Drug development in the era of precision medicine
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For the past three decades, the use of genomics to inform drug discovery and development pipelines has generated both excitement and scepticism. Although earlier efforts successfully identified some new drug targets, the overall clinical efficacy of developed drugs has remained unimpressive, owing in large part to the heterogeneous causes of disease. Recent technological and analytical advances in genomics, however, have now made it possible to rapidly identify and interpret the genetic variation underlying a single patient's disease, thereby providing a window into patient-specific mechanisms that cause or contribute to disease, which could ultimately enable the 'precise' targeting of these mechanisms. Here, we first examine and highlight the successes and limitations of the earlier phases of genomics in drug discovery and development. We then review the current major efforts in precision medicine and discuss the potential broader utility of mechanistically guided treatments going forward.
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Identifying key drivers of disease by teasing apart biology and technical variation from high throughput, high dimensional datasets will lead to more successful drug and perturbation target identification, as well as robust drug development.
“Identifying key drivers of disease by teasing apart biology and technical variation from high throughput, high dimensional datasets will lead to more successful drug and perturbation target identification, as well as robust drug development.57,58,59”
From SCING: Inference of robust, interpretable gene regulatory networks from single cell and spatial transcriptomics (2023) · cites this paper as [58]
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Looking beyond the hype: applied AI and machine learning in translational medicine
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<i>In vitro</i> functional models for human liver diseases and drug screening: beyond animal testing
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- Authors
- Dugger, S. A., Platt, A., & Goldstein, D. B.
- Affiliations
- Columbia University Irving Medical Center (US)
- AstraZeneca (United Kingdom) (GB)
- Corresponding author
- Goldstein, David B.
- Venue
- Nature Reviews Drug Discovery
- Published
- 2017-12-08
- Volume
- 17
- Issue
- 3
- Pages
- 183-196
- ISSN
- 1474-1776 (ISSN-L)
- 1474-1784
- PMC
- PMC6287751
- OpenAlex
- W2773309042
- Cited by
- 493
- References
- 167
- Citations by year
- 2026: 34 · 2025: 56 · 2024: 78 · 2023: 78 · 2022: 64 · 2021: 61 · 2020: 53 · 2019: 49 · 2018: 19
- FWCI
- 4.25
- Type
- journal article
- Language
- en
- Open access
- green
- europepmc.org
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Nature Reviews Drug Discovery (publishedVersion)
- PubMed (publishedVersion)
- Europe PMC (PubMed Central) (acceptedVersion)
- PubMed Central (acceptedVersion)
- Funders
- National Institutes of Health (TL1TR001875)
- National Institute of General Medical Sciences (T32GM007088)
- National Center for Advancing Translational Sciences
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Genetics › Genomics and Rare Diseases
- Physical Sciences › Computer Science › Computational Theory and Mathematics › Computational Drug Discovery Methods
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmacology › Pharmacogenetics and Drug Metabolism
- Keywords
- precision medicine, drug discovery, rare genetic disorders, genetic variation, therapeutic target, disease heterogeneity
- MeSH terms
- Drug Development, Pharmaceutical Preparations, Humans, Neoplasms, Genomics, Rare Diseases, Precision Medicine
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