Early-Stage Preclinical Validation of Therapeutic Leads for Diseases of Interest to the NIDDK (R01)

Early-Stage Preclinical Validation of Therapeutic Leads for Diseases of Interest to the NIDDK (R01)
Department of Health and Human ServicesSquare_TransparentBackground
National Institutes of Health


Recent significant advances in genetics, physiology, and the pathogenesis of disease coupled with technological advances in areas such as bioinformatics, chemical biology, non-mammalian disease modeling, and protein engineering have provided a rich knowledge base and strong toolbox to identify and pursue new therapeutics with the goal of generating new molecular, microbial, and cellular therapies for the treatment of disease. Despite the availability of these approaches, we are facing an increased failure rate of potential therapeutics for reasons of both safety and lack of human efficacy.  A critical need exists to develop better strategies to validate and prioritize therapeutic leads based on their likelihood of success to safely alter disease progression and outcomes in humans.


Projects submitted to this FOA should be directly focused on a therapeutic lead that will modulate a health-related outcome of interest to the NIDDK. Prior to submitting an application, investigators are strongly encouraged to contact the Scientific/Research Contact for this FOA to discuss the appropriateness of the proposed research to this FOA. Information concerning programmatic areas at NIDDK is available at: www.niddk.nih.gov/research-funding/research-programs/Pages/default.aspx. Projects in areas that are primarily within the missions of other Institutes or Centers (ICs) of the NIH are not appropriate for this FOA and will not be supported.

Staging of Therapeutic Discovery and Validation

The process of identifying and validating therapeutic leads such as small molecules or non-viral biologics for the treatment of human disease begins with a hypothesis and can be viewed as progressing along a continuum of increasing confidence leading to widespread acceptance of its use in people (www.niddk.nih.gov/research-funding/process/translational-research-therapeutic-discovery-development/Pages/default.aspx). For the purposes of this FOA, these stages are defined as:

Identification of therapeutic leads: This may occur through a variety of approaches and stages such as identifying targets, screening compound libraries, or constructing prototype non-viral biologics with preliminary evidence that they may significantly impact a disease process.

Early-stage preclinical validation: Pre-clinical hypothesis testing to generate data that, over time, increases confidence that manipulation of disease processes via a specific therapeutic strategy may be clinically efficacious and safe. This process occurs prior to clinical testing of a new therapeutic, and ideally should include the use of human-derived data, tissues, cells, and systems.

Clinical validation: Studies conducted in human patient populations to fully understand the efficacy and safety profiles of a therapeutic agent. True validation of a therapeutic agent may take decades of post-regulatory approval data accumulation.

General Information

Document Type: Grants Notice
Funding Opportunity Number: PAR-16-121
Funding Opportunity Title: Early-Stage Preclinical Validation of Therapeutic Leads for Diseases of Interest to the NIDDK (R01)
Opportunity Category: Discretionary
Opportunity Category Explanation:
Funding Instrument Type: Grant
Category of Funding Activity: Food and Nutrition
Category Explanation:
Expected Number of Awards:
CFDA Number(s): 93.847 — Diabetes, Digestive, and Kidney Diseases Extramural Research
Cost Sharing or Matching Requirement: No
Posted Date: Mar 09, 2016
Last Updated Date: Mar 09, 2016
Original Closing Date for Applications: May 07, 2019  
Current Closing Date for Applications: May 07, 2019  
Archive Date: Jun 07, 2019
Estimated Total Program Funding:
Award Ceiling:
Award Floor:


Eligible Applicants:
Small businesses
City or township governments
Native American tribal organizations (other than Federally recognized tribal governments)
Private institutions of higher education
Others (see text field entitled “Additional Information on Eligibility” for clarification)
County governments
Special district governments
Public housing authorities/Indian housing authorities
Independent school districts
Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education
For profit organizations other than small businesses
Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education
Native American tribal governments (Federally recognized)
State governments
Public and State controlled institutions of higher education
Additional Information on Eligibility: Other Eligible Applicants include the following: Alaska Native and Native Hawaiian Serving Institutions; Asian American Native American Pacific Islander Serving Institutions (AANAPISISs); Eligible Agencies of the Federal Government; Faith-based or Community-based Organizations; Hispanic-serving Institutions; Historically Black Colleges and Universities (HBCUs); Indian/Native American Tribal Governments (Other than Federally Recognized); Non-domestic (non-U.S.) Entities (Foreign Organizations); Regional Organizations; Tribally Controlled Colleges and Universities (TCCUs) ; U.S. Territory or Possession.

Additional Information

Agency Name: National Institutes of Health
Description: The overarching goal of this Funding Opportunity Announcement (FOA) is to translate basic science research into knowledge and tools that can be utilized to provide strong justification for later-phase therapeutics discovery and development efforts in health-related outcomes relevant to the National Institute of Diabetes and Digestive and Kidney Diseases. This includes outcomes relevant to obesity, diabetes and related aspects of endocrinology and metabolism, digestive diseases, liver diseases, nutrition, kidney and urological diseases, hematology, and specific aspects of cystic fibrosis. Additional information concerning programmatic areas at NIDDK is available at: http://www.niddk.nih.gov/research-funding/research-programs/Pages/default.aspx.
Link to Additional Information: http://grants.nih.gov/grants/guide/pa-files/PAR-16-121.html
Contact Information: If you have difficulty accessing the full announcement electronically, please contact:

NIH OER Webmaster FBOWebmaster@OD.NIH.GOV
If you have any problems linking to this funding announcement, please contact the NIH OER Webmaster


Leave a Reply

Fill in your details below or click an icon to log in:

WordPress.com Logo

You are commenting using your WordPress.com account. Log Out /  Change )

Google+ photo

You are commenting using your Google+ account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )


Connecting to %s