Genscience and Deerfield Management Announce Nature Publication Reporting Positive Impact of Genetic Evidence to Aid Discovery of More Effective Drug Targets

Probability of success for drug mechanisms with genetic support is 2.6 times greater than those without, according to new study

NEW YORK, April 17, 2024 /PRNewswire/ — Genscience LLC, a company applying genetic evidence and proprietary platforms for drug discovery and development and an affiliate of Deerfield Management Company (“Deerfield“), a healthcare investment firm, today announced a new study that shows incorporating genetic evidence into drug design significantly increases the probability of successful development by up to 2.6 times compared to when genetic evidence is absent. The new study appeared in the April 17, 2024 issue of the peer-reviewed journal Nature.¹ The study also documents new findings regarding how different characteristics of genetic evidence impact drug development success.

“These findings further demonstrate that genetic evidence is the most effective means to improve drug discovery and development success rates and R&D efficiency,” said Matt Nelson, PhD, Chief Executive Officer of Genscience and Vice President of Genetics and Genomics, Deerfield Discovery and Development (3DC). “This new data helps illustrate the power of genetic evidence to enrich our understanding of the relationship between a potential drug target and the disease we seek to treat.”

The new study builds on pivotal work Nelson published in 2015 ² and documents which genetic associations are more likely to differentiate successful from unsuccessful drug mechanisms across 18 therapy areas and among five discovery and development phases from preclinical to launched. Nelson and his colleagues found that the probability of success for drug mechanisms backed by genetic evidence is 2.6 times higher than those lacking such support, with a four-fold increase in hematology and metabolic therapy areas.

“This study provides a basis for optimism, that we can improve the success rate in drug discovery,” said Eric Vallabh Minikel, PhD, first author of the study, and senior group leader at the Broad Institute. “Human genetics can point us to the root molecular causes of disease and enable us to design fundamentally disease-modifying therapies.”

Nelson acknowledges that although many questions remain about the application and effectiveness of human genetics in drug discovery, the field of genetics identifies causal relationships between genes and disease in humans, providing a growing opportunity for novel target identification and improving indication selection for existing drugs and drug candidates. Increasing emphasis on drug mechanisms with supporting genetic evidence is expected to increase success rates and lower the cost of drug discovery and development.

“The implications of this study are profound, offering a promising genetic-informed approach for biotechnology and pharmaceutical companies to improve their drug development strategies and increase the likelihood of bringing life-saving medications to market,” said James Flynn, Managing Partner, Deerfield Management. “These findings also demonstrate the meaningful discoveries that advance health and clinical trials that Deerfield, 3DC and Genscience are committed to supporting.”

1Minikel EV, Painter JL, Dong CC, Nelson MR. Refining the impact of genetic evidence on clinical success. Nature. 2024. DOI: 10.1038/s41586-024-07316-0.
2Nelson MR, Tipney H, Painter JL, et al. The support of human genetic evidence for approved drug indications. Nat Genet. 2015;47(8):856-860. doi:10.1038/ng.3314

About Genscience
Genscience, spun out of Deerfield Management Company and founded by Matthew R. Nelson, PhD, in 2023, is a platform and services company, developing state-of-the science genetics capabilities for the purpose of supporting drug discovery and development across disease areas.

About Deerfield Management
Deerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. The Firm works across the healthcare ecosystem to connect people, capital, ideas and technology in bold, collaborative and inclusive ways. For more information, visit

Genscience contact:
Matthew R. Nelson, PhD
[email protected]

Deerfield contact:
Rachel Pelletier
[email protected]
(440) 409-6274

SOURCE Deerfield Management

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