Early-Stage Preclinical Validation of Therapeutic Leads for Diseases of Interest to the NIDDK (R01)
Department of Health and Human Services
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.
|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:|
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)
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)
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.|
|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