Identifying in vivo molecular targets of small
molecular and protein drugs can provide very important information
about the drug candidate, and is often a critical step in lead
validation.
For a lead or drug candidate that is discovered
from a cell-based or phenotype-based assay, identifying its molecular
targets and mechanism of action is an essential step to further
improve the efficacy and toxicity profiles of the drug molecule. With
known drug targets, scientists can use molecular assay or structure
based drug design to optimize the lead.
For a lead or candidate that is discovered via an
in vitro high throughput screening, identifying its in vivo
molecular targets provides an important approach to confirm and
validate that mechanism of drug action. It also provides information
about “off-targets”, which may be used to infer side effect or
even toxicity.
For a marketed drug, identifying its in vivo targets
may provide important clues about other novel therapeutic applications
of the drug, which is an important and effective technique in drug
lifecycle management.
Drug
Targets Identification and Validation
