Delivering probes and clinical candidates to test precision medicine therapeutic hypotheses

Abstract Background . There is a need for selective clinical candidates that allow testing of explicit precision medicine hypotheses emerging from diverse data acquisition, analysis and archiving initiatives. Candidates target pathophysiology progression mechanisms would offer the potential disease modification. Appropriate would, ideally, facilitate hypothesis in order to find right drug at dose time patient. In this regard, we developed discovery development platform focused on altering delivers novel small molecule with good bioavailability, safety profiles evaluation proof‐of‐concept trials. Method Synaptic dysfunction neuroflammation are mechanisms. Our starts molecular scaffolds pharmacoinformatic prior CNS drugs their utility. Medicinal chemistry diversification driven by chemoinformatics related properties biological systems pharmacoinformatics pharmacology toxicology potential. Campaigns can single entities (e.g., serine/threonine protein kinase), pathways cytokine biosynthesis dysfunction) or combinations serotonin receptor linked dysfunction). Efficient medicinal approaches include diversifications based [Target:Inactive Molecular Fragment] complexes us SOSA variations (systematic optimization side activities). Best‐in‐class emerge IND‐enabling preclinical tractability GMP production delivery. Results MW150, MW189 MW151 example deliverables continue exhibit utility human subjects. MW150 highly S/T kinase inhibitor deliverable entering phase 2a trials AD patients. exhibited selectivity hierarchal kinome‐wide large scale functional GPCR screens absence activity standardized ADMET liabilities. Preclinical according FDA/ICH guidelines provided foundation regulatory approval trial initiation. stage where design synthesis curated chemical library was rationale vetting attenuation dysfunction. Conclusion Overall, continues be validated delivery studies.