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US 2025/0017866 A1Published application
Polymers and nanoparticles for intramuscular nucleic acid delivery
AssigneeThe Johns Hopkins University
InventorsJordan J. GREEN, David WILSON, Stephany Yi TZENG, Yuan RUI, Sarah Y. NESHAT, Kathryn M. LULY
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Abstract
Biodegradable cationic polyesters for intramuscular delivery of nucleic acids, including self-amplifying mRNA, and methods of their use for treating conditions or diseases are disclosed.
Claims42
A nanoparticle comprising a compound of formula (I) and one or more nucleic acids:
- wherein:
- m and n are each independently an integer from 1 to 10,000;
- m1 is an integer selected from 0, 1, 2, 3, 4, 5, 6, 7, 8, 9, and 10;
- m2 is an integer selected from the group consisting of 1, 2, 3, 4, 5, 6, 7, 8, 9, 10, 11, 12, 13, 14, 15, 16, 17, 18, 19, and 20;
- q is an integer selected from 0 or 1;
- wherein —(CH2)m1—(C═C)q—(CH2)m2—CH3 comprises a hydrophobic sidechain;
- R comprises a divalent radical comprising a biodegradable ester linkage and/or a bioreducible disulfide linkage;
- R′ is hydrophilic sidechain comprising a monovalent radical derived from a hydrophilic amine monomer;
- R″ is monovalent radical derived from an amine-containing end capping group;
- and pharmaceutically acceptable salts thereof.
7 more elements
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33-36. (canceled)
42-46. (canceled)
The method of claim 46, wherein the SAM reaches a cytosol of a cell of the subject intact.
51-56. (canceled)
A kit comprising one or more of:
- one or more compounds of formula (I), one or more self-amplifying mRNAs (SAMs), one or more lipid-PEGs, one or more reagents, and instructions for use.
Dependencies are inferred from the claim text.