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NOBEL PRIZE 2026

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NIHNATIONAL INSTITUTE ON AGING

Single Cell Foundation Model enabled drug discovery for clonal hematopoiesis

Active
Institution
Vanderbilt University Medical Center, TN
Principal investigator
Alexander Bick
Funding
$1.0M
Duration
2025–2027
Open portfolio

Moonbase score

Still being scored

Abstract

Project Summary Aging is associated with increased mortality and the prevalence of diseases such as cancer, cardiovascular, neurological, and respiratory diseases. One fundamental mechanism of aging is acquiring DNA mutations, which can lead to clonal hematopoiesis of indeterminate potential (CHIP), frequently driven by mutations in in DNMT3A, TET2, and ASXL 1. We seek to identify and re purpose existing medications or investigational therapeutics to decrease or eliminate the CHIP clone as a way to address multiple agingrelated diseases. We will leverage single cell machine learning foundation models, human genetics, and electronic health records (EHR). We propose a two-phase approach: In the UG3 phase, we will utilize single cell foundation models to perform virtual screens as well as genetic and EHR data to identify existing medications to reverse CHIP pathology. In the UH3 phase, we will experimentally validate drug candidates in in vitro and in vivo CHIP models. Successful execution of this work will rapidly translate findings into the clinic by repurposing FDA-approved medications to mitigate CHIP and its related aging diseases.

Research team

  • Alexander BickPI
  • Ravi Kiran MadduriPI
  • Michael R SavonaPI

LOADING MOONBASE SCORE

NATIONAL INSTITUTE ON AGINGUG3Special Emphasis Panel[ZRG1 CN-V (55)]clonalhematopoiesisfoundation modelsmedicationssingle cellchipehrclonal hematopoiesis

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