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Publication Number
US-11773061-B2
Publication Date
2023-10-03
Expiration Date
Abstract
The present disclosure details various lipids, compositions, and/or methods of optimized systems and delivery vehicles for the delivery of nucleic acid sequences, polypeptides or peptides for use in vaccinating against infectious agents.
Core Innovation
The disclosure provides ionizable lipid compounds having the structure of Formula (CY-VI), or a pharmaceutically acceptable salt thereof, within broadly defined structural frameworks that set selections and optional substitutions for substituent groups including R1, Z1, X1, X2, X4, X5, Y1, Y2, Z2, Z3, R1a, R2a-R2c, R3a-R3c, R4a-R4c, R5a-R5c, and R6-R9. The structure includes variable definitions for A as a C3-C8 cycloalkylenyl and m and n independently ranging from 0 to 12, and the bond marked with an "*" is attached to X4 or X5. The disclosure also provides named CY compound structures and illustrative formulas within the CY series.
The disclosed embodiments are used in lipid nanoparticle (LNP) formulations together with nucleic acid payloads, including coding RNA. The LNP context includes component categories such as PEG-lipid, structural lipid, and non-ionizable lipid and/or zwitterionic lipid, and the disclosure further references coding RNA and mRNA as payload types. The same Formula (CY-VI) ionizable lipid framework is carried into the LNP claims and compositions.
Claims Coverage
The independent claims cover 3 core inventive features: an ionizable lipid of Formula (CY-VI), an LNP comprising that ionizable lipid, and an LNP comprising that ionizable lipid and a coding RNA.
Ionizable lipid of Formula (CY-VI)
A compound having the structure of Formula (CY-VI), or a pharmaceutically acceptable salt thereof, with defined substituent selections for R1, Z1, X1, X2, X4, X5, Y1, Y2, Z2, Z3, and R1a, including R2a/R2b/R2c, R3a/R3b/R3c, R4a/R4b/R4c, R5a/R5b/R5c, and R6/R7/R8/R9, with A as a C3-C8 cycloalkylenyl and m and n independently 0 to 12.
Lipid nanoparticle comprising Formula (CY-VI) ionizable lipid
A lipid nanoparticle (LNP) comprising an ionizable lipid having the structure of Formula (CY-VI), or a pharmaceutically acceptable salt thereof, with the same defined substituent framework for R1, Z1, X1, X2, X4, X5, Y1, Y2, Z2, Z3, and R1a, including A, m, and n.
Lipid nanoparticle comprising Formula (CY-VI) ionizable lipid and coding RNA
A lipid nanoparticle (LNP) comprising (A) an ionizable lipid having the structure of Formula (CY-VI), or a pharmaceutically acceptable salt thereof, under the same defined substituent framework, and (B) a coding RNA.
Across the independent claims, coverage is anchored on the Formula (CY-VI) ionizable lipid structure, extended to an LNP containing that ionizable lipid, and further extended to an LNP that includes a coding RNA.
Stated Advantages
Documented Applications
Delivery vehicles, including lipid nanoparticles, for nucleic acid payloads such as DNA and coding and non-coding RNA (including mRNA), with described effects as changes in cellular, tissue, and/or organ expression and genetic sequence.
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