Reinventing Drug Discovery for Rare CNS Genetic Diseases

Time: 2:30 pm
day: Track A - Day 1 PM


  • Leveraging patient-derived organoid models of human brain disease, scaled biology, and machine learning to drive CNS drug discovery
  • Utilizing patient-derived organoids to elucidate disease pathophysiology; to formulate key therapeutic hypotheses; and to identify and validate drug targets, cellular assays and biomarkers to guide clinical candidate selection
  • Advantages of human-first proof of efficacy obtained from brain organoid models to improve clinical translation