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.
A61K 47/42 - ProteinsPolypeptidesDegradation products thereofDerivatives thereof, e.g. albumin, gelatin or zein
A61K 48/00 - Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseasesGene therapy
C07D 251/04 - Heterocyclic compounds containing 1,3,5-triazine rings not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
C07D 295/00 - Heterocyclic compounds containing polymethylene-imine rings with at least five ring members, 3-azabicyclo [3.2.2] nonane, piperazine, morpholine or thiomorpholine rings, having only hydrogen atoms directly attached to the ring carbon atoms
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
ex vivoex 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.
Lipid nanoparticle (LNP) encapsulating self-amplifying mRNA, compositions, and methods of using the novel nucleic acid constructs and compositions are disclosed. LNP constructs include novel ionizable lipid. Novel sa-mRNA constructs encode a modified SARS-COV-2 spike protein, wherein the polynucleotide has been truncated to not include nucleotides encoding a SARS-COV-2 transmembrane domain and short cytosolic domain amino acids and immunomodulators. Sa-mRNAs are useful in for use as a therapeutic, diagnostic and/or prophylactic agent to mammalian cells or organs.
C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
4.
SYNTHETIC SELF-AMPLIFYING MRNA MOLECULES WITH SECRETION ANTIGEN AND IMMUNOMODULATOR
Lipid nanoparticle (LNP) encapsulating self-amplifying mRNA, compositions, and methods of using the novel nucleic acid constructs and compositions are disclosed. LNP constructs include novel ionizable lipid. Novel sa-mRNA constructs encode a modified SARS-CoV-2 spike protein, wherein the polynucleotide has been truncated to not include nucleotides encoding a SARS-CoV-2 transmembrane domain and short cytosolic domain amino acids and immunomodulators. Sa-mRNAs are useful in for use as a therapeutic, diagnostic and/or prophylactic agent to mammalian cells or organs.
Lipid nanoparticle (LNP) encapsulating self-amplifying mRNA, compositions, and methods of using the novel nucleic acid constructs and compositions are disclosed. LNP constructs include novel ionizable lipid. Novel sa-mRNA constructs encode a modified SARS-CoV-2 spike protein, wherein the polynucleotide has been truncated to not include nucleotides encoding a SARS-CoV-2 transmembrane domain and short cytosolic domain amino acids and immunomodulators. Sa-mRNAs are useful in for use as a therapeutic, diagnostic and/or prophylactic agent to mammalian cells or organs.
C12P 19/34 - Polynucleotides, e.g. nucleic acids, oligoribonucleotides
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
6.
IONIZABLE LIPID COMPOUNDS AND LIPID NANOPARTICLE COMPOSITIONS
Novel ionizable lipids, compositions, and methods of using the novel ionizable lipids and compositions are disclosed. Lipid nanoparticle compositions include a novel ionizable lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticle compositions further including biologically active agents such as mRNA or DNA are useful in the delivery of biologically active agents to mammalian cells or organs.
C07D 251/04 - Heterocyclic compounds containing 1,3,5-triazine rings not condensed with other rings having no double bonds between ring members or between ring members and non-ring members
C07C 229/12 - Compounds containing amino and carboxyl groups bound to the same carbon skeleton having amino and carboxyl groups bound to acyclic carbon atoms of the same carbon skeleton the carbon skeleton being acyclic and saturated having only one amino and one carboxyl group bound to the carbon skeleton the nitrogen atom of the amino group being further bound to acyclic carbon atoms or to carbon atoms of rings other than six-membered aromatic rings to carbon atoms of acyclic carbon skeletons
B82Y 40/00 - Manufacture or treatment of nanostructures
7.
IONIZABLE LIPID COMPOUNDS AND LIPID NANOPARTICLE COMPOSITIONS
Novel ionizable lipids, compositions, and methods of using the novel ionizable lipids of formula (I) and compositions are disclosed. Lipid nanoparticle compositions include a novel ionizable lipid as well as additional lipids such as phospholipids, structural lipids, and PEG lipids. Lipid nanoparticle compositions further including biologically active agents such as mRNA or DNA are useful in the delivery of biologically active agents to mammalian cells or organs.
C07D 211/58 - Nitrogen atoms attached in position 4
C07D 241/12 - Heterocyclic compounds containing 1,4-diazine or hydrogenated 1,4-diazine rings not condensed with other rings having three double bonds between ring members or between ring members and non-ring members with only hydrogen atoms, hydrocarbon or substituted hydrocarbon radicals, directly attached to ring carbon atoms
C07D 401/06 - Heterocyclic compounds containing two or more hetero rings, having nitrogen atoms as the only ring hetero atoms, at least one ring being a six-membered ring with only one nitrogen atom containing two hetero rings linked by a carbon chain containing only aliphatic carbon atoms
C07C 229/00 - Compounds containing amino and carboxyl groups bound to the same carbon skeleton
C07C 215/00 - Compounds containing amino and hydroxy groups bound to the same carbon skeleton
C12N 15/88 - Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using liposome vesicle
01 - Chemical and biological materials for industrial, scientific and agricultural use
Goods & Services
Lipids used in the manufacture of cosmetics, beverages, food products and food supplements for industry and scientific use; Nucleotides for scientific and industrial research use in the nature of mRNA and lipid nanoparticle-mRNA; nanoparticles for industrial purposes and scientific use, namely, lipid nanoparticles for mRNA delivery