Target-Based Drug Designing
Nabanita Mandal, Soumya Lipsa Rath · Apple Academic Press eBooks · 2024
Typically, a disease or disorder in the body occurs due to deregulation of a biomolecule. When a drug is developed against such defined targets, such as gene or protein, it is known as target-based drug discovery. With the advent of high throughput, genome sequencing, and virtual screening, one can easily sequence the diseased gene and identify the target protein or enzyme which impairs the normal metabolic pathway in an organism. These targets are identified, validated, and processed with in-vitro assays. In recent times, it has been possible to screen millions of small molecules against an identified target by high throughput screening of small molecules. The molecules might be either novel compounds, plant or microbial derivatives, or existing FDA-approved drugs (that are used for drug repurposing). The identified “hits” are further optimized using bioinformatics tools, for generating optimal leads. These leads are then selected for further experimentation, preclinical, and clinical trials. The process is very useful since it saves a lot of time and finance incurred in laboratory experiments considering the large number of small molecule inhibitors which are available. Thus, target-based drug discovery entails a very crucial part in the drug discovery process. Here, in this chapter, we describe in detail the background for identification of target, screening, lead optimization, and validation with special emphasis on existing software and exemplifying the process by giving the readers existing success stories in this field.