NEMAMETRIX, INC — Department of Health and Human Services SBIR Phase I: NIA

Description

Byovermillion people in the USA will be diagnosed with Alzheimerandapos s related dementia at an annual treatment cost near $billionTo contain the escalation in healthcare costsnew treatments are needed to relieve the underlying genetic causes and risk factors leading to dementia onsetIn simple animal modelsdrugs have been found that lead to lifespan extensionYet the underlying genetic mechanisms as drug targets for Alzheimerandapos s related dementia remain elusiveIn this SBIR proposalhumanized animal models are created to model genetic disorders of the Frontotemporal DementiaFTDcomponent of Alzheimerandapos s Disease Related DementiasADRDFour FTD genes are targeted for humanizationMAPTGRNC orfand TARDBPin the Celegans animal modelThe procedure for humanization resulting gene specific replacement with human cDNAUsing a CRISPR based Gene swap techniquea sequence optimized human gene is inserted as a replacement of an orthologous gene in the genome of the animal modelEach humanized animal creates a platform for installing and profiling the functional consequence of pathogenic variantsThe variants causing deviant phenotypes are screened for the capacity of longevity associated molecules to restore wildtype phenotypeIf FTD model creation and compound screening reveal a restorative capacitythe assay system can then be used for discovery of novel therapeutic moleculesLeads discovered are commercialized by internal or externally licensed drug development into therapeutics for treating FTD disease Alzheimerandapos s related dementia will effect overmillion americans and cost near $billion year byNemametrix is developing humanized animal models to discover therapeutics for the Alzheimerandapos s Disease Related DementiasADRDof Frontotemporal DementiaFTDLeads discovered are developed for use by clinicians to treat patients with dementia