JP2019500430A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2019500430A5 JP2019500430A5 JP2018551890A JP2018551890A JP2019500430A5 JP 2019500430 A5 JP2019500430 A5 JP 2019500430A5 JP 2018551890 A JP2018551890 A JP 2018551890A JP 2018551890 A JP2018551890 A JP 2018551890A JP 2019500430 A5 JP2019500430 A5 JP 2019500430A5
- Authority
- JP
- Japan
- Prior art keywords
- alkyl
- alkenyl
- glycero
- pharmaceutical composition
- group
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Granted
Links
- 125000000217 alkyl group Chemical group 0.000 claims 28
- 125000003342 alkenyl group Chemical group 0.000 claims 24
- 239000008194 pharmaceutical composition Substances 0.000 claims 20
- 229910052799 carbon Inorganic materials 0.000 claims 12
- 102000019010 Methylmalonyl-CoA Mutase Human genes 0.000 claims 5
- 108010051862 Methylmalonyl-CoA mutase Proteins 0.000 claims 5
- -1 lipid compound Chemical class 0.000 claims 5
- 150000002632 lipids Chemical class 0.000 claims 5
- 150000003839 salts Chemical class 0.000 claims 5
- 150000001875 compounds Chemical class 0.000 claims 4
- 108020004999 messenger RNA Proteins 0.000 claims 4
- 229920001184 polypeptide Polymers 0.000 claims 4
- 102000004196 processed proteins & peptides Human genes 0.000 claims 4
- 108090000765 processed proteins & peptides Proteins 0.000 claims 4
- OILXMJHPFNGGTO-UHFFFAOYSA-N (22E)-(24xi)-24-methylcholesta-5,22-dien-3beta-ol Natural products C1C=C2CC(O)CCC2(C)C2C1C1CCC(C(C)C=CC(C)C(C)C)C1(C)CC2 OILXMJHPFNGGTO-UHFFFAOYSA-N 0.000 claims 3
- 108700026244 Open Reading Frames Proteins 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- FVXDQWZBHIXIEJ-LNDKUQBDSA-N 1,2-di-[(9Z,12Z)-octadecadienoyl]-sn-glycero-3-phosphocholine Chemical compound CCCCC\C=C/C\C=C/CCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCC\C=C/C\C=C/CCCCC FVXDQWZBHIXIEJ-LNDKUQBDSA-N 0.000 claims 2
- KILNVBDSWZSGLL-KXQOOQHDSA-N 1,2-dihexadecanoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCC KILNVBDSWZSGLL-KXQOOQHDSA-N 0.000 claims 2
- SNKAWJBJQDLSFF-NVKMUCNASA-N 1,2-dioleoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC SNKAWJBJQDLSFF-NVKMUCNASA-N 0.000 claims 2
- OQMZNAMGEHIHNN-UHFFFAOYSA-N 7-Dehydrostigmasterol Natural products C1C(O)CCC2(C)C(CCC3(C(C(C)C=CC(CC)C(C)C)CCC33)C)C3=CC=C21 OQMZNAMGEHIHNN-UHFFFAOYSA-N 0.000 claims 2
- 150000001413 amino acids Chemical group 0.000 claims 2
- 125000003118 aryl group Chemical group 0.000 claims 2
- LGJMUZUPVCAVPU-UHFFFAOYSA-N beta-Sitostanol Natural products C1CC2CC(O)CCC2(C)C2C1C1CCC(C(C)CCC(CC)C(C)C)C1(C)CC2 LGJMUZUPVCAVPU-UHFFFAOYSA-N 0.000 claims 2
- HVYWMOMLDIMFJA-DPAQBDIFSA-N cholesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CCCC(C)C)[C@@]1(C)CC2 HVYWMOMLDIMFJA-DPAQBDIFSA-N 0.000 claims 2
- 229960004956 glycerylphosphorylcholine Drugs 0.000 claims 2
- 125000001072 heteroaryl group Chemical group 0.000 claims 2
- 125000000623 heterocyclic group Chemical group 0.000 claims 2
- 150000003904 phospholipids Chemical class 0.000 claims 2
- 108091033319 polynucleotide Proteins 0.000 claims 2
- 239000002157 polynucleotide Substances 0.000 claims 2
- 102000040430 polynucleotide Human genes 0.000 claims 2
- NLQLSVXGSXCXFE-UHFFFAOYSA-N sitosterol Natural products CC=C(/CCC(C)C1CC2C3=CCC4C(C)C(O)CCC4(C)C3CCC2(C)C1)C(C)C NLQLSVXGSXCXFE-UHFFFAOYSA-N 0.000 claims 2
- GVJHHUAWPYXKBD-IEOSBIPESA-N α-tocopherol Chemical compound OC1=C(C)C(C)=C2O[C@@](CCC[C@H](C)CCC[C@H](C)CCCC(C)C)(C)CCC2=C1C GVJHHUAWPYXKBD-IEOSBIPESA-N 0.000 claims 2
- KZJWDPNRJALLNS-VPUBHVLGSA-N (-)-beta-Sitosterol Natural products O[C@@H]1CC=2[C@@](C)([C@@H]3[C@H]([C@H]4[C@@](C)([C@H]([C@H](CC[C@@H](C(C)C)CC)C)CC4)CC3)CC=2)CC1 KZJWDPNRJALLNS-VPUBHVLGSA-N 0.000 claims 1
- XLKQWAMTMYIQMG-SVUPRYTISA-N (2-{[(2r)-2,3-bis[(4z,7z,10z,13z,16z,19z)-docosa-4,7,10,13,16,19-hexaenoyloxy]propyl phosphonato]oxy}ethyl)trimethylazanium Chemical compound CC\C=C/C\C=C/C\C=C/C\C=C/C\C=C/C\C=C/CCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CC\C=C/C\C=C/C\C=C/C\C=C/C\C=C/C\C=C/CC XLKQWAMTMYIQMG-SVUPRYTISA-N 0.000 claims 1
- CSVWWLUMXNHWSU-UHFFFAOYSA-N (22E)-(24xi)-24-ethyl-5alpha-cholest-22-en-3beta-ol Natural products C1CC2CC(O)CCC2(C)C2C1C1CCC(C(C)C=CC(CC)C(C)C)C1(C)CC2 CSVWWLUMXNHWSU-UHFFFAOYSA-N 0.000 claims 1
- RQOCXCFLRBRBCS-UHFFFAOYSA-N (22E)-cholesta-5,7,22-trien-3beta-ol Natural products C1C(O)CCC2(C)C(CCC3(C(C(C)C=CCC(C)C)CCC33)C)C3=CC=C21 RQOCXCFLRBRBCS-UHFFFAOYSA-N 0.000 claims 1
- WCGUUGGRBIKTOS-GPOJBZKASA-N (3beta)-3-hydroxyurs-12-en-28-oic acid Chemical compound C1C[C@H](O)C(C)(C)[C@@H]2CC[C@@]3(C)[C@]4(C)CC[C@@]5(C(O)=O)CC[C@@H](C)[C@H](C)[C@H]5C4=CC[C@@H]3[C@]21C WCGUUGGRBIKTOS-GPOJBZKASA-N 0.000 claims 1
- LVNGJLRDBYCPGB-LDLOPFEMSA-N (R)-1,2-distearoylphosphatidylethanolamine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[NH3+])OC(=O)CCCCCCCCCCCCCCCCC LVNGJLRDBYCPGB-LDLOPFEMSA-N 0.000 claims 1
- SSCDRSKJTAQNNB-DWEQTYCFSA-N 1,2-di-(9Z,12Z-octadecadienoyl)-sn-glycero-3-phosphoethanolamine Chemical compound CCCCC\C=C/C\C=C/CCCCCCCC(=O)OC[C@H](COP(O)(=O)OCCN)OC(=O)CCCCCCC\C=C/C\C=C/CCCCC SSCDRSKJTAQNNB-DWEQTYCFSA-N 0.000 claims 1
- LZLVZIFMYXDKCN-QJWFYWCHSA-N 1,2-di-O-arachidonoyl-sn-glycero-3-phosphocholine Chemical compound CCCCC\C=C/C\C=C/C\C=C/C\C=C/CCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCC\C=C/C\C=C/C\C=C/C\C=C/CCCCC LZLVZIFMYXDKCN-QJWFYWCHSA-N 0.000 claims 1
- XXKFQTJOJZELMD-JICBSJGISA-N 1,2-di-[(9Z,12Z,15Z)-octadecatrienoyl]-sn-glycero-3-phosphocholine Chemical compound CC\C=C/C\C=C/C\C=C/CCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCC\C=C/C\C=C/C\C=C/CC XXKFQTJOJZELMD-JICBSJGISA-N 0.000 claims 1
- DSNRWDQKZIEDDB-SQYFZQSCSA-N 1,2-dioleoyl-sn-glycero-3-phospho-(1'-sn-glycerol) Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OC[C@H](COP(O)(=O)OC[C@@H](O)CO)OC(=O)CCCCCCC\C=C/CCCCCCCC DSNRWDQKZIEDDB-SQYFZQSCSA-N 0.000 claims 1
- MWRBNPKJOOWZPW-NYVOMTAGSA-N 1,2-dioleoyl-sn-glycero-3-phosphoethanolamine zwitterion Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OC[C@H](COP(O)(=O)OCCN)OC(=O)CCCCCCC\C=C/CCCCCCCC MWRBNPKJOOWZPW-NYVOMTAGSA-N 0.000 claims 1
- NRJAVPSFFCBXDT-HUESYALOSA-N 1,2-distearoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCCCC NRJAVPSFFCBXDT-HUESYALOSA-N 0.000 claims 1
- WTJKGGKOPKCXLL-VYOBOKEXSA-N 1-hexadecanoyl-2-(9Z-octadecenoyl)-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCC\C=C/CCCCCCCC WTJKGGKOPKCXLL-VYOBOKEXSA-N 0.000 claims 1
- XLPHMKQBBCKEFO-DHYROEPTSA-N 2-azaniumylethyl [(2r)-2,3-bis(3,7,11,15-tetramethylhexadecanoyloxy)propyl] phosphate Chemical compound CC(C)CCCC(C)CCCC(C)CCCC(C)CC(=O)OC[C@H](COP(O)(=O)OCCN)OC(=O)CC(C)CCCC(C)CCCC(C)CCCC(C)C XLPHMKQBBCKEFO-DHYROEPTSA-N 0.000 claims 1
- KLEXDBGYSOIREE-UHFFFAOYSA-N 24xi-n-propylcholesterol Natural products C1C=C2CC(O)CCC2(C)C2C1C1CCC(C(C)CCC(CCC)C(C)C)C1(C)CC2 KLEXDBGYSOIREE-UHFFFAOYSA-N 0.000 claims 1
- OILXMJHPFNGGTO-NRHJOKMGSA-N Brassicasterol Natural products O[C@@H]1CC=2[C@@](C)([C@@H]3[C@H]([C@H]4[C@](C)([C@H]([C@@H](/C=C/[C@H](C(C)C)C)C)CC4)CC3)CC=2)CC1 OILXMJHPFNGGTO-NRHJOKMGSA-N 0.000 claims 1
- SGNBVLSWZMBQTH-FGAXOLDCSA-N Campesterol Natural products O[C@@H]1CC=2[C@@](C)([C@@H]3[C@H]([C@H]4[C@@](C)([C@H]([C@H](CC[C@H](C(C)C)C)C)CC4)CC3)CC=2)CC1 SGNBVLSWZMBQTH-FGAXOLDCSA-N 0.000 claims 1
- OKTJSMMVPCPJKN-UHFFFAOYSA-N Carbon Chemical group [C] OKTJSMMVPCPJKN-UHFFFAOYSA-N 0.000 claims 1
- LPZCCMIISIBREI-MTFRKTCUSA-N Citrostadienol Natural products CC=C(CC[C@@H](C)[C@H]1CC[C@H]2C3=CC[C@H]4[C@H](C)[C@@H](O)CC[C@]4(C)[C@H]3CC[C@]12C)C(C)C LPZCCMIISIBREI-MTFRKTCUSA-N 0.000 claims 1
- ARVGMISWLZPBCH-UHFFFAOYSA-N Dehydro-beta-sitosterol Natural products C1C(O)CCC2(C)C(CCC3(C(C(C)CCC(CC)C(C)C)CCC33)C)C3=CC=C21 ARVGMISWLZPBCH-UHFFFAOYSA-N 0.000 claims 1
- GZDFHIJNHHMENY-UHFFFAOYSA-N Dimethyl dicarbonate Chemical compound COC(=O)OC(=O)OC GZDFHIJNHHMENY-UHFFFAOYSA-N 0.000 claims 1
- DNVPQKQSNYMLRS-NXVQYWJNSA-N Ergosterol Natural products CC(C)[C@@H](C)C=C[C@H](C)[C@H]1CC[C@H]2C3=CC=C4C[C@@H](O)CC[C@]4(C)[C@@H]3CC[C@]12C DNVPQKQSNYMLRS-NXVQYWJNSA-N 0.000 claims 1
- JZNWSCPGTDBMEW-UHFFFAOYSA-N Glycerophosphorylethanolamin Natural products NCCOP(O)(=O)OCC(O)CO JZNWSCPGTDBMEW-UHFFFAOYSA-N 0.000 claims 1
- BTEISVKTSQLKST-UHFFFAOYSA-N Haliclonasterol Natural products CC(C=CC(C)C(C)(C)C)C1CCC2C3=CC=C4CC(O)CCC4(C)C3CCC12C BTEISVKTSQLKST-UHFFFAOYSA-N 0.000 claims 1
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims 1
- XYNPYHXGMWJBLV-VXPJTDKGSA-N Tomatidine Chemical compound O([C@@H]1[C@@H]([C@]2(CC[C@@H]3[C@@]4(C)CC[C@H](O)C[C@@H]4CC[C@H]3[C@@H]2C1)C)[C@@H]1C)[C@@]11CC[C@H](C)CN1 XYNPYHXGMWJBLV-VXPJTDKGSA-N 0.000 claims 1
- QMGSCYSTMWRURP-UHFFFAOYSA-N Tomatine Natural products CC1CCC2(NC1)OC3CC4C5CCC6CC(CCC6(C)C5CCC4(C)C3C2C)OC7OC(CO)C(OC8OC(CO)C(O)C(OC9OCC(O)C(O)C9OC%10OC(CO)C(O)C(O)C%10O)C8O)C(O)C7O QMGSCYSTMWRURP-UHFFFAOYSA-N 0.000 claims 1
- OILXMJHPFNGGTO-ZRUUVFCLSA-N UNPD197407 Natural products C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)C=C[C@H](C)C(C)C)[C@@]1(C)CC2 OILXMJHPFNGGTO-ZRUUVFCLSA-N 0.000 claims 1
- HZYXFRGVBOPPNZ-UHFFFAOYSA-N UNPD88870 Natural products C1C=C2CC(O)CCC2(C)C2C1C1CCC(C(C)=CCC(CC)C(C)C)C1(C)CC2 HZYXFRGVBOPPNZ-UHFFFAOYSA-N 0.000 claims 1
- NJFCSWSRXWCWHV-USYZEHPZSA-N [(2R)-2,3-bis(octadec-1-enoxy)propyl] 2-(trimethylazaniumyl)ethyl phosphate Chemical compound CCCCCCCCCCCCCCCCC=COC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC=CCCCCCCCCCCCCCCCC NJFCSWSRXWCWHV-USYZEHPZSA-N 0.000 claims 1
- METWNYLCXCBBIU-IQKBSVDISA-N [(2R)-3-[ethoxy(hydroxy)phosphoryl]oxy-2-[(9Z,12Z,15Z)-octadeca-9,12,15-trienoyl]oxypropyl] (9Z,12Z,15Z)-octadeca-9,12,15-trienoate Chemical compound C(CCCCCCC\C=C/C\C=C/C\C=C/CC)(=O)OC[C@@H](OC(CCCCCCC\C=C/C\C=C/C\C=C/CC)=O)COP(=O)(O)OCC METWNYLCXCBBIU-IQKBSVDISA-N 0.000 claims 1
- SUTHKQVOHCMCCF-QZNUWAOFSA-N [(2r)-3-[2-aminoethoxy(hydroxy)phosphoryl]oxy-2-docosa-2,4,6,8,10,12-hexaenoyloxypropyl] docosa-2,4,6,8,10,12-hexaenoate Chemical compound CCCCCCCCCC=CC=CC=CC=CC=CC=CC(=O)OC[C@H](COP(O)(=O)OCCN)OC(=O)C=CC=CC=CC=CC=CC=CCCCCCCCCC SUTHKQVOHCMCCF-QZNUWAOFSA-N 0.000 claims 1
- LNUFLCYMSVYYNW-ZPJMAFJPSA-N [(2r,3r,4s,5r,6r)-2-[(2r,3r,4s,5r,6r)-6-[(2r,3r,4s,5r,6r)-6-[(2r,3r,4s,5r,6r)-6-[[(3s,5s,8r,9s,10s,13r,14s,17r)-10,13-dimethyl-17-[(2r)-6-methylheptan-2-yl]-2,3,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydro-1h-cyclopenta[a]phenanthren-3-yl]oxy]-4,5-disulfo Chemical compound O([C@@H]1[C@@H](COS(O)(=O)=O)O[C@@H]([C@@H]([C@H]1OS(O)(=O)=O)OS(O)(=O)=O)O[C@@H]1[C@@H](COS(O)(=O)=O)O[C@@H]([C@@H]([C@H]1OS(O)(=O)=O)OS(O)(=O)=O)O[C@@H]1[C@@H](COS(O)(=O)=O)O[C@H]([C@@H]([C@H]1OS(O)(=O)=O)OS(O)(=O)=O)O[C@@H]1C[C@@H]2CC[C@H]3[C@@H]4CC[C@@H]([C@]4(CC[C@@H]3[C@@]2(C)CC1)C)[C@H](C)CCCC(C)C)[C@H]1O[C@H](COS(O)(=O)=O)[C@@H](OS(O)(=O)=O)[C@H](OS(O)(=O)=O)[C@H]1OS(O)(=O)=O LNUFLCYMSVYYNW-ZPJMAFJPSA-N 0.000 claims 1
- 229940087168 alpha tocopherol Drugs 0.000 claims 1
- MJVXAPPOFPTTCA-UHFFFAOYSA-N beta-Sistosterol Natural products CCC(CCC(C)C1CCC2C3CC=C4C(C)C(O)CCC4(C)C3CCC12C)C(C)C MJVXAPPOFPTTCA-UHFFFAOYSA-N 0.000 claims 1
- NJKOMDUNNDKEAI-UHFFFAOYSA-N beta-sitosterol Natural products CCC(CCC(C)C1CCC2(C)C3CC=C4CC(O)CCC4C3CCC12C)C(C)C NJKOMDUNNDKEAI-UHFFFAOYSA-N 0.000 claims 1
- 235000004420 brassicasterol Nutrition 0.000 claims 1
- OILXMJHPFNGGTO-ZAUYPBDWSA-N brassicasterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)/C=C/[C@H](C)C(C)C)[C@@]1(C)CC2 OILXMJHPFNGGTO-ZAUYPBDWSA-N 0.000 claims 1
- 235000000431 campesterol Nutrition 0.000 claims 1
- SGNBVLSWZMBQTH-PODYLUTMSA-N campesterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CC[C@@H](C)C(C)C)[C@@]1(C)CC2 SGNBVLSWZMBQTH-PODYLUTMSA-N 0.000 claims 1
- 125000002837 carbocyclic group Chemical group 0.000 claims 1
- 150000001783 ceramides Chemical class 0.000 claims 1
- 235000012000 cholesterol Nutrition 0.000 claims 1
- 150000001982 diacylglycerols Chemical class 0.000 claims 1
- 125000005265 dialkylamine group Chemical class 0.000 claims 1
- 150000001985 dialkylglycerols Chemical class 0.000 claims 1
- DNVPQKQSNYMLRS-SOWFXMKYSA-N ergosterol Chemical compound C1[C@@H](O)CC[C@]2(C)[C@H](CC[C@]3([C@H]([C@H](C)/C=C/[C@@H](C)C(C)C)CC[C@H]33)C)C3=CC=C21 DNVPQKQSNYMLRS-SOWFXMKYSA-N 0.000 claims 1
- PEDCQBHIVMGVHV-UHFFFAOYSA-N glycerol Substances OCC(O)CO PEDCQBHIVMGVHV-UHFFFAOYSA-N 0.000 claims 1
- 125000000592 heterocycloalkyl group Chemical group 0.000 claims 1
- VLBPIWYTPAXCFJ-XMMPIXPASA-N lysophosphatidylcholine O-16:0/0:0 Chemical compound CCCCCCCCCCCCCCCCOC[C@@H](O)COP([O-])(=O)OCC[N+](C)(C)C VLBPIWYTPAXCFJ-XMMPIXPASA-N 0.000 claims 1
- 125000002496 methyl group Chemical group [H]C([H])([H])* 0.000 claims 1
- 201000003694 methylmalonic acidemia Diseases 0.000 claims 1
- 108091079658 miR-142-1 stem-loop Proteins 0.000 claims 1
- 108091071830 miR-142-2 stem-loop Proteins 0.000 claims 1
- 239000002105 nanoparticle Substances 0.000 claims 1
- 150000008103 phosphatidic acids Chemical class 0.000 claims 1
- 150000008104 phosphatidylethanolamines Chemical class 0.000 claims 1
- PWRIIDWSQYQFQD-UHFFFAOYSA-N sisunine Natural products CC1CCC2(NC1)OC3CC4C5CCC6CC(CCC6(C)C5CCC4(C)C3C2C)OC7OC(CO)C(OC8OC(CO)C(O)C(OC9OC(CO)C(O)C(O)C9OC%10OC(CO)C(O)C(O)C%10O)C8O)C(O)C7O PWRIIDWSQYQFQD-UHFFFAOYSA-N 0.000 claims 1
- 235000015500 sitosterol Nutrition 0.000 claims 1
- KZJWDPNRJALLNS-VJSFXXLFSA-N sitosterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)CC[C@@H](CC)C(C)C)[C@@]1(C)CC2 KZJWDPNRJALLNS-VJSFXXLFSA-N 0.000 claims 1
- 229950005143 sitosterol Drugs 0.000 claims 1
- 159000000000 sodium salts Chemical class 0.000 claims 1
- HCXVJBMSMIARIN-PHZDYDNGSA-N stigmasterol Chemical compound C1C=C2C[C@@H](O)CC[C@]2(C)[C@@H]2[C@@H]1[C@@H]1CC[C@H]([C@H](C)/C=C/[C@@H](CC)C(C)C)[C@@]1(C)CC2 HCXVJBMSMIARIN-PHZDYDNGSA-N 0.000 claims 1
- 235000016831 stigmasterol Nutrition 0.000 claims 1
- 229940032091 stigmasterol Drugs 0.000 claims 1
- BFDNMXAIBMJLBB-UHFFFAOYSA-N stigmasterol Natural products CCC(C=CC(C)C1CCCC2C3CC=C4CC(O)CCC4(C)C3CCC12C)C(C)C BFDNMXAIBMJLBB-UHFFFAOYSA-N 0.000 claims 1
- 229960000984 tocofersolan Drugs 0.000 claims 1
- XYNPYHXGMWJBLV-OFMODGJOSA-N tomatidine Natural products O[C@@H]1C[C@H]2[C@@](C)([C@@H]3[C@H]([C@H]4[C@@](C)([C@H]5[C@@H](C)[C@]6(O[C@H]5C4)NC[C@@H](C)CC6)CC3)CC2)CC1 XYNPYHXGMWJBLV-OFMODGJOSA-N 0.000 claims 1
- REJLGAUYTKNVJM-SGXCCWNXSA-N tomatine Chemical compound O([C@H]1[C@H](O)[C@@H](CO)O[C@H]([C@@H]1O[C@H]1[C@@H]([C@@H](O)[C@H](O)[C@@H](CO)O1)O)O[C@H]1[C@@H](CO)O[C@H]([C@@H]([C@H]1O)O)O[C@@H]1C[C@@H]2CC[C@H]3[C@@H]4C[C@H]5[C@@H]([C@]4(CC[C@@H]3[C@@]2(C)CC1)C)[C@@H]([C@@]1(NC[C@@H](C)CC1)O5)C)[C@@H]1OC[C@@H](O)[C@H](O)[C@H]1O REJLGAUYTKNVJM-SGXCCWNXSA-N 0.000 claims 1
- PLSAJKYPRJGMHO-UHFFFAOYSA-N ursolic acid Natural products CC1CCC2(CCC3(C)C(C=CC4C5(C)CCC(O)C(C)(C)C5CCC34C)C2C1C)C(=O)O PLSAJKYPRJGMHO-UHFFFAOYSA-N 0.000 claims 1
- 229940096998 ursolic acid Drugs 0.000 claims 1
- 239000002076 α-tocopherol Substances 0.000 claims 1
- 235000004835 α-tocopherol Nutrition 0.000 claims 1
Applications Claiming Priority (23)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201562269089P | 2015-12-17 | 2015-12-17 | |
| US201562269092P | 2015-12-17 | 2015-12-17 | |
| US62/269,089 | 2015-12-17 | ||
| US62/269,092 | 2015-12-17 | ||
| US201562273112P | 2015-12-30 | 2015-12-30 | |
| US201562273108P | 2015-12-30 | 2015-12-30 | |
| US62/273,112 | 2015-12-30 | ||
| US62/273,108 | 2015-12-30 | ||
| US201662274733P | 2016-01-04 | 2016-01-04 | |
| US201662274726P | 2016-01-04 | 2016-01-04 | |
| US201662274727P | 2016-01-04 | 2016-01-04 | |
| US201662274722P | 2016-01-04 | 2016-01-04 | |
| US62/274,726 | 2016-01-04 | ||
| US62/274,727 | 2016-01-04 | ||
| US62/274,722 | 2016-01-04 | ||
| US62/274,733 | 2016-01-04 | ||
| US201662338456P | 2016-05-18 | 2016-05-18 | |
| US201662338478P | 2016-05-18 | 2016-05-18 | |
| US62/338,478 | 2016-05-18 | ||
| US62/338,456 | 2016-05-18 | ||
| US201662409343P | 2016-10-17 | 2016-10-17 | |
| US62/409,343 | 2016-10-17 | ||
| PCT/US2016/067393 WO2017106799A1 (en) | 2015-12-17 | 2016-12-16 | POLYNUCLEOTIDES ENCODING METHYLMALONYL-CoA MUTASE |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2019500430A JP2019500430A (ja) | 2019-01-10 |
| JP2019500430A5 true JP2019500430A5 (enExample) | 2020-01-23 |
| JP7065036B2 JP7065036B2 (ja) | 2022-05-11 |
Family
ID=57738001
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018551890A Active JP7065036B2 (ja) | 2015-12-17 | 2016-12-16 | メチルマロニルCoAムターゼをコードするポリヌクレオチド |
Country Status (8)
| Country | Link |
|---|---|
| US (5) | US10406112B2 (enExample) |
| EP (1) | EP3390630B1 (enExample) |
| JP (1) | JP7065036B2 (enExample) |
| AU (1) | AU2016369612B2 (enExample) |
| CA (1) | CA3007108A1 (enExample) |
| IL (2) | IL259667B (enExample) |
| MA (1) | MA43568A (enExample) |
| WO (1) | WO2017106799A1 (enExample) |
Families Citing this family (94)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US10704021B2 (en) | 2012-03-15 | 2020-07-07 | Flodesign Sonics, Inc. | Acoustic perfusion devices |
| CA2935960C (en) | 2014-01-08 | 2023-01-10 | Bart Lipkens | Acoustophoresis device with dual acoustophoretic chamber |
| DK3134131T3 (en) | 2014-04-23 | 2022-02-07 | Modernatx Inc | Nucleic acid vaccines |
| US11377651B2 (en) | 2016-10-19 | 2022-07-05 | Flodesign Sonics, Inc. | Cell therapy processes utilizing acoustophoresis |
| US11708572B2 (en) | 2015-04-29 | 2023-07-25 | Flodesign Sonics, Inc. | Acoustic cell separation techniques and processes |
| US11364292B2 (en) | 2015-07-21 | 2022-06-21 | Modernatx, Inc. | CHIKV RNA vaccines |
| WO2017020026A1 (en) | 2015-07-30 | 2017-02-02 | Modernatx, Inc. | Concatemeric peptide epitopes rnas |
| US11564893B2 (en) | 2015-08-17 | 2023-01-31 | Modernatx, Inc. | Methods for preparing particles and related compositions |
| RS63030B1 (sr) | 2015-09-17 | 2022-04-29 | Modernatx Inc | Jedinjenja i kompozicije za intracelularno isporučivanje terapeutskih sredstava |
| EP3364950A4 (en) | 2015-10-22 | 2019-10-23 | ModernaTX, Inc. | VACCINES AGAINST TROPICAL DISEASES |
| MD3718565T2 (ro) | 2015-10-22 | 2022-09-30 | Modernatx Inc | Vaccinuri împotriva virusului respirator |
| CN108430456B (zh) | 2015-10-22 | 2022-01-18 | 摩登纳特斯有限公司 | 癌症疫苗 |
| WO2017070601A1 (en) | 2015-10-22 | 2017-04-27 | Modernatx, Inc. | Nucleic acid vaccines for varicella zoster virus (vzv) |
| EP3364981A4 (en) | 2015-10-22 | 2019-08-07 | ModernaTX, Inc. | VACCINE AGAINST THE HUMAN CYTOMEGALOVIRUS |
| CA3007955A1 (en) | 2015-12-10 | 2017-06-15 | Modernatx, Inc. | Lipid nanoparticles for delivery of therapeutic agents |
| EP3390630B1 (en) | 2015-12-17 | 2025-01-22 | ModernaTX, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
| JP7114465B2 (ja) | 2015-12-22 | 2022-08-08 | モデルナティエックス インコーポレイテッド | 薬剤の細胞内送達のための化合物および組成物 |
| US11214789B2 (en) | 2016-05-03 | 2022-01-04 | Flodesign Sonics, Inc. | Concentration and washing of particles with acoustics |
| MA45032A (fr) | 2016-05-18 | 2019-03-27 | Fundacion Para La Investig Medica Aplicada | Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë |
| US20190298657A1 (en) * | 2016-05-18 | 2019-10-03 | Modernatx, Inc. | Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency |
| US12128113B2 (en) | 2016-05-18 | 2024-10-29 | Modernatx, Inc. | Polynucleotides encoding JAGGED1 for the treatment of Alagille syndrome |
| US12123030B2 (en) | 2016-05-18 | 2024-10-22 | Modernatx, Inc. | Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia |
| US11001861B2 (en) | 2016-05-18 | 2021-05-11 | Modernatx, Inc. | Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1 |
| MA45053A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique |
| WO2017201349A1 (en) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
| JP6983455B2 (ja) | 2016-09-14 | 2021-12-17 | モデルナティーエックス, インコーポレイテッド | 高純度rna組成物及びその調製のための方法 |
| AU2017345766A1 (en) | 2016-10-21 | 2019-05-16 | Modernatx, Inc. | Human cytomegalovirus vaccine |
| US12491261B2 (en) | 2016-10-26 | 2025-12-09 | Acuitas Therapeutics, Inc. | Lipid nanoparticle formulations |
| US11583504B2 (en) | 2016-11-08 | 2023-02-21 | Modernatx, Inc. | Stabilized formulations of lipid nanoparticles |
| CN110167587A (zh) | 2016-11-11 | 2019-08-23 | 摩登纳特斯有限公司 | 流感疫苗 |
| EP3551193A4 (en) | 2016-12-08 | 2020-08-19 | Modernatx, Inc. | NUCLEIC ACID VACCINES AGAINST RESPIRATORY VIRUSES |
| EP3555289A1 (en) | 2016-12-13 | 2019-10-23 | ModernaTX, Inc. | Rna affinity purification |
| MA47787A (fr) | 2017-03-15 | 2020-01-22 | Modernatx Inc | Vaccin contre le virus respiratoire syncytial |
| US11752206B2 (en) * | 2017-03-15 | 2023-09-12 | Modernatx, Inc. | Herpes simplex virus vaccine |
| WO2018170270A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Varicella zoster virus (vzv) vaccine |
| WO2018170245A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Broad spectrum influenza virus vaccine |
| ES3020935T3 (en) | 2017-03-15 | 2025-05-23 | Modernatx Inc | Lipid nanoparticle formulation |
| CA3056133A1 (en) | 2017-03-15 | 2018-09-20 | Modernatx, Inc. | Crystal forms of amino lipids |
| KR20190132405A (ko) | 2017-03-15 | 2019-11-27 | 모더나티엑스, 인크. | 치료제의 세포내 전달을 위한 화합물 및 조성물 |
| WO2018170347A1 (en) | 2017-03-17 | 2018-09-20 | Modernatx, Inc. | Zoonotic disease rna vaccines |
| WO2018187590A1 (en) | 2017-04-05 | 2018-10-11 | Modernatx, Inc. | Reduction or elimination of immune responses to non-intravenous, e.g., subcutaneously administered therapeutic proteins |
| US12077501B2 (en) | 2017-06-14 | 2024-09-03 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of agents |
| US20200131498A1 (en) * | 2017-06-14 | 2020-04-30 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
| WO2018232357A1 (en) | 2017-06-15 | 2018-12-20 | Modernatx, Inc. | Rna formulations |
| CN111212905A (zh) | 2017-08-18 | 2020-05-29 | 摩登纳特斯有限公司 | Rna聚合酶变体 |
| WO2019036683A1 (en) | 2017-08-18 | 2019-02-21 | Modernatx, Inc. | ANALYTICAL METHODS BY HPLC |
| WO2019036685A1 (en) | 2017-08-18 | 2019-02-21 | Modernatx, Inc. | METHODS FOR HPLC ANALYSIS |
| WO2019046809A1 (en) | 2017-08-31 | 2019-03-07 | Modernatx, Inc. | METHODS OF MANUFACTURING LIPID NANOPARTICLES |
| EP3679379A2 (en) * | 2017-09-08 | 2020-07-15 | The U.S.A. as represented by the Secretary, Department of Health and Human Services | Biomarkers of organic acidemias |
| US10653767B2 (en) | 2017-09-14 | 2020-05-19 | Modernatx, Inc. | Zika virus MRNA vaccines |
| SG11202003907WA (en) | 2017-12-14 | 2020-05-28 | Flodesign Sonics Inc | Acoustic transducer drive and controller |
| JP7398115B2 (ja) | 2017-12-22 | 2023-12-14 | ノース カロライナ ステート ユニバーシティ | ポリマーフルオロフォア、それを含む組成物、ならびにその調製および使用方法 |
| WO2019148101A1 (en) | 2018-01-29 | 2019-08-01 | Modernatx, Inc. | Rsv rna vaccines |
| US20190314291A1 (en) * | 2018-01-30 | 2019-10-17 | Modernatx, Inc. | Compositions and methods for delivery of agents to immune cells |
| CN108704162A (zh) * | 2018-08-08 | 2018-10-26 | 中国科学院长春应用化学研究所 | 一种吸水敷料及其制备方法 |
| US12564645B2 (en) * | 2018-09-14 | 2026-03-03 | Translate Bio, Inc. | Methods for treatment of methylmalonic acidemia |
| AU2019345067A1 (en) | 2018-09-19 | 2021-04-08 | Modernatx, Inc. | High-purity peg lipids and uses thereof |
| WO2020061367A1 (en) | 2018-09-19 | 2020-03-26 | Modernatx, Inc. | Compounds and compositions for intracellular delivery of therapeutic agents |
| US12151029B2 (en) | 2018-09-19 | 2024-11-26 | Modernatx, Inc. | PEG lipids and uses thereof |
| WO2020061457A1 (en) | 2018-09-20 | 2020-03-26 | Modernatx, Inc. | Preparation of lipid nanoparticles and methods of administration thereof |
| US12331320B2 (en) | 2018-10-10 | 2025-06-17 | The Research Foundation For The State University Of New York | Genome edited cancer cell vaccines |
| US12583816B2 (en) | 2018-10-18 | 2026-03-24 | Acuitas Therapeutics, Inc. | Lipids for lipid nanoparticle delivery of active agents |
| EP3883592A1 (en) | 2018-11-21 | 2021-09-29 | Translate Bio, Inc. | Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr |
| JP2022521094A (ja) | 2019-02-20 | 2022-04-05 | モデルナティエックス インコーポレイテッド | 共転写キャッピング用rnaポリメラーゼバリアント |
| US11851694B1 (en) | 2019-02-20 | 2023-12-26 | Modernatx, Inc. | High fidelity in vitro transcription |
| US20220162278A1 (en) * | 2019-02-27 | 2022-05-26 | The Regents Of The University Of Michigan | Carbamoyl phosphate synthatase-1 for the treatment and prevention of liver injury |
| CN113874502A (zh) | 2019-03-11 | 2021-12-31 | 摩登纳特斯有限公司 | 补料分批体外转录方法 |
| WO2020190750A1 (en) | 2019-03-15 | 2020-09-24 | Modernatx, Inc. | Hiv rna vaccines |
| MX2022003269A (es) | 2019-09-19 | 2022-07-04 | Modernatx Inc | Compuestos lipidicos de cola ramificada y composiciones para la administracion intracelular de agentes terapeuticos. |
| WO2021055609A1 (en) | 2019-09-20 | 2021-03-25 | Translate Bio, Inc. | Mrna encoding engineered cftr |
| KR102924173B1 (ko) | 2020-04-09 | 2026-02-06 | 쑤저우 아보젠 바이오사이언시스 컴퍼니 리미티드 | 지질 나노입자 조성물 |
| WO2021204179A1 (en) | 2020-04-09 | 2021-10-14 | Suzhou Abogen Biosciences Co., Ltd. | Nucleic acid vaccines for coronavirus |
| CN113521269A (zh) | 2020-04-22 | 2021-10-22 | 生物技术Rna制药有限公司 | 冠状病毒疫苗 |
| JP7798855B2 (ja) | 2020-07-16 | 2026-01-14 | アクイタス セラピューティクス インコーポレイテッド | 脂質ナノ粒子に使用するためのカチオン性脂質 |
| US11406703B2 (en) | 2020-08-25 | 2022-08-09 | Modernatx, Inc. | Human cytomegalovirus vaccine |
| WO2022150717A1 (en) | 2021-01-11 | 2022-07-14 | Modernatx, Inc. | Seasonal rna influenza virus vaccines |
| US20240226025A1 (en) * | 2021-03-24 | 2024-07-11 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia |
| US20220363937A1 (en) | 2021-05-14 | 2022-11-17 | Armstrong World Industries, Inc. | Stabilization of antimicrobial coatings |
| US20240269248A1 (en) * | 2021-05-19 | 2024-08-15 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase for the treatment of methylmalonic acidemia |
| WO2022245980A1 (en) * | 2021-05-20 | 2022-11-24 | City Of Hope | Mir-142 compounds and uses thereof |
| IL309505A (en) | 2021-09-03 | 2024-02-01 | CureVac SE | Novel lipid nanoparticles for delivery of nucleic acids |
| US20250027108A1 (en) | 2021-10-29 | 2025-01-23 | CureVac SE | Improved circular rna for expressing therapeutic proteins |
| US12186387B2 (en) | 2021-11-29 | 2025-01-07 | BioNTech SE | Coronavirus vaccine |
| US12529047B1 (en) | 2021-12-21 | 2026-01-20 | Modernatx, Inc. | mRNA quantification methods |
| US20250099614A1 (en) | 2022-01-28 | 2025-03-27 | CureVac SE | Nucleic acid encoded transcription factor inhibitors |
| CA3248209A1 (en) | 2022-05-25 | 2023-11-30 | CureVac SE | Escherichia coli Antigenic Polypeptide-Based Nucleic Acid Vaccine (FIMH) |
| WO2024002985A1 (en) | 2022-06-26 | 2024-01-04 | BioNTech SE | Coronavirus vaccine |
| EP4608442A1 (en) | 2022-10-28 | 2025-09-03 | GlaxoSmithKline Biologicals S.A. | Nucleic acid based vaccine |
| JP2026509258A (ja) | 2023-03-08 | 2026-03-17 | キュアバック エスイー | 核酸送達のための新規な脂質ナノ粒子製剤 |
| WO2024230934A1 (en) | 2023-05-11 | 2024-11-14 | CureVac SE | Therapeutic nucleic acid for the treatment of ophthalmic diseases |
| WO2025064906A1 (en) * | 2023-09-21 | 2025-03-27 | The Board Of Regents Of The University Of Texas System | Endogenous mitochondria targeting signal peptides and uses thereof |
| WO2025126242A1 (en) * | 2023-12-11 | 2025-06-19 | Dr. Reddy's Institute Of Life Sciences | Nucleic acid sequence(s) and construct(s) for treating methylmalonic acidemia and method(s) thereof |
| CN118360253A (zh) * | 2024-05-11 | 2024-07-19 | 中山大学附属第六医院 | 一种干扰甲基丙二酰辅酶a变位酶表达的细胞系及其构建方法与应用 |
| WO2026014954A1 (ko) * | 2024-07-10 | 2026-01-15 | 주식회사 엣진 | 개선된 염기 교정 효율을 갖는 미토콘드리아 dna 염기 에디터 |
Family Cites Families (43)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5034506A (en) | 1985-03-15 | 1991-07-23 | Anti-Gene Development Group | Uncharged morpholino-based polymers having achiral intersubunit linkages |
| US5602240A (en) | 1990-07-27 | 1997-02-11 | Ciba Geigy Ag. | Backbone modified oligonucleotide analogs |
| US5489677A (en) | 1990-07-27 | 1996-02-06 | Isis Pharmaceuticals, Inc. | Oligonucleoside linkages containing adjacent oxygen and nitrogen atoms |
| WO1995027512A2 (en) | 1994-04-11 | 1995-10-19 | Baylor College Of Medicine | Compositions and methods for gene therapy to treat disease |
| US6652886B2 (en) | 2001-02-16 | 2003-11-25 | Expression Genetics | Biodegradable cationic copolymers of poly (alkylenimine) and poly (ethylene glycol) for the delivery of bioactive agents |
| WO2007024997A2 (en) | 2005-08-22 | 2007-03-01 | Fermalogic, Inc. | Methods of increasing production of secondary metabolites |
| HUE043492T2 (hu) | 2005-08-23 | 2019-08-28 | Univ Pennsylvania | Módosított nukleozidokat tartalmazó RNS és eljárások az alkalmazására |
| WO2009030254A1 (en) | 2007-09-04 | 2009-03-12 | Curevac Gmbh | Complexes of rna and cationic peptides for transfection and for immunostimulation |
| CN107028886A (zh) * | 2009-11-04 | 2017-08-11 | 不列颠哥伦比亚大学 | 含有核酸的脂质粒子及相关的方法 |
| PL3338765T3 (pl) | 2009-12-01 | 2019-06-28 | Translate Bio, Inc. | Pochodna steroidowa dla dostarczania mrna w ludzkich chorobach genetycznych |
| SE535869C2 (sv) * | 2010-04-01 | 2013-01-22 | Elways Ab | Ett för ett eller flera, elektriskt framdrivbara, fordon anpassat system (Laddningssystem). |
| WO2013086373A1 (en) * | 2011-12-07 | 2013-06-13 | Alnylam Pharmaceuticals, Inc. | Lipids for the delivery of active agents |
| SG11201404741VA (en) | 2012-02-07 | 2014-09-26 | Global Bio Therapeutics Usa Inc | Compartmentalized method of nucleic acid delivery and compositions and uses thereof |
| CN108949772A (zh) * | 2012-04-02 | 2018-12-07 | 现代泰克斯公司 | 用于产生与人类疾病相关的生物制剂和蛋白质的修饰多核苷酸 |
| WO2013151663A1 (en) * | 2012-04-02 | 2013-10-10 | modeRNA Therapeutics | Modified polynucleotides for the production of membrane proteins |
| US9255250B2 (en) * | 2012-12-05 | 2016-02-09 | Sangamo Bioscience, Inc. | Isolated mouse or human cell having an exogenous transgene in an endogenous albumin gene |
| JP2016504050A (ja) * | 2013-01-17 | 2016-02-12 | モデルナ セラピューティクス インコーポレイテッドModerna Therapeutics,Inc. | 細胞表現型の改変のためのシグナルセンサーポリヌクレオチド |
| WO2014151927A1 (en) * | 2013-03-14 | 2014-09-25 | Ipg Photonics Corporation | Universal fiber optic connector |
| ES2647832T3 (es) | 2013-03-14 | 2017-12-26 | Translate Bio, Inc. | Ácidos ribonucleicos con nucleótidos modificados con 4-tio y procedimientos relacionados |
| US9944918B2 (en) | 2013-03-15 | 2018-04-17 | The United States Of America, As Represented By The Secretary, Dept. Of Health & Human Services | Synthetic methylmalonyl-CoA mutase transgene for the treatment of MUT class methylmalonic acidemia (MMA) |
| EP2968602B1 (en) * | 2013-03-15 | 2017-08-30 | The United States of America, as represented by the Secretary, Department of Health and Human Services | Synthetic methylmalonyl-coa mutase transgene for the treatment of mut class methylmalonic acidemia (mma) |
| AU2014296278C1 (en) * | 2013-07-30 | 2023-02-02 | Genevant Sciences Gmbh | Block copolymers and their conjugates or complexes with oligonucleotides |
| IL316479A (en) * | 2014-06-25 | 2024-12-01 | Acuitas Therapeutics Inc | Novel lipids and lipid nanoparticle compositions for nucleic acid delivery |
| WO2016077125A1 (en) | 2014-11-10 | 2016-05-19 | Moderna Therapeutics, Inc. | Alternative nucleic acid molecules containing reduced uracil content and uses thereof |
| CN114642735A (zh) | 2015-01-21 | 2022-06-21 | 菲泽尔克斯公司 | 用于将治疗剂和诊断剂递送到细胞中的方法、组合物和系统 |
| WO2016172574A2 (en) * | 2015-04-24 | 2016-10-27 | The United States Of America, As Represented By The Secretary, Department Of Health And Human Services | Tagged form of mut enzyme, genetic constructs incorporating it, and its use in gene therapy |
| RS63030B1 (sr) | 2015-09-17 | 2022-04-29 | Modernatx Inc | Jedinjenja i kompozicije za intracelularno isporučivanje terapeutskih sredstava |
| RS63986B1 (sr) | 2015-10-28 | 2023-03-31 | Acuitas Therapeutics Inc | Novi lipidi i lipidne formulacije nanočestica za isporuku nukleinskih kiselina |
| WO2017100551A1 (en) | 2015-12-09 | 2017-06-15 | Alexion Pharmaceuticals, Inc. | HETEROLOGOUS UTR SEQUENCES FOR ENHANCED mRNA EXPRESSION |
| EP3390630B1 (en) | 2015-12-17 | 2025-01-22 | ModernaTX, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
| WO2017147720A1 (en) | 2016-03-04 | 2017-09-08 | Exerkine Corporation | Method for treating a central nervous system disorder |
| EP3426775A1 (en) | 2016-03-10 | 2019-01-16 | Novartis AG | Chemically modified messenger rna's |
| WO2017191274A2 (en) | 2016-05-04 | 2017-11-09 | Curevac Ag | Rna encoding a therapeutic protein |
| US20180126003A1 (en) | 2016-05-04 | 2018-05-10 | Curevac Ag | New targets for rna therapeutics |
| US20190298657A1 (en) | 2016-05-18 | 2019-10-03 | Modernatx, Inc. | Polynucleotides Encoding Acyl-CoA Dehydrogenase, Very Long-Chain for the Treatment of Very Long-Chain Acyl-CoA Dehydrogenase Deficiency |
| MA45053A (fr) | 2016-05-18 | 2019-03-27 | Modernatx Inc | Polynucléotides codant pour un régulateur de conductance transmembranaire de fibrose kystique pour le traitement de la fibrose kystique |
| US12123030B2 (en) | 2016-05-18 | 2024-10-22 | Modernatx, Inc. | Polynucleotides encoding lipoprotein lipase for the treatment of hyperlipidemia |
| US11001861B2 (en) | 2016-05-18 | 2021-05-11 | Modernatx, Inc. | Polynucleotides encoding galactose-1-phosphate uridylyltransferase for the treatment of galactosemia type 1 |
| MA45032A (fr) | 2016-05-18 | 2019-03-27 | Fundacion Para La Investig Medica Aplicada | Polynucléotides codant pour la porphobilinogène désaminase destinés au traitement de la porphyrie intermittente aiguë |
| WO2017201349A1 (en) | 2016-05-18 | 2017-11-23 | Modernatx, Inc. | Polynucleotides encoding citrin for the treatment of citrullinemia type 2 |
| US20200368162A1 (en) | 2017-02-24 | 2020-11-26 | Modernatx, Inc. | Nucleic Acid-Based Therapy of Muscular Dystrophies |
| US20200131498A1 (en) | 2017-06-14 | 2020-04-30 | Modernatx, Inc. | Polynucleotides encoding methylmalonyl-coa mutase |
| US12564645B2 (en) | 2018-09-14 | 2026-03-03 | Translate Bio, Inc. | Methods for treatment of methylmalonic acidemia |
-
2016
- 2016-12-16 EP EP16822592.8A patent/EP3390630B1/en active Active
- 2016-12-16 WO PCT/US2016/067393 patent/WO2017106799A1/en not_active Ceased
- 2016-12-16 JP JP2018551890A patent/JP7065036B2/ja active Active
- 2016-12-16 CA CA3007108A patent/CA3007108A1/en active Pending
- 2016-12-16 MA MA043568A patent/MA43568A/fr unknown
- 2016-12-16 AU AU2016369612A patent/AU2016369612B2/en not_active Ceased
-
2018
- 2018-05-29 IL IL259667A patent/IL259667B/en unknown
- 2018-06-07 US US16/002,376 patent/US10406112B2/en active Active
- 2018-06-07 US US16/002,472 patent/US10426738B2/en active Active
- 2018-08-27 US US16/113,814 patent/US20190022019A1/en not_active Abandoned
-
2019
- 2019-07-18 US US16/515,256 patent/US11504337B2/en active Active
-
2021
- 2021-12-19 IL IL289098A patent/IL289098A/en unknown
-
2022
- 2022-11-18 US US17/989,941 patent/US20230270685A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2019519601A5 (enExample) | ||
| JP2020514366A5 (enExample) | ||
| JPWO2016104580A1 (ja) | カチオン性脂質 | |
| JP2019508371A5 (enExample) | ||
| JP2020532528A5 (enExample) | ||
| JP2018532721A5 (enExample) | ||
| JP2022502451A5 (enExample) | ||
| JP2015515992A5 (enExample) | ||
| JP2019006815A5 (enExample) | ||
| CA2935914C (en) | Cationic lipid | |
| JP2023517275A5 (enExample) | ||
| JP2020517694A5 (enExample) | ||
| JP2018533573A5 (enExample) | ||
| JP2019516710A5 (enExample) | ||
| EP3083556A1 (en) | Lipids and lipid compositions for the delivery of active agents | |
| WO2018225871A1 (ja) | カチオン性脂質としての化合物 | |
| JP2021502346A (ja) | 生物学的活性分子を送達するための融合性化合物 | |
| WO2017222016A1 (ja) | カチオン性脂質 | |
| JP6240570B2 (ja) | 脂質粒子および核酸送達キャリア | |
| JP2025523587A (ja) | 核酸送達のための局所送達用カチオン性コレステロールを使用した脂質ナノ粒子 | |
| TW202428269A (zh) | 可離子化脂質及包含其的脂質奈米顆粒 | |
| EP4658636A1 (en) | Lipid compounds and uses thereof | |
| TWI760319B (zh) | 乳癌治療 | |
| JP2025516637A5 (enExample) | ||
| JP2024543272A (ja) | マルチモチーフデンドロン及びその超分子構造ならびにその使用 |