Voyager Therapeutics Halts ALS Gene Therapy Development Due to Off-Target Effects
Voyager Therapeutics has suspended development of VY9323, their gene therapy candidate for SOD1 ALS13.
The decision was based on emerging preclinical data showing off-target effects from the siRNA payload component13.
The off-target effect resulted in a narrowed therapeutic window for the drug13.
Voyager plans to assess alternate payloads while keeping the novel capsid component unchanged13.
This development delays the expected timeline for Investigational New Drug (IND) application filing, previously planned for mid-202513.
The delay extends Voyager's cash runway into mid-20273.
Other Voyager programs, including those for GBA1 Parkinson's disease and Friedreich's ataxia, remain on track with expected IND filings in 20251.
The company emphasizes the importance of maintaining high standards in drug development, despite the disappointment of this setback13.
This situation highlights the ongoing challenges in developing gene therapies, particularly in managing off-target effects28.
The incident underscores the critical nature of thorough preclinical testing in gene therapy development to ensure safety and efficacy25.
Sources:
1. https://ir.voyagertherapeutics.com/news-releases/news-release-details/voyager-provides-update-sod1-als-gene-therapy-program
2. https://www.frontiersin.org/journals/bioengineering-and-biotechnology/articles/10.3389/fbioe.2023.1339189/full
3. https://www.fiercebiotech.com/biotech/journey-voyagers-als-gene-therapy-clinic-hindered-need-find-alternative-payload
5. https://blog.addgene.org/crispr-101-off-target-effects
8. https://www.scendea.com/articles/blog-post-title-two-8e2bx