RNAi and the drug discovery process

doi:10.1017/CBO9780511546402.027

24 - RNAi and the drug discovery process

Published online by Cambridge University Press: 31 July 2009

Jeff Aalfs ,

Paul Yaworsky and

Edited by

Foreword by

Andrew Fire and

Marshall Nirenberg

Show author details

Steven A. Haney: Affiliation:
Wyeth Research
Peter Lapan: Affiliation:
Wyeth Research
Jeff Aalfs: Affiliation:
Wyeth Research
Chris Miller: Affiliation:
Wyeth Research
Paul Yaworsky: Affiliation:
Wyeth Research
Chris Childs: Affiliation:
Wyeth Research
Krishnarao Appasani: Affiliation:
GeneExpression Systems, Inc., Massachusetts
Andrew Fire: Affiliation:
Stanford University, California

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Summary

Introduction

Drug discovery is a complex process that seeks to identify therapeutics for treating human disease. It has very high failure rate, and by one estimate, the total cost for a therapeutic successfully brought to market is 803 million dollars (DiMasi et al., 2003). Failure occurs at all points in the process, with failures at the pre-development stages being the most common, and failures at the clinical stages being the most costly. Scientists working in drug discovery are continually challenged to identify ways to improve the process. Current efforts are largely “target-based” approaches. Once chosen, the target may be studied in vitro for more than a year, and in model systems of the disease for up to four years. Errors in determining whether a given target is truly effective in treating a disease may not be detected until Phase II or Phase III clinical studies, which follow many years of study and a financial investment of tens or hundreds of millions of dollars. As such, target validation is a critical aspect of the drug discovery process.

The pharmaceutical industry has invested heavily in genomics because of its promise to provide a continuing supply of drug targets (Wiley, 1998; Ohlstein et al., 2000; Baba, 2001). Implicit in this investment has been the expectation that the targets provided by genomics are highly validated (Debouck and Metcalf, 2000). Thus, genomics has grown broadly across the drug discovery process, from target identification to phamacogenomics.

Information

Type: Chapter
Information: RNA Interference Technology
From Basic Science to Drug Development
, pp. 331 - 346

DOI: https://doi.org/10.1017/CBO9780511546402.027 [Opens in a new window]

Publisher: Cambridge University Press

Print publication year: 2005

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