Developing a Novel ATXN2-Targeting AAV Gene Therapy Treatment for Amyotrophic Lateral Sclerosis
Time: 11:00 am
day: Clinical Stream Day 1 AM
Details:
- Using human genetics, bioinformatics, and functional screens to identify a potent miRNA payload and an inert stuffer sequence
- Demonstrating preclinical efficacy in disease models of ALS
- Evaluating pharmacology, biodistribution in nonhuman primates in preparation for IND-enabling studies