iPSC-Derived Neuronal Models of Alzheimer's Disease for Phenotypic Drug Screening

PROJECT SUMMARY Alzheimer’s disease (AD), a progressive late onset neurodegenerative disease, represents a serious challenge for the development of novel drugs. There have been no new AD drugs since 2003, a growing number of researchers and stakeholders are proposing that AD drug discovery modeling requires a refocus. 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. Therefore, the overall goal of this phase II SBIR project is to further develop, validate, and commercialize an in vitro high throughput, high content phenotypic AD disease modeling called the “PhenoFingerprint platform (PFP)”. This Phenovista proprietary PTP will be 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 for AD drug discovery and development.PROJECT NARRATIVE As the world’s aging population grows, patients with late onset diseases like Alzheimer’s Disease (AD) are also growing in incidence. AD therapeutics remains an elusive area for drug discovery and development. A successful development and commercialization of next-generation, low-cost, high-performance technology for predicting the effects of investigational drugs on human neurons, a critical cell type in the brain, would help improve the development process for new Alzheimer’s therapies. Therefore, the overall goal of this PHASE III SBIR project is to develop, validate, and commercialize an in vitro high content phenotypic AD model called PhenoFingerprint Platform (PFP). This Phenovista’s proprietary PFP will be a next-generation in vitro system for ranking the relative efficacy of AD therapeutics, and this new model system will be incorporated as a part of the regulatory framework for therapeutic commercialization.