b'12ABSTR ACT / BIOGR APHYAngela Cacace, Ph.D.Vice President of Neuroscience and Platform Biology, ArvinasApplication of Human Stem Cell Models for Drug Discoveryin Monogenic Neurologic DiseasesRecent advances in induced pluripotent stem cells and primary myoblasts have enabled modeling of human neurologic diseases with patient derived cell models. Utilization of iPSC-derived neurons and myoblasts enablesscreeningandevaluationofdrugefficacyaswellasdevelopmentofpatient stratification and efficacy biomarkers. These cells have the genetic backgroundofpatientsthatmorepreciselymodeldisease-specific pathophysiology and phenotypes. Neural cells derived from iPSCs and muscle cells derived from myoblasts can be produced in large quantity to enable drug discovery applications and facilitate translational research. Case studies will be presented for neurodegeneration (monogenic tauopathy, FTLD-tau), epilepsy disorders (Dravet Syndrome), neurodevelopmentaldisorders(FragileXSyndrome,FXS),andneuromusculardisorders (Facioscapulohumeral muscular dystrophy, FSHD).Dr. Cacace has more than two decades of bio-pharmaceutical research experience, contributing to four marketed drugs and over 18 development candidates. At Arvinas, she is leading the Neurology and Platform biology research efforts drivinginnovative expansion of the targeted degrader small molecule platform. Prior to joining Arvinas, Angela served as the Vice President of Biology at Fulcrum Therapeutics, where, together with her team, she built the rare disease biology platform and delivered their first development candidate for the treatment of FSHD and discovery efforts for SCD. Previously, she was the Director of Neuroscience and Genetically Defined Diseases at Bristol-Myers Squibb where she spearheaded the tau platform, alternative therapeutic modalities and was a coinventor on several development candidate patents for novel antisense oligonucleotide molecules. While at Bristol-Myers Squibb, Angela held leadership positions of increasingresponsibility, building research-wide teams including centralized High Throughput DMPK Profiling, the GPCR High Throughput Screening Team and the Cellular Resource Team.While serving as a Sr. Principal Scientist in Cancer Biology at Pfizer, she was responsible for the discovery of a novel anti-angiogenic antibody development candidate. Angela received herPh.D. in pharmacology from Columbia University and completed herpostdoctoral research in Oncology at Bristol-Myers Squibb and theNational Cancer Institute.'