Public Funding for Stratastem Limited

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Recent advances have deepened our understanding of the pathology of Alzheimer's disease and resulted in revised criteria, clinical trial assessment and disease management with a focus on early-stage manifestations of the disease. Using iPSC technology, StrataStem have derived pluripotent cells from clinically diagnosed fAD patients and differentiated these to neuronal networks using a rapid and novel proprietary method. We have established a number of bespoke _in vitro_ cellular assays to detect specific phenotypes typical of preclinical AD neuronal pathology. Of significant importance, our analysis has demonstrated that discrimination (stratification) between specific gene mutations is possible, even within neuronal networks exhibiting an **immature** foetal phenotype. In addition, our novel network analysis approach predicts several key causal upstream regulatory pathways that are not currently associated with AD and suggests that the later stage of amyloid deposits and Tau protein tangles are a very late stage **effect**, and not a cause, of the disease. Therefore, we believe that AD results from abnormal cellular processes starting within the foetus and occurring throughout life, eventually manifesting as an observable phenotype in the older patient. Our paradigm shift in AD pathology cause-effect may explain why drugs which target late stage effects of the disease are ineffective in clinical trials. In this project we will build upon our novel and exciting results by deriving iPSCs from clinically diagnosed sporadic late-onset AD patients (representing approximately 95% of all AD cases), with full clinical and lifestyle histories, to determine AD-associated phenotypes and use RNA-seq analysis to allow phenotype-genotype stratification compared to healthy age-and gender-matched donors. This funding will allow us to generate a patient biobank with anonymised clinical, lifestyle, genetic and phenotypic information that will be the key product for our future growth to provide an intelligent screening model for compound discovery that enables the **right drug** to be given to the **right patient** at the **right time**. In the longer term, this will deliver significant social and economic benefits to society and facilitate earlier intervention and life planning for individuals with this devastating disease.