Skip to main content

Thank you for visiting You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Opinion
  • Published:

The druggable genome


An assessment of the number of molecular targets that represent an opportunity for therapeutic intervention is crucial to the development of post-genomic research strategies within the pharmaceutical industry. Now that we know the size of the human genome, it is interesting to consider just how many molecular targets this opportunity represents. We start from the position that we understand the properties that are required for a good drug, and therefore must be able to understand what makes a good drug target.

This is a preview of subscription content, access via your institution

Access options

Buy this article

Prices may be subject to local taxes which are calculated during checkout

Figure 1: Drug-target families.
Figure 2: Number of drug targets.
Figure 3: Novel drug launches.
Figure 4: Marketed small-molecule drug targets by biochemical class.
Figure 5: Exploitation of the genome, as measured by known leads.

Similar content being viewed by others


  1. Lipinski, C., Lombardo, F., Dominy, B. & Feeney, P. Experimental and computational approaches to estimate solubility and permeability in drug discovery and development settings. Adv. Drug Deliv. Rev. 23, 3–25 (1997).

    Article  CAS  Google Scholar 

  2. Drews, J. Genomic sciences and the medicine of tomorrow. Nature Biotechnol. 14, 1516–1518 (1996).

    Article  CAS  Google Scholar 

  3. Drews, J. & Ryser, S. Classic drug targets. Nature Biotechnol. 15, 1318–1319 (1997).

    CAS  Google Scholar 

  4. Drews, J. Drug discovery: a historical perspective. Science 287, 1960–1964 (2000).

    Article  CAS  PubMed  Google Scholar 

  5. Bailey, D., Zanders, E. & Dean, P. The end of the beginning for genomic medicine. Nature Biotechnol. 19, 207–209 (2001).

    Article  CAS  Google Scholar 

  6. Apweiler, R. et al. The InterPro database, an integrated documentation resource for protein families, domains and functional sites. Nucleic Acids Res. 29, 37–40 (2001).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

  7. Proteome Analysis Database [online], (version analysed with release date 29 Oct 01) 〈〉 (2001).

  8. Lander, E. et al. Initial sequencing and analysis of the human genome. Nature 409, 860–921 (2001).

    Article  CAS  PubMed  Google Scholar 

  9. Venter, J. C. et al. The sequence of the human genome. Science 291, 1304–1351 (2001).

    Article  CAS  PubMed  Google Scholar 

  10. Tweeddale, H., Notley-McRobb, L. & Ferenci, T. Effect of slow growth on metabolism of Escherichia coli, as revealed by global metabolite pool ('metabolome') analysis. J. Bacteriol. 180, 5109–5119 (1998).

    CAS  PubMed  PubMed Central  Google Scholar 

  11. Fiehn, O. et al. Metabolite profiling for plant functional genomics. Nature Biotechnol. 18, 1157–1161 (2000).

    Article  CAS  Google Scholar 

  12. Raamsdonk, L. et al. A functional genomics strategy that uses metabolome data to reveal the phenotype of silent mutations. Nature Biotechnol. 19, 45–50 (2001).

    Article  CAS  Google Scholar 

  13. Claverie, J.-M. What if there are only 30,000 human genes? Science 291, 1255–1257 (2001).

    Article  CAS  PubMed  Google Scholar 

  14. Walke, D. W. et al. In vivo drug target discovery: identifying the best targets from the genome. Curr. Opin. Biotechnol. 12, 626–631 (2001)

    Article  CAS  PubMed  Google Scholar 

  15. Lehman Brothers. The Fruits of Genomics (Lehman Brothers, 2001).

  16. Rubin, al Comparative genomics of the eukaryotes. Science 287, 2204–2215 (2000).

    Article  CAS  PubMed  PubMed Central  Google Scholar 

Download references


We are indebted to J. P. Overington (Inpharmatica, London), A. Alex and L. Beeley for their contributions to the ideas on the physico-chemical limits for protein-binding sites. We also thank R. W. Spencer and C. Lipinski (Pfizer, Groton, Connecticut, USA) for much stimulating discussion.

Author information

Authors and Affiliations


Corresponding author

Correspondence to Colin R. Groom.

Supplementary information

Related links

Related links



Protein Data Bank

Proteome Analysis Database

Rights and permissions

Reprints and permissions

About this article

Cite this article

Hopkins, A., Groom, C. The druggable genome. Nat Rev Drug Discov 1, 727–730 (2002).

Download citation

  • Issue Date:

  • DOI:

This article is cited by


Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing