Next-generation sequencing in drug development: target identification and genetically stratified clinical trials

Drug Discov Today. 2018 Oct;23(10):1776-1783. doi: 10.1016/j.drudis.2018.05.015. Epub 2018 May 24.

Abstract

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.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Clinical Trials as Topic / methods
  • Drug Development / methods*
  • Genotype
  • High-Throughput Nucleotide Sequencing / methods*
  • Humans
  • Molecular Targeted Therapy*
  • Phenotype
  • Precision Medicine / methods