approved in the United States for DMD in patients two years and older. The annual report also describes Upstaza and Kebilidi (eladocagene exuparvovec) for AADC deficiency, Tegsedi and Waylivra rights in Latin America and the Caribbean under an agreement with Ionis, and Evrysdi (risdiplam) for spinal muscular atrophy, developed with Roche. The annual report describes a splicing platform that includes votoplam for Huntington's disease, which PTC licensed to Novartis in November 2024. The release notes that PTC may have to make regulatory milestone payments on ST-920. The annual report also records a setback in its Duchenne franchise: in March 2025 the European Commission adopted the negative CHMP opinion not to renew Translarna's conditional marketing authorization for nonsense mutation Duchenne muscular dystrophy, although individual EU countries can allow continued commercial use. In May 2025 PTC announced that the Phase 2 study of votoplam met its primary endpoints of blood HTT lowering and safety. ## Financial position The annual report filed February 19, 2026 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 in 2025, compared with negative $107.7 million in 2024. The same filing reports year-end 2024 cash and cash equivalents of $779.7 million and short-term investments of $360.0 million. Operating cash flow turned from negative in 2024 to positive in 2025 alongside the swing to net income. The release does not state the price paid for ST-920. ## What comes next PTC expects to complete the rolling BLA for ST-920 in the fourth quarter of 2026. The release says there are no guarantees that ST-920 will receive regulatory approval. It gives no patient numbers, enzyme or Gb3 values, or safety data behind its claim of durable clinical benefit, and the BLA is not yet complete. Accelerated approval is the company's goal, not an FDA agreement, and PTC may owe regulatory milestone payments to Sangamo. [Report an error](https://aibio.space/reputation/#correction) ## Companies in this story PTCT PTC Therapeutics, Inc. 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Cite as: aibio.space, "PTC completes acquisition of ST-920 Fabry gene therapy from Sangamo", 2026-09-21, https://aibio.space/news/ptc-completes-acquisition-st-920-fabry-gene-therapy-sangamo/