Advancing AMT‑162 Gene Therapy for SOD1‑ALS Clinical Progress & Future Opportunities
- Overview of AMT‑162, a one-time intrathecal gene therapy designed to silence SOD1 expression in patients with
- SOD1‑linked ALS and the rationale for targeting this mutation
- Early clinical progress from the Phase I/II EPISOD1 trial, including first patient dosing and favorable independent safety data supporting progression to the second dose cohort
- Key exploratory efficacy and biomarker strategies in the AMT‑162 program, including use of neurofilament light and SOD1 protein levels to assess potential impact on disease progression
- Challenges and opportunities in gene therapy for ALS, including regulatory considerations, patient selection, and the potential for one-time treatment paradigms in rare genetic subtypes