Target Identification & Validation for Early Drug Discovery

Early stages of drug discovery start with initial steps of target identification and moves to the later stages of lead optimization. Multiple sources including academic research, clinical works and commercial sector help in the identification of a suitable disease target. The chosen target is then used by the pharmaceutical industry and more recently by some academic centers to identify molecules for making acceptable drugs. The process involves various early steps.

Early Drug Discovery

1. Target Identification & Validation

Target Identification & Characterization

Target identification and characterization begins with identifying the function of a possible therapeutic target (gene/protein) and its role in the disease. Identification of the target is followed by characterization of the molecular mechanisms addressed by the target. A good target should be efficacious, safe, meet clinical and commercial requirements and be ‘druggable’.


  • Data mining using bioinformatics
        — identifying, selecting and prioritizing potential disease targets
  • Genetic association
        — genetic polymorphism and connection with the disease
  • Expression profile
        — changes in mRNA/protein levels
  • Pathway and phenotypic analysis
        — In vitro
    cell-based mechanistic studies
  • Functional screening
        — knockdown, knockout or using target specific tools
Tools for Target Identification & Validation
Disease association (genetics and expression changes)
Bioactive molecules
Cell based models
Protein interactions (pull-down assays, yeast 2 hybrid)
Analysis of signaling pathways
Functional analysis (overexpression, transgenics, antisense RNA, gene variants)

Target Validation

Target Validation shows that a molecular target is directly involved in a disease process, and that modulation of the target is likely to have a therapeutic effect. The most important criteria for target validation is to take multi-validation approach.


  • Genetic manipulation of target genes (in vitro)
        — knocking down the gene (shRNA, siRNA, miRNA), knocking out the gene (CRISPR, ZFNs), knocking in the gene
             (viral transfection of mutant genes)
  • Antibodies
        — interacting to the target with high affinity and blocking further interactions
  • Chemical genomics
        — chemical approaches against genome encoding protein

Is your lab having difficulty discovering novel targets? Our broad portfolio of assays, reagents, and libraries can help you find the right lock so you can begin the work to unlock it. From the Sanger Whole Genome CRISPR Library to Duolink® PLA to measure protein-protein interactions, and bioactive small molecules, we provide you with the right tools to enable the identification, validation, and characterization of the novel targets you’re looking for.

Products supporting Target Identification, Characterization and Validation

Antibodies Primary, secondary and supplementary products for WB, IHC, IP, ELISA, ChIP etc.
Bioactive Small Molecules Biologically active drug-like molecules (non-protein, naturally-occurring or synthetic compounds) for target identification and validation
Cell Design Studio™ Engineering model cell lines for high content analysis, high throughput screening, target validation and other cell-based assays
CRISPR CRISPR plasmids, lentivirus, paired nickases, guide RNA, Cas9 mRNA, pooled screening libraries and our exclusive Sanger-arrayed lentiviral screening library
Duolink® Proximity Ligation Assay (PLA®) Detect, quantify and visualize protein-protein interactions, single target protein expression levels and post translational modification events.
Early ADME-Tox Innovative genetically engineered cell lines and validated assays designed to improve drug metabolism, transporter and safety testing, as well as reagents, consumables and labware
Flow Cytometry Characterization of cells by looking at cell surface markers (ie. biomarkers, fluorescently labeled proteins, etc).
Immunoassay Platforms Ultrasensitive detection with Singulex® assay, maximum throughput with Gyrolab® workstation, robust ELISAs or profiling data with multiplex panel using Luminex® technology
Lentiviral Pools Knockdown (RNAi), Knockout (CRISPR) and Express (LentiORF)
MISSION® TRC3 LentiORF Whole genome expression library of Human Open Reading Frames
Nucleic Acid Purification Kits and reagents for all nucleic acid purification needs
RNAi Arrayed Libraries Leading functional genomics technologies including RNA interference (RNAi) tools for gene silencing (siRNA, esiRNA, shRNA, miRNA) and systems for gene editing and gene knockout



  1. Hughes, James P., et al. "Principles of early drug discovery." British journal of pharmacology 162.6 (2011): 1239-1249.
  2. Kuhn, Max, Ian Peers, and Stan Altan. Nonclinical statistics for pharmaceutical and biotechnology industries. Springer, 2016.
  3. Gashaw, Isabella, et al. "What makes a good drug target?. Drug discovery today 17 (2012): S24-S30.



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