This STTR Phase I Project Grant award from the National Institute on Aging (NIA), under the Aging Research federal grant program (CFDA 93.866), provides $499,883 to Degrome Therapeutics, Inc. to develop small-molecule degraders of the amyloid precursor protein (APP) as potential first-in-class drugs for Alzheimer's disease (AD) therapy.
The key objectives are to optimize the potency, solubility, and blood-brain barrier permeability of the lead APP degrader compounds through computational chemistry approaches and iterative compound testing. The research will validate the activity of the top-ranked compounds in reducing APP and amyloid-beta levels in induced pluripotent stem cell models derived from AD patients. The most promising compounds will then undergo pharmacokinetic and therapeutic evaluation in AD mouse models to determine if they can improve learning and reduce APP, amyloid-beta, and amyloid plaques in the brain. This one-year project aims to provide proof-of-concept for using small-molecule APP degraders as a novel approach to treat Alzheimer's disease.
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