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

This text is about a funding opportunity notice, also known as NOFO, that is centered around Preclinical Proof of Concept Studies for Rare Diseases. Specifically, the aim of this NOFO is to support efficacy studies that are carried out in an already established model for preclinical rare disease research. These studies should be able to showcase that a proposed therapeutic agent is worthy of further development.

The types of therapeutic agents can range from small molecules, biologics, to biotechnology-derived products. Moreover, this funding opportunity also caters to pharmacodynamic and pharmacokinetic studies that accompany the preclinical efficacy studies.

One of the primary objectives of this NOFO is to stimulate the development of therapies for rare diseases by advancing pertinent projects. This would be achieved when these projects reach a point where they draw further investment, which would then support the full development of an Investigational New Drug (IND) application. Alternatively, it could also lead to its progression to clinical trials, especially when it comes to repurposing or repositioning.