Advancing a Splice-Modulating ASO Restoring UNC13A protein for ALS: Nonclinical Studies Guided Optimization of the Clinical Trial Design
Time: 11:00 am
day: Day 2
Details:
- Nonclinical approach to support clinical trials with a splice modulating ASO designed to correct UNC13A mis-splicing associated with TDP-43 pathology in ALS
- Learnings from nonclinical studies and the path toward initiating first-inhuman trials within the year
- Impact of regulatory feedback on nonclinical strategy and the clinical trial design
- Optimizing clinical trial design and dosing strategy to maximize likelihood of therapeutic impact
