Drug Discov Today. 2018 May 11. pii: S1359-6446(18)30040-0. doi: 10.1016/j.drudis.2018.05.015. [Epub ahead of print]
Next-generation sequencing in drug development: target identification and genetically stratified clinical trials.
Next-generation sequencing (NGS) enabled high-throughput analysis of genotype-phenotype relationships on human populations, ushering in a new era of genetics-informed drug development. The year 2017 was remarkable, with the first FDA-approved gene therapy for cancer (Kymriah™) and for inherited diseases (LUXTURNA™), the first multiplex NGS panel for companion diagnostics (MSK-IMPACT™) and the first drug targeting a genetic signature rather than a disease (Keytruda®). We envision that population-scale NGS with paired electronic health records (EHRs) will become a routine measure in the drug development process for the identification of novel drug targets, and that genetically stratified clinical trials could be widely adopted to improve power in precision-medicine-guided drug development.
Next-generation sequencing; drug discovery; genetically stratified clinical trial; target identification