Funding Opportunity Number: PAR-25-122
Deadlines: February 16, 2025, March 16, 2025
Introduction
The National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH), is transforming the translational science process so that new treatments and cures for disease can be delivered to patients faster, delivering more treatments for all people more quickly. NCATS strives to develop innovations to reduce, remove or bypass costly and time-consuming bottlenecks in the translational research pipeline in an effort to speed the delivery of new drugs, diagnostics and medical devices to patients.
This notice of funding opportunity (NOFO) aims to promote innovative research to increase knowledge of understudied proteins associated with rare diseases. The submission of small research grant (R03) applications is encouraged from organizations proposing projects leading to a better understanding of eligible proteins listed below within the context of rare disease.
Small research (R03) grants provide flexibility for initiating discrete, well-defined projects that realistically can be completed in one year and require only limited levels of funding. This program supports different types of projects including, but not limited to, the following:
- Pilot or feasibility studies;
- Small, self-contained research projects;
- Development of research methodology;
- Development of assays to support compound screening projects; and/or
- Development of human cell or animal based models.
These awards will support generation of preliminary data and tools around eligible understudied protein(s) with the intent of elucidating the function of these proteins in the context of rare disease and obtaining sufficient preliminary data and/or research resources for subsequent grant applications and/or drug discovery projects. These grants are non-renewable.
Objectives and Scope
The goal of this specific solicitation is to provide a needed opportunity for the collection of preliminary data around the role of understudied proteins associated with rare diseases. This NOFO will provide funding to support research that will characterize new targets for treatment of human disease among the understudied proteins of the Druggable Proteome. These projects should be carried out in a short period of time with limited resources as defined by the funding mechanism.
It has been recognized through workshops and publications that understudied proteins become illuminated when (1) there are tools to study the protein (e.g., tools that modulate protein activity) and/or (2) there is biochemical, cellular, or animal model evidence of disease/physiological relevance. This NOFO was developed to address the need for expanded research and validation experiments on eligible understudied protein(s), with the intent of producing preliminary data to address the lack of biochemical, cellular, or animal model data associated with many understudied proteins. It is expected that the award will be used to obtain preliminary data and/or research resources for subsequent grant applications and/or drug discovery projects.
The NIH supports research on a broad range of diseases that are defined as rare; that is diseases affecting fewer than 200,000 individuals in the United States (per the Rare Disease Act of 2002). Collectively, there are an estimated 10,000 rare diseases, which cumulatively affect millions of people in the United States. Most are serious or life-threatening, with a disproportionate number of rare diseases affecting children. At this time, effective treatments are available for fewer than 5%. Pharos has linked over 6,000 proteins to rare diseases, with over 80% of these proteins considered extremely understudied. For the purposes of this NOFO, eligible proteins are those that have an association with rare disease through data mining of the NCATS Genetic and Rare Diseases Information Center (GARD), are considered understudied (those proteins that lack small molecule binders and/or have limited biological characterization, and have low numbers of associated publications) and are within a protein family that is traditionally considered druggable.
For more information, please see the opportunity webpage.