From Target Validation Through Nonclinical Development to First-in-Human: Lessons Learned Developing an ASO Therapy for Most People Living with ALS
- Key learnings from nonclinical development of an ASO intended for most people with ALS, including validation of potency, durability, and CNS biodistribution
- How the candidate ASO nonclinical profile informed dosing strategy, injection frequency, and the Phase 1/2 trial design
- Insights from regulatory interactions shaped by the unique profile of the candidate ASO guided the overall clinical development plans.