Modeling Neurological Disorders Using Patient-Derived Human Brain Organoids

Time: 9:55 am
day: Day Two


  • CNS drug discovery needs a paradigm shift. Patient-derived brain organoids can revolutionize CNS drug discovery
  • Patient-derived Rett syndrome and CDKL5 deficiency disorder cortical brain organoids exhibit aberrant functional disease phenotypes amenable to HTS. Phenotypic screening identified clinical candidates for Rett syndrome advancing to a Phase 2 clinical trial funded by the DoD
  • Midbrain organoids from familial Parkinson’s disease patients with pathogenic mutations in the GBA1 gene exhibit clinical biomarkers amenable to HTS