The Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed) funding from the National Institutes of Health (NIH) supports efficacy studies in an established rare disease preclinical model to demonstrate that a proposed therapeutic agent warrants further development. In addition to preclinical efficacy, accompanying pharmacodynamic and pharmacokinetic studies would be supported. Therapeutic agents include small molecules, biologics or biotechnology-derived products. The program seeks to spur therapeutic development for rare diseases by advancing projects to the point where they would attract subsequent investment supporting full Investigational New Drug (IND) application development or progression to clinical trials in the case of repurposing or repositioning. Applications are open to researchers at research institutions in the USA, including foreign components of US institutions. Applications are also open to small businesses and for-profit organizations in the USA. Applicants may apply for up to 275,000 USD, plus indirect costs for up to 2 years.
Opportunity number: RFA-TR-23-016
The next letters of intent are due: May 1, 2024. Applications are due: June 3, 2024.
June 3, 2024