Common Stock, $0.001 par value per share
- Symbol
- PTCT
- Exchange
- NASDAQ
- Classification
- COMMON
- Status
- Current
Current Report · Items 7.01, 8.01, 9.01 · 8-K
PTC Therapeutics, Inc.
PTCTNASDAQEQUITYCurrent
Regulation FD Disclosure · Other Events
Item 7.01. Regulation FD Disclosure. On September 21, 2026, PTC Therapeutics, Inc. (the “Company”) issued a press release announcing that it completed the previously announced agreement with Sangamo Therapeutics, Inc. (“Sangamo”) to acquire ST-920, a BLA-stage one-time administered AAV gene therapy product candidate for Fabry disease, (the “Acquisition”).…
Company context
PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company’s strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders.
Current securities
Registered securities in this filing
PTC THERAPEUTICS, INC. · 8-K · Filed 2026-09-21
Disclosure sections
Item 7.01Item 7.01 - Regulation FD Disclosure
Item 7.01. Regulation FD Disclosure.
On September 21, 2026, PTC Therapeutics, Inc. (the “Company”) issued a press release announcing that it completed the previously announced agreement with Sangamo Therapeutics, Inc. (“Sangamo”) to acquire ST-920, a BLA-stage one-time administered AAV gene therapy product candidate for Fabry disease, (the “Acquisition”). The press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K (this “Report”) and is incorporated by reference into this Item 7.01.
The information in this Item 7.01, including Exhibit 99.1, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing. All website addresses given in this Report or incorporated herein by reference are for information only and are not intended to be an active link or to incorporate any website information into this Report.
Item 8.01Item 8.01 - Other Events
Item 8.01. Other Information.
On September 21, 2026, the Company issued a press release announcing it completed the Acquisition. As previously disclosed, a rolling biologics license application (“BLA”) submission to the U.S. Food and Drug Administration for accelerated approval of ST-920 is expected to be completed in the fourth quarter of 2026. The BLA is based on the evidence of meaningful favorable clinical effect on renal function and safety and tolerability profile over 52 weeks in the Phase 1/2 STAAR study of ST-920. The Company expects to also pursue regulatory approval of ST-920 outside of the United States.
Filed exhibits (1)
Exhibit 99.1 PTC Completes Acquisition of ST-920 Fabry Disease Gene Therapy - Completion of BLA submission expected in Q4 2026 - WARREN, N.J., Sept. 21, 2026 - PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced that it completed the previously announced agreement with Sangamo Therapeutics, Inc. to acquire ST-920 - a BLA-stage, one-time administered AAV gene therapy for Fabry disease. A rolling BLA submission to FDA for accelerated approval of ST-920 is expected to be completed in Q4 2026. “Our team looks forward to completing the ST-920 BLA submission and potentially bringing a one-time administered, safe and effective durable disease treatment that addresses the limitations of chronic enzyme replacement therapy to the Fabry community,” said Matthew B. Klein, M.D., Chief Executive Officer. About ST-920 ST-920 or isaralgagene civaparvovec is a one-time administered AAV gene therapy product candidate for the treatment of Fabry disease. In clinical studies, isaralgagene civaparvovec enabled long-term production of the deficient alpha-galactosidase A (α-Gal A) enzyme and significant reduction in globotriaosylceramide (Gb3) levels with durable clinical benefit and reduction of…
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