uniQure N.V. announced on January 30, 2025, that the Independent Data Monitoring Committee (IDMC) for its Phase I/II EPISOD1 clinical trial of AMT-162 for SOD1-ALS has reviewed 28-day safety data from the first study cohort. The IDMC identified no significant safety concerns, providing a positive assessment for the investigational gene therapy.
Following this favorable review, the IDMC recommended proceeding with enrollment in the second cohort of the trial. uniQure expects to initiate enrollment of this second dose cohort in the first quarter of 2025, marking a meaningful step in the clinical development of AMT-162.
AMT-162 is an AAVrh10-based gene therapy designed to knock down the expression of the mutated SOD1 protein, which is implicated in the progression of ALS. The ability to safely escalate doses is critical for determining the optimal therapeutic regimen and advancing this potential one-time treatment for a fatal neurodegenerative disease.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.