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Synthesis and Biological Activity Assessment of Different Diastereomers in siRNA Drug LEQVIO (Inclisiran) (U01) Clinical Trial Not Allowed
The purpose of this research is to systematically evaluate the diastereomeric composition of LEQVIO (Inclisiran), an FDA-approved, N-acetylgalactosamine (GalNAc)-conjugated siRNA drug, and to understand the biological/pharmacological activity of each diastereomer in LEQVIO through stereochemically-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 stereochemically pure form; 2) assessment of the biological activity of each stereochemically 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.
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 stereochemically pure form; 2) assessment of the biological activity of each stereochemically 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.
Agency: Department of Health and Human Services
Office: Food and Drug Administration
Estimated Funding: $1,200,000
Office: Food and Drug Administration
Estimated Funding: $1,200,000
Who's Eligible
Obtain Full Opportunity Text:
FULL ANNOUNCEMENT
Additional Information of Eligibility:
Eligible OrganizationsHigher Education InstitutionsPublic/State Controlled Institutions of Higher EducationPrivate Institutions of Higher EducationThe following types of Higher Education Institutions are always encouraged to apply for FDA support as Public or Private Institutions of Higher Education:Hispanic-serving InstitutionsHistorically Black Colleges and Universities (HBCUs)Tribally Controlled Colleges and Universities (TCCUs)Alaska Native and Native Hawaiian Serving InstitutionsAsian American Native American Pacific Islander Serving Institutions (AANAPISIs)Nonprofits Other Than Institutions of Higher EducationNonprofits with 501(c)(3) IRS Status (Other than Institutions of Higher Education)Nonprofits without 501(c)(3) IRS Status (Other than Institutions of Higher Education)For-Profit OrganizationsSmall BusinessesFor-Profit Organizations (Other than Small Businesses)Local GovernmentsState GovernmentsCounty GovernmentsCity or Township GovernmentsSpecial District GovernmentsIndian/Native American Tribal Governments (Federally Recognized)Indian/Native American Tribal Governments (Other than Federally Recognized)U. S. Territory or PossessionOtherIndependent School DistrictsPublic Housing Authorities/Indian Housing AuthoritiesNative American Tribal Organizations (other than Federally recognized tribal governments)Faith-based or Community-based OrganizationsRegional OrganizationsNon-domestic (non-U.S.) Entities (Foreign Institutions)
Full Opportunity Web Address:
http://grants.nih.gov/grants/guide/rfa-files/RFA-FD-23-012.html
Contact:
Agency Email Description:
shashi.malhotra@fda.hhs.gov
Agency Email:
Date Posted:
2022-12-01
Application Due Date:
Archive Date:
2023-04-21
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