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2432856

Project Grant

Overview

Grant Description
SBIR Phase I: High-throughput platform for small molecule, in-cell targeting of undruggable proteins via their mRNAs.

The broader impact of this Small Business Innovation Research (SBIR) Phase I project will be improvement of the health and welfare of Americans by providing an efficient and cost-effective approach for developing therapeutics for previously undruggable diseases, including hard to treat neurodegenerative diseases and highly aggressive cancers.

Most diseases are targeted at the protein level using small molecule drugs, but only 600 proteins have ever been drugged directly.

This collective human effort has left the majority of roughly 3,000 disease-related genes in humans undrugged and unable to be drugged using conventional pharmaceutical technology.

Messenger RNAs (mRNAs) lie upstream of protein expression and, in principle, can be targeted to modulate protein function and treat disease.

However, the physical and chemical properties of mRNAs present unique challenges not faced during protein-based drug discovery, and there has been little success in targeting mRNAs using small molecules.

This project will address this unmet need by developing small molecule drugs against hard to treat and previously undruggable diseases by targeting their mRNAs directly.

The proposed project will enable critical technical innovations needed to ensure the technical and commercial viability of a nascent drug discovery platform making it high-throughput and cost effective.

The high throughput drug discovery platform to be developed creates an efficient path to screen for high-value drug assets and creates multiple pathways to new classes of therapeutics.

The drug discovery platform currently has outstanding robustness and accuracy in defining interactions between small molecules and mRNAs in cells.

However, the platform includes bespoke and hands-on steps and will remain a research-lab-only tool without critical innovations.

The proposed project will improve the platform to be capable of fully automated ligand screening in cells using a library of complex small molecules optimized to bind mRNA.

Methods will be developed to screen multiplexed samples, in a quantitative way.

Automation will require the integration of diverse, novel methodologies, and data deconvolution.

This award reflects NSF's statutory mission and has been deemed worthy of support through evaluation using the Foundation's intellectual merit and broader impacts review criteria.

Subawards are not planned for this award.
Funding Goals
THE GOAL OF THIS FUNDING OPPORTUNITY, "NSF SMALL BUSINESS INNOVATION RESEARCH (SBIR)/ SMALL BUSINESS TECHNOLOGY TRANSFER (STTR) PROGRAMS PHASE I", IS IDENTIFIED IN THE LINK: HTTPS://WWW.NSF.GOV/PUBLICATIONS/PUB_SUMM.JSP?ODS_KEY=NSF23515
Awarding / Funding Agency
Place of Performance
Research Triangle Park, North Carolina 27709-0962 United States
Geographic Scope
Single Zip Code
Foragr Medicines was awarded Project Grant 2432856 worth $275,000 from National Science Foundation in January 2025 with work to be completed primarily in Research Triangle Park North Carolina United States. The grant has a duration of 1 year and was awarded through assistance program 47.084 NSF Technology, Innovation, and Partnerships. The Project Grant was awarded through grant opportunity NSF Small Business Innovation Research / Small Business Technology Transfer Phase I Programs.

SBIR Details

Research Type
SBIR Phase I
Title
SBIR Phase I: High-throughput platform for small molecule, in-cell targeting of undruggable proteins via their mRNAs
Abstract
The broader impact of this Small Business Innovation Research (SBIR) Phase I project will be improvement of the health and welfare of Americans by providing an efficient and cost-effective approach for developing therapeutics for previously undruggable diseases, including hard to treat neurodegenerative diseases and highly aggressive cancers. Most diseases are targeted at the protein level using small molecule drugs, but only 600 proteins have ever been drugged directly. This collective human effort has left the majority of roughly 3,000 disease-related genes in humans undrugged and unable to be drugged using conventional pharmaceutical technology. Messenger RNAs (mRNAs) lie upstream of protein expression and, in principle, can be targeted to modulate protein function and treat disease. However, the physical and chemical properties of mRNAs present unique challenges not faced during protein-based drug discovery, and there has been little success in targeting mRNAs using small molecules. This project will address this unmet need by developing small molecule drugs against hard to treat and previously undruggable diseases by targeting their mRNAs directly. The proposed project will enable critical technical innovations needed to ensure the technical and commercial viability of a nascent drug discovery platform making it high-throughput and cost effective. The high throughput drug discovery platform to be developed creates an efficient path to screen for high-value drug assets and creates multiple pathways to new classes of therapeutics. The drug discovery platform currently has outstanding robustness and accuracy in defining interactions between small molecules and mRNAs in cells. However, the platform includes bespoke and hands-on steps and will remain a research-lab-only tool without critical innovations. The proposed project will improve the platform to be capable of fully automated ligand screening in cells using a library of complex small molecules optimized
Topic Code
BT
Solicitation Number
NSF 23-515

Status
(Ongoing)

Last Modified 1/22/25

Period of Performance
1/15/25
Start Date
12/31/25
End Date
76.0% Complete

Funding Split
$275.0K
Federal Obligation
$0.0
Non-Federal Obligation
$275.0K
Total Obligated
100.0% Federal Funding
0.0% Non-Federal Funding

Activity Timeline

Interactive chart of timeline of amendments to 2432856

Additional Detail

Award ID FAIN
2432856
SAI Number
None
Award ID URI
SAI EXEMPT
Awardee Classifications
Small Business
Awarding Office
491503 TRANSLATIONAL IMPACTS
Funding Office
491503 TRANSLATIONAL IMPACTS
Awardee UEI
CLEQGYM6VKY3
Awardee CAGE
14JB6
Performance District
NC-04
Senators
Thom Tillis
Ted Budd
Modified: 1/22/25