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

Speakers: