JPWO2021236479A5 - - Google Patents
Download PDFInfo
- Publication number
- JPWO2021236479A5 JPWO2021236479A5 JP2022570302A JP2022570302A JPWO2021236479A5 JP WO2021236479 A5 JPWO2021236479 A5 JP WO2021236479A5 JP 2022570302 A JP2022570302 A JP 2022570302A JP 2022570302 A JP2022570302 A JP 2022570302A JP WO2021236479 A5 JPWO2021236479 A5 JP WO2021236479A5
- Authority
- JP
- Japan
- Prior art keywords
- lipid
- pharma
- acceptable salt
- ceutically acceptable
- disease
- 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.)
- Pending
Links
- 150000002632 lipids Chemical class 0.000 claims description 172
- 239000002105 nanoparticle Substances 0.000 claims description 90
- 150000003839 salts Chemical class 0.000 claims description 44
- 125000000217 alkyl group Chemical group 0.000 claims description 22
- 230000007812 deficiency Effects 0.000 claims description 19
- 208000026350 Inborn Genetic disease Diseases 0.000 claims description 15
- 208000016361 genetic disease Diseases 0.000 claims description 15
- -1 cationic lipid Chemical class 0.000 claims description 14
- 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 description 12
- MBLBDJOUHNCFQT-UHFFFAOYSA-N N-acetyl-D-galactosamine Natural products CC(=O)NC(C=O)C(O)C(O)C(O)CO MBLBDJOUHNCFQT-UHFFFAOYSA-N 0.000 claims description 10
- MWRBNPKJOOWZPW-CLFAGFIQSA-N dioleoyl phosphatidylethanolamine Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OCC(COP(O)(=O)OCCN)OC(=O)CCCCCCC\C=C/CCCCCCCC MWRBNPKJOOWZPW-CLFAGFIQSA-N 0.000 claims description 10
- 239000002245 particle Substances 0.000 claims description 9
- 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 description 8
- 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 description 8
- 206010013801 Duchenne Muscular Dystrophy Diseases 0.000 claims description 8
- OVRNDRQMDRJTHS-KEWYIRBNSA-N N-acetyl-D-galactosamine Chemical compound CC(=O)N[C@H]1C(O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-KEWYIRBNSA-N 0.000 claims description 8
- 229930182558 Sterol Natural products 0.000 claims description 8
- 125000003342 alkenyl group Chemical group 0.000 claims description 8
- 108020004707 nucleic acids Proteins 0.000 claims description 8
- 102000039446 nucleic acids Human genes 0.000 claims description 8
- 150000007523 nucleic acids Chemical class 0.000 claims description 8
- 239000004055 small Interfering RNA Substances 0.000 claims description 8
- 150000003432 sterols Chemical class 0.000 claims description 8
- 235000003702 sterols Nutrition 0.000 claims description 8
- JZNWSCPGTDBMEW-UHFFFAOYSA-N Glycerophosphorylethanolamin Natural products NCCOP(O)(=O)OCC(O)CO JZNWSCPGTDBMEW-UHFFFAOYSA-N 0.000 claims description 6
- 206010053185 Glycogen storage disease type II Diseases 0.000 claims description 6
- 201000003533 Leber congenital amaurosis Diseases 0.000 claims description 6
- 108700011259 MicroRNAs Proteins 0.000 claims description 6
- 206010056886 Mucopolysaccharidosis I Diseases 0.000 claims description 6
- 206010028095 Mucopolysaccharidosis IV Diseases 0.000 claims description 6
- 239000004698 Polyethylene Substances 0.000 claims description 6
- 201000002150 Progressive familial intrahepatic cholestasis Diseases 0.000 claims description 6
- 125000002947 alkylene group Chemical group 0.000 claims description 6
- 235000012000 cholesterol Nutrition 0.000 claims description 6
- 125000001559 cyclopropyl group Chemical group [H]C1([H])C([H])([H])C1([H])* 0.000 claims description 6
- 239000003446 ligand Substances 0.000 claims description 6
- 239000002679 microRNA Substances 0.000 claims description 6
- 208000022018 mucopolysaccharidosis type 2 Diseases 0.000 claims description 6
- 239000008194 pharmaceutical composition Substances 0.000 claims description 6
- WTJKGGKOPKCXLL-RRHRGVEJSA-N phosphatidylcholine Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCC=CCCCCCCCC WTJKGGKOPKCXLL-RRHRGVEJSA-N 0.000 claims description 6
- 230000008685 targeting Effects 0.000 claims description 6
- 102100022641 Coagulation factor IX Human genes 0.000 claims description 5
- 239000000203 mixture Substances 0.000 claims description 5
- SDEURMLKLAEUAY-JFSPZUDSSA-N (2-{[(2r)-2,3-bis[(13z)-docos-13-enoyloxy]propyl phosphonato]oxy}ethyl)trimethylazanium Chemical compound CCCCCCCC\C=C/CCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCC\C=C/CCCCCCCC SDEURMLKLAEUAY-JFSPZUDSSA-N 0.000 claims description 4
- 125000003161 (C1-C6) alkylene group Chemical group 0.000 claims description 4
- 125000005913 (C3-C6) cycloalkyl group Chemical group 0.000 claims description 4
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims description 4
- CITHEXJVPOWHKC-UUWRZZSWSA-N 1,2-di-O-myristoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCC(=O)OC[C@H](COP([O-])(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCC CITHEXJVPOWHKC-UUWRZZSWSA-N 0.000 claims description 4
- 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 description 4
- SLKDGVPOSSLUAI-PGUFJCEWSA-N 1,2-dihexadecanoyl-sn-glycero-3-phosphoethanolamine zwitterion Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP(O)(=O)OCCN)OC(=O)CCCCCCCCCCCCCCC SLKDGVPOSSLUAI-PGUFJCEWSA-N 0.000 claims description 4
- WTBFLCSPLLEDEM-JIDRGYQWSA-N 1,2-dioleoyl-sn-glycero-3-phospho-L-serine Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OC[C@H](COP(O)(=O)OC[C@H](N)C(O)=O)OC(=O)CCCCCCC\C=C/CCCCCCCC WTBFLCSPLLEDEM-JIDRGYQWSA-N 0.000 claims description 4
- LVNGJLRDBYCPGB-UHFFFAOYSA-N 1,2-distearoylphosphatidylethanolamine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OCC(COP([O-])(=O)OCC[NH3+])OC(=O)CCCCCCCCCCCCCCCCC LVNGJLRDBYCPGB-UHFFFAOYSA-N 0.000 claims description 4
- BIABMEZBCHDPBV-MPQUPPDSSA-N 1,2-palmitoyl-sn-glycero-3-phospho-(1'-sn-glycerol) Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP(O)(=O)OC[C@@H](O)CO)OC(=O)CCCCCCCCCCCCCCC BIABMEZBCHDPBV-MPQUPPDSSA-N 0.000 claims description 4
- 201000006935 Becker muscular dystrophy Diseases 0.000 claims description 4
- 102100022548 Beta-hexosaminidase subunit alpha Human genes 0.000 claims description 4
- 108010039209 Blood Coagulation Factors Proteins 0.000 claims description 4
- 102000015081 Blood Coagulation Factors Human genes 0.000 claims description 4
- 208000010975 Dystrophic epidermolysis bullosa Diseases 0.000 claims description 4
- 201000003542 Factor VIII deficiency Diseases 0.000 claims description 4
- 208000033173 Generalized arterial calcification of infancy Diseases 0.000 claims description 4
- 208000032007 Glycogen storage disease due to acid maltase deficiency Diseases 0.000 claims description 4
- 208000009292 Hemophilia A Diseases 0.000 claims description 4
- 208000015204 Hurler-Scheie syndrome Diseases 0.000 claims description 4
- 102100024640 Low-density lipoprotein receptor Human genes 0.000 claims description 4
- 102100026001 Lysosomal acid lipase/cholesteryl ester hydrolase Human genes 0.000 claims description 4
- 208000008955 Mucolipidoses Diseases 0.000 claims description 4
- 206010072927 Mucolipidosis type I Diseases 0.000 claims description 4
- RWKUXQNLWDTSLO-GWQJGLRPSA-N N-hexadecanoylsphingosine-1-phosphocholine Chemical compound CCCCCCCCCCCCCCCC(=O)N[C@@H](COP([O-])(=O)OCC[N+](C)(C)C)[C@H](O)\C=C\CCCCCCCCCCCCC RWKUXQNLWDTSLO-GWQJGLRPSA-N 0.000 claims description 4
- 208000002537 Neuronal Ceroid-Lipofuscinoses Diseases 0.000 claims description 4
- 108091034117 Oligonucleotide Proteins 0.000 claims description 4
- 102000007981 Ornithine carbamoyltransferase Human genes 0.000 claims description 4
- 101710198224 Ornithine carbamoyltransferase, mitochondrial Proteins 0.000 claims description 4
- FVJZSBGHRPJMMA-IOLBBIBUSA-N PG(18:0/18:0) Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP(O)(=O)OC[C@@H](O)CO)OC(=O)CCCCCCCCCCCCCCCCC FVJZSBGHRPJMMA-IOLBBIBUSA-N 0.000 claims description 4
- 201000002883 Scheie syndrome Diseases 0.000 claims description 4
- 108091027967 Small hairpin RNA Proteins 0.000 claims description 4
- 108020004459 Small interfering RNA Proteins 0.000 claims description 4
- 108010055297 Sterol Esterase Proteins 0.000 claims description 4
- DSNRWDQKZIEDDB-GCMPNPAFSA-N [(2r)-3-[2,3-dihydroxypropoxy(hydroxy)phosphoryl]oxy-2-[(z)-octadec-9-enoyl]oxypropyl] (z)-octadec-9-enoate Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OC[C@H](COP(O)(=O)OCC(O)CO)OC(=O)CCCCCCC\C=C/CCCCCCCC DSNRWDQKZIEDDB-GCMPNPAFSA-N 0.000 claims description 4
- NONFBHXKNNVFMO-UHFFFAOYSA-N [2-aminoethoxy(tetradecanoyloxy)phosphoryl] tetradecanoate Chemical compound CCCCCCCCCCCCCC(=O)OP(=O)(OCCN)OC(=O)CCCCCCCCCCCCC NONFBHXKNNVFMO-UHFFFAOYSA-N 0.000 claims description 4
- MWRBNPKJOOWZPW-XPWSMXQVSA-N [3-[2-aminoethoxy(hydroxy)phosphoryl]oxy-2-[(e)-octadec-9-enoyl]oxypropyl] (e)-octadec-9-enoate Chemical compound CCCCCCCC\C=C\CCCCCCCC(=O)OCC(COP(O)(=O)OCCN)OC(=O)CCCCCCC\C=C\CCCCCCCC MWRBNPKJOOWZPW-XPWSMXQVSA-N 0.000 claims description 4
- 206010002026 amyotrophic lateral sclerosis Diseases 0.000 claims description 4
- 239000000074 antisense oligonucleotide Substances 0.000 claims description 4
- 238000012230 antisense oligonucleotides Methods 0.000 claims description 4
- 208000004900 arterial calcification of infancy Diseases 0.000 claims description 4
- 239000003114 blood coagulation factor Substances 0.000 claims description 4
- 229960003724 dimyristoylphosphatidylcholine Drugs 0.000 claims description 4
- 229960005160 dimyristoylphosphatidylglycerol Drugs 0.000 claims description 4
- BPHQZTVXXXJVHI-AJQTZOPKSA-N ditetradecanoyl phosphatidylglycerol Chemical compound CCCCCCCCCCCCCC(=O)OC[C@H](COP(O)(=O)OC[C@@H](O)CO)OC(=O)CCCCCCCCCCCCC BPHQZTVXXXJVHI-AJQTZOPKSA-N 0.000 claims description 4
- 208000004298 epidermolysis bullosa dystrophica Diseases 0.000 claims description 4
- 229960004222 factor ix Drugs 0.000 claims description 4
- 229960000301 factor viii Drugs 0.000 claims description 4
- 201000004502 glycogen storage disease II Diseases 0.000 claims description 4
- 208000009429 hemophilia B Diseases 0.000 claims description 4
- 201000002273 mucopolysaccharidosis II Diseases 0.000 claims description 4
- 208000025919 mucopolysaccharidosis type 7 Diseases 0.000 claims description 4
- 239000000546 pharmaceutical excipient Substances 0.000 claims description 4
- 150000008104 phosphatidylethanolamines Chemical class 0.000 claims description 4
- 239000013603 viral vector Substances 0.000 claims description 4
- ZLGYVWRJIZPQMM-HHHXNRCGSA-N 2-azaniumylethyl [(2r)-2,3-di(dodecanoyloxy)propyl] phosphate Chemical compound CCCCCCCCCCCC(=O)OC[C@H](COP(O)(=O)OCCN)OC(=O)CCCCCCCCCCC ZLGYVWRJIZPQMM-HHHXNRCGSA-N 0.000 claims description 3
- 102100026735 Coagulation factor VIII Human genes 0.000 claims description 3
- 208000002972 Hepatolenticular Degeneration Diseases 0.000 claims description 3
- 101000911390 Homo sapiens Coagulation factor VIII Proteins 0.000 claims description 3
- 101000801643 Homo sapiens Retinal-specific phospholipid-transporting ATPase ABCA4 Proteins 0.000 claims description 3
- 102100033448 Lysosomal alpha-glucosidase Human genes 0.000 claims description 3
- 208000035719 Maculopathy Diseases 0.000 claims description 3
- 201000011252 Phenylketonuria Diseases 0.000 claims description 3
- 102100033617 Retinal-specific phospholipid-transporting ATPase ABCA4 Human genes 0.000 claims description 3
- 208000014769 Usher Syndromes Diseases 0.000 claims description 3
- 208000018839 Wilson disease Diseases 0.000 claims description 3
- 208000002780 macular degeneration Diseases 0.000 claims description 3
- 208000012253 mucopolysaccharidosis IVA Diseases 0.000 claims description 3
- 208000012091 mucopolysaccharidosis type IVB Diseases 0.000 claims description 3
- 208000007056 sickle cell anemia Diseases 0.000 claims description 3
- ASWBNKHCZGQVJV-UHFFFAOYSA-N (3-hexadecanoyloxy-2-hydroxypropyl) 2-(trimethylazaniumyl)ethyl phosphate Chemical compound CCCCCCCCCCCCCCCC(=O)OCC(O)COP([O-])(=O)OCC[N+](C)(C)C ASWBNKHCZGQVJV-UHFFFAOYSA-N 0.000 claims description 2
- 125000004169 (C1-C6) alkyl group Chemical group 0.000 claims description 2
- 102000040650 (ribonucleotides)n+m Human genes 0.000 claims description 2
- 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 description 2
- PORPENFLTBBHSG-MGBGTMOVSA-N 1,2-dihexadecanoyl-sn-glycerol-3-phosphate Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP(O)(O)=O)OC(=O)CCCCCCCCCCCCCCC PORPENFLTBBHSG-MGBGTMOVSA-N 0.000 claims description 2
- TZCPCKNHXULUIY-RGULYWFUSA-N 1,2-distearoyl-sn-glycero-3-phosphoserine Chemical compound CCCCCCCCCCCCCCCCCC(=O)OC[C@H](COP(O)(=O)OC[C@H](N)C(O)=O)OC(=O)CCCCCCCCCCCCCCCCC TZCPCKNHXULUIY-RGULYWFUSA-N 0.000 claims description 2
- PAZGBAOHGQRCBP-ZCXUNETKSA-N 1-Palmitoyl-2-oleoylglycero-3-phosphoglycerol Chemical compound CCCCCCCCCCCCCCCC(=O)OCC(COP(O)(=O)OCC(O)CO)OC(=O)CCCCCCC\C=C/CCCCCCCC PAZGBAOHGQRCBP-ZCXUNETKSA-N 0.000 claims description 2
- RYCNUMLMNKHWPZ-SNVBAGLBSA-N 1-acetyl-sn-glycero-3-phosphocholine Chemical compound CC(=O)OC[C@@H](O)COP([O-])(=O)OCC[N+](C)(C)C RYCNUMLMNKHWPZ-SNVBAGLBSA-N 0.000 claims description 2
- IIZPXYDJLKNOIY-JXPKJXOSSA-N 1-palmitoyl-2-arachidonoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCC(=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 IIZPXYDJLKNOIY-JXPKJXOSSA-N 0.000 claims description 2
- 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 description 2
- CFWRDBDJAOHXSH-SECBINFHSA-N 2-azaniumylethyl [(2r)-2,3-diacetyloxypropyl] phosphate Chemical compound CC(=O)OC[C@@H](OC(C)=O)COP(O)(=O)OCCN CFWRDBDJAOHXSH-SECBINFHSA-N 0.000 claims description 2
- 208000029602 Alpha-N-acetylgalactosaminidase deficiency Diseases 0.000 claims description 2
- 208000024827 Alzheimer disease Diseases 0.000 claims description 2
- 208000031277 Amaurotic familial idiocy Diseases 0.000 claims description 2
- 206010068220 Aspartylglucosaminuria Diseases 0.000 claims description 2
- 206010003594 Ataxia telangiectasia Diseases 0.000 claims description 2
- 102000014461 Ataxins Human genes 0.000 claims description 2
- 108010078286 Ataxins Proteins 0.000 claims description 2
- 102100028282 Bile salt export pump Human genes 0.000 claims description 2
- 208000005692 Bloom Syndrome Diseases 0.000 claims description 2
- 125000000739 C2-C30 alkenyl group Chemical group 0.000 claims description 2
- 108090000994 Catalytic RNA Proteins 0.000 claims description 2
- 102000053642 Catalytic RNA Human genes 0.000 claims description 2
- 102000005572 Cathepsin A Human genes 0.000 claims description 2
- 108010059081 Cathepsin A Proteins 0.000 claims description 2
- 206010008025 Cerebellar ataxia Diseases 0.000 claims description 2
- 206010010356 Congenital anomaly Diseases 0.000 claims description 2
- 206010053138 Congenital aplastic anaemia Diseases 0.000 claims description 2
- 201000003883 Cystic fibrosis Diseases 0.000 claims description 2
- 206010011777 Cystinosis Diseases 0.000 claims description 2
- 208000011518 Danon disease Diseases 0.000 claims description 2
- 208000024720 Fabry Disease Diseases 0.000 claims description 2
- 108010076282 Factor IX Proteins 0.000 claims description 2
- 108010054218 Factor VIII Proteins 0.000 claims description 2
- 102000001690 Factor VIII Human genes 0.000 claims description 2
- 201000004939 Fanconi anemia Diseases 0.000 claims description 2
- 208000024412 Friedreich ataxia Diseases 0.000 claims description 2
- 208000001905 GM2 Gangliosidoses Diseases 0.000 claims description 2
- 201000008905 GM2 gangliosidosis Diseases 0.000 claims description 2
- 208000017462 Galactosialidosis Diseases 0.000 claims description 2
- 208000015872 Gaucher disease Diseases 0.000 claims description 2
- 208000010055 Globoid Cell Leukodystrophy Diseases 0.000 claims description 2
- ZWZWYGMENQVNFU-UHFFFAOYSA-N Glycerophosphorylserin Natural products OC(=O)C(N)COP(O)(=O)OCC(O)CO ZWZWYGMENQVNFU-UHFFFAOYSA-N 0.000 claims description 2
- 208000001500 Glycogen Storage Disease Type IIb Diseases 0.000 claims description 2
- 208000035148 Glycogen storage disease due to LAMP-2 deficiency Diseases 0.000 claims description 2
- 101001045440 Homo sapiens Beta-hexosaminidase subunit alpha Proteins 0.000 claims description 2
- 101000701363 Homo sapiens Phospholipid-transporting ATPase IC Proteins 0.000 claims description 2
- 101000785523 Homo sapiens Tight junction protein ZO-2 Proteins 0.000 claims description 2
- 208000023105 Huntington disease Diseases 0.000 claims description 2
- 208000015178 Hurler syndrome Diseases 0.000 claims description 2
- 108700037017 Hyaluronidase Deficiency Proteins 0.000 claims description 2
- 208000005503 Hyaluronidase deficiency Diseases 0.000 claims description 2
- 208000000563 Hyperlipoproteinemia Type II Diseases 0.000 claims description 2
- 208000022559 Inflammatory bowel disease Diseases 0.000 claims description 2
- 208000028226 Krabbe disease Diseases 0.000 claims description 2
- 108010001831 LDL receptors Proteins 0.000 claims description 2
- 208000009625 Lesch-Nyhan syndrome Diseases 0.000 claims description 2
- 102100038225 Lysosome-associated membrane glycoprotein 2 Human genes 0.000 claims description 2
- 101710116771 Lysosome-associated membrane glycoprotein 2 Proteins 0.000 claims description 2
- 108010093662 Member 11 Subfamily B ATP Binding Cassette Transporter Proteins 0.000 claims description 2
- 201000011442 Metachromatic leukodystrophy Diseases 0.000 claims description 2
- 208000002678 Mucopolysaccharidoses Diseases 0.000 claims description 2
- 206010056893 Mucopolysaccharidosis VII Diseases 0.000 claims description 2
- 208000025915 Mucopolysaccharidosis type 6 Diseases 0.000 claims description 2
- OVRNDRQMDRJTHS-CBQIKETKSA-N N-Acetyl-D-Galactosamine Chemical compound CC(=O)N[C@H]1[C@@H](O)O[C@H](CO)[C@H](O)[C@@H]1O OVRNDRQMDRJTHS-CBQIKETKSA-N 0.000 claims description 2
- 208000014060 Niemann-Pick disease Diseases 0.000 claims description 2
- 208000018737 Parkinson disease Diseases 0.000 claims description 2
- 102100030448 Phospholipid-transporting ATPase IC Human genes 0.000 claims description 2
- 239000002202 Polyethylene glycol Substances 0.000 claims description 2
- 102000009609 Pyrophosphatases Human genes 0.000 claims description 2
- 108010009413 Pyrophosphatases Proteins 0.000 claims description 2
- 208000007014 Retinitis pigmentosa Diseases 0.000 claims description 2
- 201000000582 Retinoblastoma Diseases 0.000 claims description 2
- 208000013608 Salla disease Diseases 0.000 claims description 2
- 208000021811 Sandhoff disease Diseases 0.000 claims description 2
- 208000000828 Sialic Acid Storage Disease Diseases 0.000 claims description 2
- 201000001828 Sly syndrome Diseases 0.000 claims description 2
- 208000010346 Sphingolipidoses Diseases 0.000 claims description 2
- 201000001307 Sphingolipidosis Diseases 0.000 claims description 2
- 208000009415 Spinocerebellar Ataxias Diseases 0.000 claims description 2
- 208000022292 Tay-Sachs disease Diseases 0.000 claims description 2
- 208000002903 Thalassemia Diseases 0.000 claims description 2
- 102100026637 Tight junction protein ZO-2 Human genes 0.000 claims description 2
- 208000035317 Total hypoxanthine-guanine phosphoribosyl transferase deficiency Diseases 0.000 claims description 2
- 108700001567 Type I Schindler Disease Proteins 0.000 claims description 2
- 206010045261 Type IIa hyperlipidaemia Diseases 0.000 claims description 2
- 108020000999 Viral RNA Proteins 0.000 claims description 2
- 201000006083 Xeroderma Pigmentosum Diseases 0.000 claims description 2
- CWRILEGKIAOYKP-SSDOTTSWSA-M [(2r)-3-acetyloxy-2-hydroxypropyl] 2-aminoethyl phosphate Chemical compound CC(=O)OC[C@@H](O)COP([O-])(=O)OCCN CWRILEGKIAOYKP-SSDOTTSWSA-M 0.000 claims description 2
- ATBOMIWRCZXYSZ-XZBBILGWSA-N [1-[2,3-dihydroxypropoxy(hydroxy)phosphoryl]oxy-3-hexadecanoyloxypropan-2-yl] (9e,12e)-octadeca-9,12-dienoate Chemical compound CCCCCCCCCCCCCCCC(=O)OCC(COP(O)(=O)OCC(O)CO)OC(=O)CCCCCCC\C=C\C\C=C\CCCCC ATBOMIWRCZXYSZ-XZBBILGWSA-N 0.000 claims description 2
- 201000010275 acute porphyria Diseases 0.000 claims description 2
- 208000006682 alpha 1-Antitrypsin Deficiency Diseases 0.000 claims description 2
- AWUCVROLDVIAJX-UHFFFAOYSA-N alpha-glycerophosphate Natural products OCC(O)COP(O)(O)=O AWUCVROLDVIAJX-UHFFFAOYSA-N 0.000 claims description 2
- 201000004562 autosomal dominant cerebellar ataxia Diseases 0.000 claims description 2
- 229940076810 beta sitosterol Drugs 0.000 claims description 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 description 2
- 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 description 2
- 229930183167 cerebroside Natural products 0.000 claims description 2
- 125000004093 cyano group Chemical group *C#N 0.000 claims description 2
- RNPXCFINMKSQPQ-UHFFFAOYSA-N dicetyl hydrogen phosphate Chemical compound CCCCCCCCCCCCCCCCOP(O)(=O)OCCCCCCCCCCCCCCCC RNPXCFINMKSQPQ-UHFFFAOYSA-N 0.000 claims description 2
- 229940093541 dicetylphosphate Drugs 0.000 claims description 2
- ZGSPNIOCEDOHGS-UHFFFAOYSA-L disodium [3-[2,3-di(octadeca-9,12-dienoyloxy)propoxy-oxidophosphoryl]oxy-2-hydroxypropyl] 2,3-di(octadeca-9,12-dienoyloxy)propyl phosphate Chemical compound [Na+].[Na+].CCCCCC=CCC=CCCCCCCCC(=O)OCC(OC(=O)CCCCCCCC=CCC=CCCCCC)COP([O-])(=O)OCC(O)COP([O-])(=O)OCC(OC(=O)CCCCCCCC=CCC=CCCCCC)COC(=O)CCCCCCCC=CCC=CCCCCC ZGSPNIOCEDOHGS-UHFFFAOYSA-L 0.000 claims description 2
- 201000001386 familial hypercholesterolemia Diseases 0.000 claims description 2
- 208000007345 glycogen storage disease Diseases 0.000 claims description 2
- 201000008977 glycoproteinosis Diseases 0.000 claims description 2
- 125000005843 halogen group Chemical group 0.000 claims description 2
- 208000033552 hepatic porphyria Diseases 0.000 claims description 2
- 208000006359 hepatoblastoma Diseases 0.000 claims description 2
- 125000000623 heterocyclic group Chemical group 0.000 claims description 2
- 208000017482 infantile neuronal ceroid lipofuscinosis Diseases 0.000 claims description 2
- 230000002452 interceptive effect Effects 0.000 claims description 2
- 208000017476 juvenile neuronal ceroid lipofuscinosis Diseases 0.000 claims description 2
- 208000025014 late infantile neuronal ceroid lipofuscinosis Diseases 0.000 claims description 2
- 229940067606 lecithin Drugs 0.000 claims description 2
- 239000000787 lecithin Substances 0.000 claims description 2
- 235000010445 lecithin Nutrition 0.000 claims description 2
- 210000004185 liver Anatomy 0.000 claims description 2
- 201000001441 melanoma Diseases 0.000 claims description 2
- 108020004999 messenger RNA Proteins 0.000 claims description 2
- 208000030159 metabolic disease Diseases 0.000 claims description 2
- 201000007769 mucolipidosis Diseases 0.000 claims description 2
- 206010028093 mucopolysaccharidosis Diseases 0.000 claims description 2
- 208000000690 mucopolysaccharidosis VI Diseases 0.000 claims description 2
- 208000011045 mucopolysaccharidosis type 3 Diseases 0.000 claims description 2
- 201000008051 neuronal ceroid lipofuscinosis Diseases 0.000 claims description 2
- 201000007607 neuronal ceroid lipofuscinosis 3 Diseases 0.000 claims description 2
- 125000004433 nitrogen atom Chemical group N* 0.000 claims description 2
- 150000003905 phosphatidylinositols Chemical class 0.000 claims description 2
- 239000013612 plasmid Substances 0.000 claims description 2
- 229920001223 polyethylene glycol Polymers 0.000 claims description 2
- 108091092562 ribozyme Proteins 0.000 claims description 2
- 208000011985 sialidosis Diseases 0.000 claims description 2
- 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 description 2
- 229950005143 sitosterol Drugs 0.000 claims description 2
- 208000002320 spinal muscular atrophy Diseases 0.000 claims description 2
- 239000000758 substrate Substances 0.000 claims description 2
- 239000013598 vector Substances 0.000 claims description 2
- JSPNNZKWADNWHI-PNANGNLXSA-N (2r)-2-hydroxy-n-[(2s,3r,4e,8e)-3-hydroxy-9-methyl-1-[(2r,3r,4s,5s,6r)-3,4,5-trihydroxy-6-(hydroxymethyl)oxan-2-yl]oxyoctadeca-4,8-dien-2-yl]heptadecanamide Chemical compound CCCCCCCCCCCCCCC[C@@H](O)C(=O)N[C@H]([C@H](O)\C=C\CC\C=C(/C)CCCCCCCCC)CO[C@@H]1O[C@H](CO)[C@@H](O)[C@H](O)[C@H]1O JSPNNZKWADNWHI-PNANGNLXSA-N 0.000 claims 1
- 102000003951 Erythropoietin Human genes 0.000 claims 1
- 108090000394 Erythropoietin Proteins 0.000 claims 1
- RIZIAUKTHDLMQX-UHFFFAOYSA-N cerebroside D Natural products CCCCCCCCCCCCCCCCC(O)C(=O)NC(C(O)C=CCCC=C(C)CCCCCCCCC)COC1OC(CO)C(O)C(O)C1O RIZIAUKTHDLMQX-UHFFFAOYSA-N 0.000 claims 1
- 229940105423 erythropoietin Drugs 0.000 claims 1
- 208000027866 inflammatory disease Diseases 0.000 claims 1
- 125000002347 octyl group Chemical group [H]C([*])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])C([H])([H])[H] 0.000 claims 1
- OXCMYAYHXIHQOA-UHFFFAOYSA-N potassium;[2-butyl-5-chloro-3-[[4-[2-(1,2,4-triaza-3-azanidacyclopenta-1,4-dien-5-yl)phenyl]phenyl]methyl]imidazol-4-yl]methanol Chemical compound [K+].CCCCC1=NC(Cl)=C(CO)N1CC1=CC=C(C=2C(=CC=CC=2)C2=N[N-]N=N2)C=C1 OXCMYAYHXIHQOA-UHFFFAOYSA-N 0.000 claims 1
- 238000000034 method Methods 0.000 description 16
- 108020004414 DNA Proteins 0.000 description 9
- 125000006850 spacer group Chemical group 0.000 description 5
- FAPWRFPIFSIZLT-UHFFFAOYSA-M Sodium chloride Chemical compound [Na+].[Cl-] FAPWRFPIFSIZLT-UHFFFAOYSA-M 0.000 description 4
- 241000702421 Dependoparvovirus Species 0.000 description 3
- BJEPYKJPYRNKOW-REOHCLBHSA-N (S)-malic acid Chemical compound OC(=O)[C@@H](O)CC(O)=O BJEPYKJPYRNKOW-REOHCLBHSA-N 0.000 description 2
- 102000003638 Glucose-6-Phosphatase Human genes 0.000 description 2
- 108010086800 Glucose-6-Phosphatase Proteins 0.000 description 2
- TWRXJAOTZQYOKJ-UHFFFAOYSA-L Magnesium chloride Chemical compound [Mg+2].[Cl-].[Cl-] TWRXJAOTZQYOKJ-UHFFFAOYSA-L 0.000 description 2
- BJEPYKJPYRNKOW-UHFFFAOYSA-N alpha-hydroxysuccinic acid Natural products OC(=O)C(O)CC(O)=O BJEPYKJPYRNKOW-UHFFFAOYSA-N 0.000 description 2
- 235000011090 malic acid Nutrition 0.000 description 2
- 239000001630 malic acid Substances 0.000 description 2
- 239000011780 sodium chloride Substances 0.000 description 2
- GVNVAWHJIKLAGL-UHFFFAOYSA-N 2-(cyclohexen-1-yl)cyclohexan-1-one Chemical compound O=C1CCCCC1C1=CCCCC1 GVNVAWHJIKLAGL-UHFFFAOYSA-N 0.000 description 1
- 241001655883 Adeno-associated virus - 1 Species 0.000 description 1
- 241000702423 Adeno-associated virus - 2 Species 0.000 description 1
- 241000202702 Adeno-associated virus - 3 Species 0.000 description 1
- 241000580270 Adeno-associated virus - 4 Species 0.000 description 1
- 241001634120 Adeno-associated virus - 5 Species 0.000 description 1
- 241000972680 Adeno-associated virus - 6 Species 0.000 description 1
- 241001164823 Adeno-associated virus - 7 Species 0.000 description 1
- 241001164825 Adeno-associated virus - 8 Species 0.000 description 1
- 241000649045 Adeno-associated virus 10 Species 0.000 description 1
- 241000649046 Adeno-associated virus 11 Species 0.000 description 1
- 241000649047 Adeno-associated virus 12 Species 0.000 description 1
- 241000272814 Anser sp. Species 0.000 description 1
- 241000256135 Chironomus thummi Species 0.000 description 1
- 101150065749 Churc1 gene Proteins 0.000 description 1
- 102000053602 DNA Human genes 0.000 description 1
- 206010016077 Factor IX deficiency Diseases 0.000 description 1
- 208000020322 Gaucher disease type I Diseases 0.000 description 1
- 208000020916 Gaucher disease type II Diseases 0.000 description 1
- 208000028735 Gaucher disease type III Diseases 0.000 description 1
- 235000010469 Glycine max Nutrition 0.000 description 1
- 244000068988 Glycine max Species 0.000 description 1
- 208000032003 Glycogen storage disease due to glucose-6-phosphatase deficiency Diseases 0.000 description 1
- 206010018464 Glycogen storage disease type I Diseases 0.000 description 1
- 101000955481 Homo sapiens Phosphatidylcholine translocator ABCB4 Proteins 0.000 description 1
- 241000702617 Human parvovirus B19 Species 0.000 description 1
- 208000025923 Mucopolysaccharidosis type 4B Diseases 0.000 description 1
- 102100039032 Phosphatidylcholine translocator ABCB4 Human genes 0.000 description 1
- 102100038239 Protein Churchill Human genes 0.000 description 1
- 241000125945 Protoparvovirus Species 0.000 description 1
- 241000700605 Viruses Species 0.000 description 1
- 150000001784 cerebrosides Chemical class 0.000 description 1
- 239000003153 chemical reaction reagent Substances 0.000 description 1
- UREBDLICKHMUKA-CXSFZGCWSA-N dexamethasone Chemical compound C1CC2=CC(=O)C=C[C@]2(C)[C@]2(F)[C@@H]1[C@@H]1C[C@@H](C)[C@@](C(=O)CO)(O)[C@@]1(C)C[C@@H]2O UREBDLICKHMUKA-CXSFZGCWSA-N 0.000 description 1
- 229950000812 dexamethasone palmitate Drugs 0.000 description 1
- ZBCBWPMODOFKDW-UHFFFAOYSA-N diethanolamine Chemical compound OCCNCCO ZBCBWPMODOFKDW-UHFFFAOYSA-N 0.000 description 1
- 201000010099 disease Diseases 0.000 description 1
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 description 1
- 201000004541 glycogen storage disease I Diseases 0.000 description 1
- 229910001629 magnesium chloride Inorganic materials 0.000 description 1
- 208000010978 mucopolysaccharidosis type 4 Diseases 0.000 description 1
- 230000008488 polyadenylation Effects 0.000 description 1
- 108090000623 proteins and genes Proteins 0.000 description 1
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063026479P | 2020-05-18 | 2020-05-18 | |
| US63/026,479 | 2020-05-18 | ||
| PCT/US2021/032676 WO2021236479A2 (fr) | 2020-05-18 | 2021-05-17 | Nouveaux lipides et compositions de nanoparticules de ceux-ci |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2023527747A JP2023527747A (ja) | 2023-06-30 |
| JPWO2021236479A5 true JPWO2021236479A5 (fr) | 2024-05-24 |
Family
ID=78707483
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2022570302A Pending JP2023527747A (ja) | 2020-05-18 | 2021-05-17 | 新規脂質及びそれらのナノ粒子組成物 |
Country Status (9)
| Country | Link |
|---|---|
| US (1) | US20230181764A1 (fr) |
| EP (1) | EP4153144A4 (fr) |
| JP (1) | JP2023527747A (fr) |
| CN (1) | CN115968280A (fr) |
| AU (1) | AU2021274481A1 (fr) |
| CA (1) | CA3175957A1 (fr) |
| IL (1) | IL298241A (fr) |
| MX (1) | MX2022014484A (fr) |
| WO (1) | WO2021236479A2 (fr) |
Families Citing this family (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2020077007A1 (fr) | 2018-10-09 | 2020-04-16 | The University Of British Columbia | Compositions et systèmes comprenant des vésicules compétentes pour la transfection exemptes de solvants organiques et de détergents, et procédés associés |
| CN114716355B (zh) * | 2022-04-02 | 2023-09-05 | 华南理工大学 | 一种脂质化合物、包含其的组合物及应用 |
| CN117925729A (zh) * | 2024-01-19 | 2024-04-26 | 晟迪生物医药(苏州)有限公司 | 基于空白脂质纳米颗粒的转染试剂、其制备方法和应用 |
Family Cites Families (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| GB0610479D0 (en) * | 2006-05-26 | 2006-07-05 | Ge Healthcare Bio Sciences Ab | A method for generating metal chelating affinity ligands |
| CN103930398B (zh) * | 2011-11-18 | 2016-08-24 | 日油株式会社 | 具有改进的细胞内动力学的阳离子脂质 |
| JP6640750B2 (ja) * | 2015-01-30 | 2020-02-05 | 日油株式会社 | カチオン性脂質 |
| WO2017061150A1 (fr) * | 2015-10-08 | 2017-04-13 | 日油株式会社 | Émulsion de type h/e |
| EP3778572A4 (fr) * | 2018-03-27 | 2021-12-22 | NOF Corporation | Nouveau lipide cationique présentant une dynamique intracellulaire améliorée |
| CA3150452A1 (fr) * | 2019-09-06 | 2021-03-11 | Generation Bio Co. | Compositions de nanoparticules lipidiques comprenant de l'adn a extremites fermees et des lipides clivables et leurs procedes d'utilisation |
| US20220354793A1 (en) * | 2019-09-26 | 2022-11-10 | Nof Corporation | Lipid nanoparticle lyophilized composition |
| BR112022007481A2 (pt) * | 2019-11-22 | 2022-07-12 | Generation Bio Co | Lipídios ionizáveis e composições de nanopartículas dos mesmos |
| CN114901360A (zh) * | 2019-12-20 | 2022-08-12 | 库瑞瓦格股份公司 | 用于递送核酸的新型脂质纳米颗粒 |
-
2021
- 2021-05-17 CA CA3175957A patent/CA3175957A1/fr active Pending
- 2021-05-17 JP JP2022570302A patent/JP2023527747A/ja active Pending
- 2021-05-17 MX MX2022014484A patent/MX2022014484A/es unknown
- 2021-05-17 IL IL298241A patent/IL298241A/en unknown
- 2021-05-17 WO PCT/US2021/032676 patent/WO2021236479A2/fr unknown
- 2021-05-17 EP EP21808224.6A patent/EP4153144A4/fr active Pending
- 2021-05-17 CN CN202180035954.4A patent/CN115968280A/zh active Pending
- 2021-05-17 AU AU2021274481A patent/AU2021274481A1/en active Pending
- 2021-05-17 US US17/925,404 patent/US20230181764A1/en active Pending
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JPWO2021102411A5 (fr) | ||
| KR20230052895A (ko) | 폴리뉴클레오타이드를 감소된 크기의 지질 나노입자로 캡슐화하는 방법 및 신규한 지질 나노입자 제형 | |
| US20240294474A1 (en) | Novel lipids and nanoparticle compositions thereof | |
| WO2021102411A1 (fr) | Lipides ionisables et compositions de nanoparticules associées | |
| US20220280427A1 (en) | Lipid nanoparticle compositions comprising closed-ended dna and cleavable lipids and methods of use thereof | |
| JPWO2022016089A5 (fr) | ||
| US20240293574A1 (en) | Cationic lipids and compositions thereof | |
| US20240226325A1 (en) | Cationic lipids and compositions thereof | |
| US20230181764A1 (en) | Novel lipids and nanoparticle compositions thereof | |
| JPWO2021195529A5 (fr) | ||
| JPWO2021046265A5 (fr) | ||
| JPWO2021236479A5 (fr) | ||
| RU2023129760A (ru) | Катионные липиды и их композиции | |
| RU2023135629A (ru) | Катионные липиды и их композиции |