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From target discovery to clinical drug development with human genetics
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Journal Article
From target discovery to clinical drug development with human genetics
2023
Overview
The substantial investments in human genetics and genomics made over the past three decades were anticipated to result in many innovative therapies. Here we investigate the extent to which these expectations have been met, excluding cancer treatments. In our search, we identified 40 germline genetic observations that led directly to new targets and subsequently to novel approved therapies for 36 rare and 4 common conditions. The median time between genetic target discovery and drug approval was 25 years. Most of the genetically driven therapies for rare diseases compensate for disease-causing loss-of-function mutations. The therapies approved for common conditions are all inhibitors designed to pharmacologically mimic the natural, disease-protective effects of rare loss-of-function variants. Large biobank-based genetic studies have the power to identify and validate a large number of new drug targets. Genetics can also assist in the clinical development phase of drugs—for example, by selecting individuals who are most likely to respond to investigational therapies. This approach to drug development requires investments into large, diverse cohorts of deeply phenotyped individuals with appropriate consent for genetically assisted trials. A robust framework that facilitates responsible, sustainable benefit sharing will be required to capture the full potential of human genetics and genomics and bring effective and safe innovative therapies to patients quickly.
This Review provides a perspective on the development of non-cancer therapies based on human genetics studies and suggests measures that can be taken to streamline the pipeline from initial genetic discovery to approved therapy.
Publisher
Nature Publishing Group UK,Nature Publishing Group
Subject
/ Drug Approval - statistics & numerical data
/ Drug Development - statistics & numerical data
/ Drugs
/ Genes
/ Genetics
/ Genomics
/ Humanities and Social Sciences
/ Humans
/ Molecular Targeted Therapy - methods
/ Molecular Targeted Therapy - statistics & numerical data
/ Mutation
/ Research & development expenditures
/ Review
/ Science
