Preclinical Proof of Concept Studies for Rare Diseases (R21 Clinical Trial Not Allowed)

This notice of funding opportunity (NOFO) provides funding to conduct efficacy studies in an established rare disease preclinical model to demonstrate that a proposed therapeutic agent warrants further development. The goal of this NOFO is to spur therapeutic development for a variety of 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. Key points to consider: • The funding will support preclinical efficacy studies, as well as accompanying pharmacodynamic and pharmacokinetic studies. • Therapeutic agents include small molecules, biologics, or biotechnology-derived products. • The opportunity is open to various types of applicants, including special district governments, nonprofits, small businesses, and public and state-controlled institutions of higher education. • The application deadline is June 2, 2026.