The National Institutes of Health (NIH), a part of the U.S. Department of Health and Human Services, is the nation’s medical research agency — making important discoveries that improve health and save lives.
The grant program aims to fund efficacy studies in established rare disease preclinical models to demonstrate that a proposed therapeutic agent is ready for further development. It supports studies on therapeutic agents, including small molecules, biologics, or biotechnology-derived products, focusing on preclinical efficacy along with pharmacodynamic and pharmacokinetic studies. The objective is to advance therapeutic development for various rare diseases, encouraging projects to reach a stage that would attract further investment for full Investigational New Drug (IND) application development or progression to clinical trials, especially in cases of repurposing or repositioning existing treatments.