Compositions and methods for delivering molecules
AssigneeSunVax mRNA Therapeutics Inc.
InventorsYingzhong LI, Libin ZHANG
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Abstract
Compositions comprising mRNA, self-amplifying mRNA (sa-mRNA), modified mRNA, or circular RNA constructs comprising a gene-of-interest, and lipids and LNPs for use in therapy are disclosed. A method of delivering a payload to immune cells ex vivo by contacting immune cells with a lipid nanoparticle (LNP) encapsulating a payload encoding at least one polypeptide of interest, wherein the polypeptide of interest is an antigen receptor or an antibody.
Claims30
A lipid compound of Formula I:
- wherein
- each R1, R2, R3, R4 and R5 is independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each R6, R7, R8 and R9 are independently selected from H, C1-C24 alkyl, C1-C24 alkenyl, C1-C24 alkynyl, substituted alkyl, substituted alkenyl, substituted alkynyl, substituted acyl, substituted carbocyclyl, substituted heterocyclyl, substituted aryl, substituted heteroaryl,
- a, b and c are each independently an integer from 0-24;
- each X is independently selected from CH, N;
- each Y is independently selected from CH2, NH, O, or S;
- each Z is independently selected from CH or N;
- or a salt or isomer thereof.
A method of delivering a payload to immune cells ex vivo comprising contacting immune cells with a lipid nanoparticle (LNP) encapsulating a payload encoding at least one polypeptide of interest, wherein the polypeptide of interest is an antigen receptor or an antibody, or delivering a payload to immune cells in vivo comprising administering an LNP encapsulating a payload encoding at least one polypeptide of interest to a subject, wherein the polypeptide of interest is antigen receptor or antibody.
A method of increasing transfection efficiency of mRNA, wherein the mRNA comprises at least one GOI encoding a CAR, a TCR, a GFR, a HR, a Nb, a scFv, or a reporter, said method comprising substituting uridine (U) nucleotide residues of a reference mRNA with nucleotide residues selected from the group consisting of 1-methylpseudouridine (m1Ψ) and pseudouridine (Ψ), thereby rendering modified mRNA, wherein the modified mRNA shows increased transfection efficiency than the reference mRNA.
A method of treating a subject in need of cell therapy, comprising modifying an immune cell by (i) delivering a mRNA encoding at least one polypeptide of interest, wherein the polypeptide of interest is an antigen receptor or antibody, (ii) maintaining the immune cell under conditions suitable for expression of the polypeptide of interest to produce a modified immune cell, and (iii) administering the modified immune cell to the subject, or comprising modifying a T-cell by (i) delivering a mRNA encoding CAR to the T-cell, (ii) maintaining the T-cell under conditions suitable for expression of CAR to produce a modified T-cell, and (iii) administering the modified T-cell to the subject.
Dependencies are inferred from the claim text.