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Investing.com -- Shares of Voyager Therapeutics (NASDAQ:VYGR) plummeted 20% as the company announced a shift in its gene therapy program for amyotrophic lateral sclerosis (ALS), which will involve assessing alternate payloads. This adjustment is expected to push the company’s cash runway into mid-2027.
The biotechnology firm, which specializes in neurogenetic medicines, revealed that the siRNA payload component of its development candidate VY9323 did not meet the desired standards due to an off-target effect that narrowed the therapeutic window. As a result, the investigational new drug (IND) application for VY9323, previously slated for mid-2025, will be delayed as the company seeks an alternative payload.
Voyager’s CEO, Dr. Alfred W. Sandrock, Jr., expressed disappointment in the development candidate VY9323 not advancing but remained hopeful for identifying a new payload and continuing the program. He also highlighted the consistent performance of the company’s novel TRACER capsids across multiple programs, which he believes could transform gene therapy for central nervous system (CNS) diseases.
Despite the setback with VY9323, Voyager Therapeutics anticipates IND filings in 2025 for gene therapy candidates targeting GBA1 Parkinson’s and other GBA1-mediated diseases, as well as Friedreich’s ataxia, in partnership with Neurocrine (NASDAQ:NBIX) Biosciences. Additionally, an IND filing for VY1706 is expected in 2026.
The company’s decision to reassess the VY9323 program does not affect its other gene therapy programs. Further updates on the expected timing for the SOD1 ALS program will be provided when appropriate.
Voyager Therapeutics will be presenting additional data at the Oppenheimer 25th Annual Healthcare Life Sciences Conference, and the presentation will be available on the company’s website for at least 90 days following the event.
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