Therapeutics and diagnostics discovery

Abstract

In any therapeutics discovery and development effort, multiple critical steps must be accomplished to arrive at a compound that is safe and efficacious, while also exhibiting the complex array of desired drug-like behaviors that warrant advancement to the clinic. These tasks include target identification and validation, screening for active compounds, chemical modification of candidate compounds to achieve optimized pharmacology, formulating the final drug product, and establishing efficacy and safety in appropriate preclinical models. New drug discovery tools and novel drug modalities have evolved in recent years, including cell and gene therapies; many of them made their first steps in academia. "Repurposing" drugs can provide a faster, less risky, and more cost-effective route to bring a new therapy to patients. Preventive vaccines have a tremendous impact on human health, but are generally administered to healthy people and must be exceptionally safe. Finally, biomarker and diagnostic discovery provides important tools for clinical practice and importantly, contributes to effective drug development.