Drug discovery

Drug discovery is a long process which can be divided into different phases. These range from early drug discovery which includes target identification and validation, compound screening, and lead optimization, through pre-clinical research to establish safety and efficacy, and finally clinical testing and regulatory approval. The process can take 12-15 years per new drug molecule and is costly. New small molecules are typically identified by high throughput screening (HTS) of large libraries containing millions of compounds, with hit compounds optimized by medicinal chemistry methods. Artificial intelligence (AI) and machine learning algorithms are key developments in drug discovery, to help design and optimize new molecules, predict protein shapes, interpret complex responses in phenotypic assays, and deconvolve genetic pathways. The availability of high-resolution cryo-EM protein structures and drug-binding sites facilitates structure-based drug design. Finally, a better translation of preclinical drug candidates to the clinic is possible using human patients and disease iPSC 2D cultures and 3D organoids, and the availability of human donor tissue. This also raises the possibility of personalized medicine, to identify patient-specific drug regimes.