Reinventing Drug Discovery for Rare CNS Genetic Diseases

Time: 9:30 am
day: Day Two


  • 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 for clinical translation