Leveraging Accurate Human Disease Models for Target Validation & Drug Optimization to Rapidly Advance Novel & Effective Treatments for ALS

Time: 11:45 am
day: Day One AM


  • Discovering novel and broadly effective therapeutic targets, such as PIKFYVE and SYF, through unbiased screening in sporadic ALS patient models
  • Optimizing ASO drug candidates’ potency to reverse aberrant UNC13A splicing in ALS patient models
  • Investigating safety, potency and biodistribution of lead ASO candidate AS-202, a near-to-clinic PIKFYVE suppressing ASO