This funding opportunity supports efficacy studies in established rare disease preclinical models to demonstrate that therapeutic agents, including small molecules, biologics, or biotechnology-derived products, warrant further development. It also funds pharmacodynamic and pharmacokinetic studies. The goal is to advance projects to attract investment for IND application development or clinical trials in repurposing cases. Clinical trials are not allowed under this award.
Eligible applicants include public and private higher education institutions, nonprofits, for-profit organizations, small businesses, tribal governments, and various government entities. Specific eligibility details are provided in the full announcement.
