This technology is a comprehensive compound design and preclinical testing platform that can be used to develop multifunctional drugs for Alzheimer’s disease, and has already led to the development of several promising lead compounds.
Many diseases including Alzheimer’s disease are multifaceted and likely require interventions in several cellular processes. However, current drug discovery and testing pipelines focus on single targets and rely on costly and reductionist in vitro assays. Using rodents in pre-clinical tests are costly.
This customizable platform establishes and screens drug candidates for a multi-target directed ligand Alzheimer’s treatment approach, based on in vitro and in vivo efficacy measures. The platform has led to the identification of several promising and preclinically verified lead compounds, ready for further development as Alzheimer’s therapeutics. Combining organic synthesis with an in vitro pharmacophore iteration model enables cytotoxicity studies, membrane permeability readouts, among other pharmacodynamic outcomes. These methods are complemented by the inclusion of rapid, large-scale preclinical animal testing. A humanized zebrafish model, harboring amyloid and tau toxicity with humanized genetic alterations associated with Alzheimer’s disease can be used in a modular manner to assess the efficacy of lead compounds. Single cell transcriptomics, histopathological assessment, cognitive behavioral tests are used to measure synaptic protection, immunomodulatory activities, vascular integrity and neurogenesis.
This technology has been validated through the discovery of several promising lead candidate compounds for multi-target Alzheimer’s treatment.
IR CU23156
Licensing Contact: Joan Martinez
