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Drug development

Genetic validation of drug targets improves chance of success

Since the completion of the Human Genome Project there has been rapid progress in identifying genes that influence human health and disease. In the image, DNA strands

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When genetic evidence supported a drug’s mechanism, it was more likely to progress through the development pipeline

Since the completion of the Human Genome Project there has been rapid progress in identifying genes that influence human health and disease. Now, research in Nature Genetics (online, 29 June 2015) suggests that by harnessing these data to support the selection of candidate drugs, developers could double their success rates.

Matthew Nelson, from GlaxoSmithKline, and colleagues evaluated the extent to which genetic evidence can predict clinically efficacious drug mechanisms. They found that, among well-studied diseases, the proportion of drug mechanisms with “direct genetic support” increased from 2.0% at the preclinical stage to 8.2% for approved compounds. This shows that genetic support increased the chance of a drug successfully progressing through the development pipeline.

The team suggests that increasing the proportion of drug discovery and development activities focused on targets with genetic support should lead to lower rates of failure.

Citation: The Pharmaceutical Journal DOI: 10.1211/PJ.2015.20068960

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  • Since the completion of the Human Genome Project there has been rapid progress in identifying genes that influence human health and disease. In the image, DNA strands

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