iPSC Derived Neuronal Models of Alzheimer s Disease for Drug Screening

PROJECT SUMMARY Central nervous system CNS diseases including Alzheimerandapos s disease AD represent a daunting challenge for the development of novel drugs The successful development and commercialization of next generation low cost high performance technology for modeling both disease biology and the effects of investigational drugs on neurons would help improve the development process for new therapies thereby reducing national healthcare costs and improving patient outcomes throughout the nation The ability to prepare disease models from stable reproducible neuronal cultures in vitro would allow screening and prioritization of further testing in a rapid and cost effective manner Ideally this system could be expanded to incorporate disease models that would mimic in vivo as well as clinical findings Therefore the overall goal of this multi phase SBIR project is to develop validate and commercialize an in vitro high throughput high content phenotypic neuronal assay for AD to allow Phenovistaandapos s proprietary neuronal culture screening system a next generation in vitro system for ranking the relative efficacy and toxicity of CNS therapies to be incorporated as a part of the regulatory framework for therapeutic commercialization PROJECT NARRATIVE Alzheimerandapos s Disease represents an underserved area for drug discovery Successful development and commercialization of next generation low cost high performance technology for predicting the effects of investigational drugs on iPSC derived neurons a critical cell type in the brain would help improve the development process for new Alzheimerandapos s therapies The complexity of the human brain has made it difficult to study many brain disorders in model organisms highlighting the need for an in vitro model of human brain development that uses human neurons Therefore the overall goal of this multi phase SBIR project is to develop validate and commercialize an in vitro high throughput high content phenotypic neuronal assay using Phenovistaandapos s proprietary neuronal culture screening system as a next generation in vitro system for ranking the relative efficacy of CNS therapies and to have this assay incorporated as a part of the regulatory framework for therapeutic commercialization