The Food and Drug Administration (FDA) has released a grant opportunity, identified as FOR-FD-24-007, aimed at conducting research on the synthesis and biological activity assessment of different diastereomers in the siRNA drug LEQVIO (Inclisiran). This cooperative agreement seeks to advance scientific understanding of the pharmacological activity of each diastereomer within LEQVIO, a drug that has garnered significant attention in the medical community for its innovative approach to cholesterol management. The grant falls under the categories of agriculture, consumer protection, and food and nutrition, underscoring its relevance in public health and safety.
This funding opportunity is open to a broad range of applicants, including educational institutions, government entities, non-profits, and private sector organizations. The research aims to synthesize each diastereomer of LEQVIO in stereochemically pure form and assess their individual biological activities, particularly in inhibiting PCSK9 activity, using both in vitro assays and animal models. Additionally, the project will focus on developing analytical methods to identify and characterize the stereochemical structure of each diastereomer and assess their contribution to the overall pharmacological activity of LEQVIO. With an award ceiling of $300,000, this grant represents a crucial step in enhancing the understanding and quality control of oligonucleotide drugs like LEQVIO, contributing significantly to the field of genetic medicine and the evaluation of generic siRNAs. The research supported by this grant could have far-reaching implications in the development and regulation of similar siRNA-based therapeutics.
Opportunity ID: 351130
General Information
| Document Type: | Grants Notice |
| Opportunity Number: | FOR-FD-24-007 |
| Opportunity Title: | Synthesis and Biological Activity Assessment of Different Diastereomers in siRNA Drug LEQVIO (Inclisiran) (U01) Clinical Trial Not Allowed |
| Opportunity Category: | Discretionary |
| Opportunity Category Explanation: | |
| Funding Instrument Type: | Cooperative Agreement |
| Category of Funding Activity: | Agriculture Consumer Protection Food and Nutrition |
| Category Explanation: | |
| Expected Number of Awards: | 1 |
| CFDA Number(s): | 93.103 — Food and Drug Administration Research |
| Cost Sharing or Matching Requirement: | No |
| Version: | Forecast 1 |
| Forecasted Date: | Nov 24, 2023 |
| Last Updated Date: | Nov 24, 2023 |
| Estimated Post Date: | |
| Estimated Application Due Date: | |
| Estimated Award Date: | |
| Estimated Project Start Date: | |
| Fiscal Year: | 2024 |
| Archive Date: | |
| Estimated Total Program Funding: | |
| Award Ceiling: | $ 300,000 |
| Award Floor: | $ 300,000 |
Eligibility
| Eligible Applicants: | Public housing authorities/Indian housing authorities Special district governments Native American tribal organizations (other than Federally recognized tribal governments) City or township governments State governments For profit organizations other than small businesses Nonprofits having a 501(c)(3) status with the IRS, other than institutions of higher education Nonprofits that do not have a 501(c)(3) status with the IRS, other than institutions of higher education County governments Unrestricted (i.e., open to any type of entity above), subject to any clarification in text field entitled “Additional Information on Eligibility” Private institutions of higher education Independent school districts Public and State controlled institutions of higher education Others (see text field entitled “Additional Information on Eligibility” for clarification) Small businesses Native American tribal governments (Federally recognized) |
| Additional Information on Eligibility: | Applicant organizations may submit more than one application, provided that each application is scientifically distinct. The FDA will not accept duplicate or highly overlapping applications under review at the same time per 2.3.7.4 Submission of Resubmission Application. This means that the NIH or FDA will not accept: •A new (A0) application that is submitted before issuance of the summary statement from the review of an overlapping new (A0) or resubmission (A1) application. •A resubmission (A1) application that is submitted before issuance of the summary statement from the review of the previous new (A0) application. •An application that has substantial overlap with another application pending appeal of initial peer review (see 2.3.9.4 Similar, Essentially Identical, or Identical Applications). |
Additional Information
| Agency Name: | Food and Drug Administration |
| Description: | The purpose of this research is to systematically evaluate the diastereomeric composition of LEQVIO (Inclisiran), an FDA-approved, N-acetyl galactosamine (GalNAc)-conjugated siRNA drug, and to understand the biological/pharmacological activity of each diastereomer in LEQVIO through stereo chemically controlled synthesis and biological activity assessment using in vitro and animal models. The proposed studies will focus on 1) synthesis of each diastereomer of LEQVIO (Inclisiran) in stereo chemically pure form; 2) assessment of the biological activity of each stereo chemically pure diastereomer in inhibiting PCSK9 activity using in vitro assays and in a transgenic mouse model; 3) development of analytical methods to identify and characterize the stereochemical structure of each diastereomer in LEQVIO; and 4) assessment of the individual contribution of each diastereomer to the overall pharmacological activity of LEQVIO. Tools developed in this research can also be applied to other similar GalNAc-conjugated siRNAs specifically, and other siRNAs in general. Knowledge gained from this research will also contribute to the sameness evaluation of generic siRNAs, and to the quality control of oligonucleotide drugs. |
| Link to Additional Information: | |
| Grantor Contact Information: |
Terrin Brown
Grantor 2403387494
|