JPWO2020146344A5 - - Google Patents
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- JPWO2020146344A5 JPWO2020146344A5 JP2021539435A JP2021539435A JPWO2020146344A5 JP WO2020146344 A5 JPWO2020146344 A5 JP WO2020146344A5 JP 2021539435 A JP2021539435 A JP 2021539435A JP 2021539435 A JP2021539435 A JP 2021539435A JP WO2020146344 A5 JPWO2020146344 A5 JP WO2020146344A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- mrna
- seq
- glycero
- dnah5
- Prior art date
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- 239000000203 mixture Substances 0.000 claims description 90
- 108020004999 Messenger RNA Proteins 0.000 claims description 60
- 229920002106 messenger RNA Polymers 0.000 claims description 60
- 101710031971 DNAH5 Proteins 0.000 claims description 25
- 102100017497 DNAH5 Human genes 0.000 claims description 25
- 229920001320 Leader sequence (mRNA) Polymers 0.000 claims description 22
- 239000002502 liposome Substances 0.000 claims description 20
- 229920001405 Coding region Polymers 0.000 claims description 18
- 125000002091 cationic group Chemical group 0.000 claims description 16
- -1 DLenDMA Chemical compound 0.000 claims description 14
- 150000002632 lipids Chemical class 0.000 claims description 13
- 108020003589 5' Untranslated Regions Proteins 0.000 claims description 11
- ZDTFMPXQUSBYRL-UUOKFMHZSA-N 2-Aminoadenosine Chemical compound C12=NC(N)=NC(N)=C2N=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O ZDTFMPXQUSBYRL-UUOKFMHZSA-N 0.000 claims description 10
- KILNVBDSWZSGLL-KXQOOQHDSA-O 1,2-di-O-palmitoyl-sn-glycero-3-phosphocholine Chemical compound CCCCCCCCCCCCCCCC(=O)OC[C@H](COP(O)(=O)OCC[N+](C)(C)C)OC(=O)CCCCCCCCCCCCCCC KILNVBDSWZSGLL-KXQOOQHDSA-O 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
- WALUVDCNGPQPOD-UHFFFAOYSA-M 2,3-di(tetradecoxy)propyl-(2-hydroxyethyl)-dimethylazanium;bromide Chemical compound [Br-].CCCCCCCCCCCCCCOCC(C[N+](C)(C)CCO)OCCCCCCCCCCCCCC WALUVDCNGPQPOD-UHFFFAOYSA-M 0.000 claims description 8
- ZAYHVCMSTBRABG-JXOAFFINSA-N 5-Methylcytidine Chemical compound O=C1N=C(N)C(C)=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 ZAYHVCMSTBRABG-JXOAFFINSA-N 0.000 claims description 8
- REZZEXDLIUJMMS-UHFFFAOYSA-M Dimethyldioctadecylammonium chloride Chemical compound [Cl-].CCCCCCCCCCCCCCCCCC[N+](C)(C)CCCCCCCCCCCCCCCCCC REZZEXDLIUJMMS-UHFFFAOYSA-M 0.000 claims description 8
- RAXXELZNTBOGNW-UHFFFAOYSA-N Imidazole Chemical compound C1=CNC=N1 RAXXELZNTBOGNW-UHFFFAOYSA-N 0.000 claims description 8
- 229920000401 Three prime untranslated region Polymers 0.000 claims description 8
- 125000003729 nucleotide group Chemical group 0.000 claims description 8
- 108020005345 3' Untranslated Regions Proteins 0.000 claims description 6
- 210000004072 Lung Anatomy 0.000 claims description 6
- 230000001809 detectable Effects 0.000 claims description 6
- 210000000979 Axoneme Anatomy 0.000 claims description 5
- NEZDNQCXEZDCBI-WJOKGBTCSA-N (2-aminoethoxy)[(2R)-2,3-bis(tetradecanoyloxy)propoxy]phosphinic acid Chemical compound CCCCCCCCCCCCCC(=O)OC[C@H](COP(O)(=O)OCCN)OC(=O)CCCCCCCCCCCCC NEZDNQCXEZDCBI-WJOKGBTCSA-N 0.000 claims description 4
- RIFDKYBNWNPCQK-IOSLPCCCSA-N (2R,3S,4R,5R)-2-(hydroxymethyl)-5-(6-imino-3-methylpurin-9-yl)oxolane-3,4-diol Chemical compound C1=2N(C)C=NC(=N)C=2N=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O RIFDKYBNWNPCQK-IOSLPCCCSA-N 0.000 claims description 4
- ZKWQSBFSGZJNFP-UHFFFAOYSA-N 1,2-Bis(dimethylphosphino)ethane Chemical compound CP(C)CCP(C)C ZKWQSBFSGZJNFP-UHFFFAOYSA-N 0.000 claims description 4
- QFMZQPDHXULLKC-UHFFFAOYSA-N 1,2-Bis(diphenylphosphino)ethane Chemical compound C=1C=CC=CC=1P(C=1C=CC=CC=1)CCP(C=1C=CC=CC=1)C1=CC=CC=C1 QFMZQPDHXULLKC-UHFFFAOYSA-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
- 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 4
- XIWUJDSJKCVNRX-UGKPPGOTSA-N 1-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)-2-prop-1-ynyloxolan-2-yl]pyrimidine-2,4-dione Chemical compound C1=CC(=O)NC(=O)N1[C@]1(C#CC)O[C@H](CO)[C@@H](O)[C@H]1O XIWUJDSJKCVNRX-UGKPPGOTSA-N 0.000 claims description 4
- RKSLVDIXBGWPIS-UAKXSSHOSA-N 1-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-iodopyrimidine-2,4-dione Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(I)=C1 RKSLVDIXBGWPIS-UAKXSSHOSA-N 0.000 claims description 4
- QLOCVMVCRJOTTM-TURQNECASA-N 1-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-prop-1-ynylpyrimidine-2,4-dione Chemical compound O=C1NC(=O)C(C#CC)=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 QLOCVMVCRJOTTM-TURQNECASA-N 0.000 claims description 4
- PISWNSOQFZRVJK-XLPZGREQSA-N 1-[(2R,4S,5R)-4-hydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-methyl-2-sulfanylidenepyrimidin-4-one Chemical compound S=C1NC(=O)C(C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)C1 PISWNSOQFZRVJK-XLPZGREQSA-N 0.000 claims description 4
- JRYMOPZHXMVHTA-DAGMQNCNSA-N 2-amino-7-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-1H-pyrrolo[2,3-d]pyrimidin-4-one Chemical compound C1=CC=2C(=O)NC(N)=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O JRYMOPZHXMVHTA-DAGMQNCNSA-N 0.000 claims description 4
- SHCCKWGIFIPGNJ-NSUCVBPYSA-N 2-aminoethyl [(2R)-2,3-bis[(Z)-octadec-9-enoxy]propyl] hydrogen phosphate Chemical compound CCCCCCCC\C=C/CCCCCCCCOC[C@H](COP(O)(=O)OCCN)OCCCCCCCC\C=C/CCCCCCCC SHCCKWGIFIPGNJ-NSUCVBPYSA-N 0.000 claims description 4
- RHFUOMFWUGWKKO-XVFCMESISA-N 2-thiocytidine Chemical compound S=C1N=C(N)C=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 RHFUOMFWUGWKKO-XVFCMESISA-N 0.000 claims description 4
- LMMLLWZHCKCFQA-UGKPPGOTSA-N 4-amino-1-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)-2-prop-1-ynyloxolan-2-yl]pyrimidin-2-one Chemical compound C1=CC(N)=NC(=O)N1[C@]1(C#CC)O[C@H](CO)[C@@H](O)[C@H]1O LMMLLWZHCKCFQA-UGKPPGOTSA-N 0.000 claims description 4
- XXSIICQLPUAUDF-TURQNECASA-N 4-amino-1-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-prop-1-ynylpyrimidin-2-one Chemical compound O=C1N=C(N)C(C#CC)=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 XXSIICQLPUAUDF-TURQNECASA-N 0.000 claims description 4
- AGFIRQJZCNVMCW-UAKXSSHOSA-N 5-Bromouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(Br)=C1 AGFIRQJZCNVMCW-UAKXSSHOSA-N 0.000 claims description 4
- FHIDNBAQOFJWCA-UAKXSSHOSA-N 5-fluorouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C(F)=C1 FHIDNBAQOFJWCA-UAKXSSHOSA-N 0.000 claims description 4
- BXJHWYVXLGLDMZ-UHFFFAOYSA-N 6-O-Methylguanine Chemical compound COC1=NC(N)=NC2=C1NC=N2 BXJHWYVXLGLDMZ-UHFFFAOYSA-N 0.000 claims description 4
- UEHOMUNTZPIBIL-UUOKFMHZSA-N 6-amino-9-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-7H-purin-8-one Chemical compound O=C1NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O UEHOMUNTZPIBIL-UUOKFMHZSA-N 0.000 claims description 4
- HCAJQHYUCKICQH-VPENINKCSA-N 8-Oxo-7,8-dihydro-2'-deoxyguanosine Chemical compound C1=2NC(N)=NC(=O)C=2NC(=O)N1[C@H]1C[C@H](O)[C@@H](CO)O1 HCAJQHYUCKICQH-VPENINKCSA-N 0.000 claims description 4
- OHBIZLMAUVTUPW-UHFFFAOYSA-N CCCCCCCCCCC(O)CN(CCCCC1OC(=O)C(CCCCN(CC(O)CCCCCCCCCC)CC(O)CCCCCCCCCC)OC1=O)CC(O)CCCCCCCCC Chemical compound CCCCCCCCCCC(O)CN(CCCCC1OC(=O)C(CCCCN(CC(O)CCCCCCCCCC)CC(O)CCCCCCCCCC)OC1=O)CC(O)CCCCCCCCC OHBIZLMAUVTUPW-UHFFFAOYSA-N 0.000 claims description 4
- 241000282472 Canis lupus familiaris Species 0.000 claims description 4
- XULFJDKZVHTRLG-JDVCJPALSA-N DOSPA trifluoroacetate Chemical compound [O-]C(=O)C(F)(F)F.CCCCCCCC\C=C/CCCCCCCCOCC(C[N+](C)(C)CCNC(=O)C(CCCNCCCN)NCCCN)OCCCCCCCC\C=C/CCCCCCCC XULFJDKZVHTRLG-JDVCJPALSA-N 0.000 claims description 4
- UGQMRVRMYYASKQ-KMPDEGCQSA-N Inosine Natural products O[C@H]1[C@H](O)[C@@H](CO)O[C@@H]1N1C(N=CNC2=O)=C2N=C1 UGQMRVRMYYASKQ-KMPDEGCQSA-N 0.000 claims description 4
- UGQMRVRMYYASKQ-KQYNXXCUSA-N Inosine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(O)=C2N=C1 UGQMRVRMYYASKQ-KQYNXXCUSA-N 0.000 claims description 4
- KCTZOTUQSGYWLV-UHFFFAOYSA-N N1C=NC=C2N=CC=C21 Chemical compound N1C=NC=C2N=CC=C21 KCTZOTUQSGYWLV-UHFFFAOYSA-N 0.000 claims description 4
- PTJWIQPHWPFNBW-GBNDHIKLSA-N Pseudouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1C1=CNC(=O)NC1=O PTJWIQPHWPFNBW-GBNDHIKLSA-N 0.000 claims description 4
- HDZZVAMISRMYHH-LITAXDCLSA-N Tubercidin Chemical compound C1=CC=2C(N)=NC=NC=2N1[C@@H]1O[C@@H](CO)[C@H](O)[C@H]1O HDZZVAMISRMYHH-LITAXDCLSA-N 0.000 claims description 4
- 125000000217 alkyl group Chemical group 0.000 claims description 4
- UMGXUWVIJIQANV-UHFFFAOYSA-M didecyl(dimethyl)azanium;bromide Chemical compound [Br-].CCCCCCCCCC[N+](C)(C)CCCCCCCCCC UMGXUWVIJIQANV-UHFFFAOYSA-M 0.000 claims description 4
- DSNRWDQKZIEDDB-CLFAGFIQSA-N dioleoyl phosphatidylglycerol Chemical compound CCCCCCCC\C=C/CCCCCCCC(=O)OCC(COP(O)(=O)OCC(O)CO)OC(=O)CCCCCCC\C=C/CCCCCCCC DSNRWDQKZIEDDB-CLFAGFIQSA-N 0.000 claims description 4
- 230000004064 dysfunction Effects 0.000 claims description 4
- 125000003827 glycol group Chemical group 0.000 claims description 4
- 229960003786 inosine Drugs 0.000 claims description 4
- 229920001223 polyethylene glycol Polymers 0.000 claims description 4
- 102000004169 proteins and genes Human genes 0.000 claims description 3
- 108090000623 proteins and genes Proteins 0.000 claims description 3
- RKDVKSZUMVYZHH-UHFFFAOYSA-N 1,4-dioxane-2,5-dione Chemical compound O=C1COC(=O)CO1 RKDVKSZUMVYZHH-UHFFFAOYSA-N 0.000 claims description 2
- OIRDTQYFTABQOQ-GAWUUDPSSA-N 9-β-D-XYLOFURANOSYL-ADENINE Chemical compound C1=NC=2C(N)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@H](O)[C@H]1O OIRDTQYFTABQOQ-GAWUUDPSSA-N 0.000 claims description 2
- OIRDTQYFTABQOQ-SXVXDFOESA-N Adenosine Natural products Nc1ncnc2c1ncn2[C@@H]3O[C@@H](CO)[C@H](O)[C@@H]3O OIRDTQYFTABQOQ-SXVXDFOESA-N 0.000 claims description 2
- 210000004556 Brain Anatomy 0.000 claims description 2
- 239000002126 C01EB10 - Adenosine Substances 0.000 claims description 2
- 108020004705 Codon Proteins 0.000 claims description 2
- 210000002216 Heart Anatomy 0.000 claims description 2
- 210000000936 Intestines Anatomy 0.000 claims description 2
- 210000003734 Kidney Anatomy 0.000 claims description 2
- 210000004185 Liver Anatomy 0.000 claims description 2
- 210000001672 Ovary Anatomy 0.000 claims description 2
- 210000000952 Spleen Anatomy 0.000 claims description 2
- 210000002784 Stomach Anatomy 0.000 claims description 2
- 210000001550 Testis Anatomy 0.000 claims description 2
- 229960005305 adenosine Drugs 0.000 claims description 2
- 230000001886 ciliary Effects 0.000 claims description 2
- 230000003111 delayed Effects 0.000 claims description 2
- 238000007918 intramuscular administration Methods 0.000 claims description 2
- 238000001990 intravenous administration Methods 0.000 claims description 2
- 239000002773 nucleotide Substances 0.000 claims description 2
- 210000000056 organs Anatomy 0.000 claims description 2
- 239000008194 pharmaceutical composition Substances 0.000 claims description 2
- 238000007920 subcutaneous administration Methods 0.000 claims description 2
- ANGOYQCNNLBDLX-UHFFFAOYSA-N CCCCCCCCCCC(O)CN(CCCC(C)C1OC(=O)C(OC1=O)C(C)CCCN(CC(O)CCCCCCCCCC)CC(O)CCCCCCCCCC)CC(O)CCCCCCCCC Chemical compound CCCCCCCCCCC(O)CN(CCCC(C)C1OC(=O)C(OC1=O)C(C)CCCN(CC(O)CCCCCCCCCC)CC(O)CCCCCCCCCC)CC(O)CCCCCCCCC ANGOYQCNNLBDLX-UHFFFAOYSA-N 0.000 claims 1
- 101000346496 DNAH5 Proteins 0.000 description 1
- 102000035639 human DNAH5 protein Human genes 0.000 description 1
- 230000004048 modification Effects 0.000 description 1
- 238000006011 modification reaction Methods 0.000 description 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2024010105A JP2024028651A (ja) | 2019-01-07 | 2024-01-26 | 原発性線毛機能不全症の治療のための組成物および方法 |
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962789414P | 2019-01-07 | 2019-01-07 | |
| US62/789,414 | 2019-01-07 | ||
| PCT/US2020/012529 WO2020146344A1 (en) | 2019-01-07 | 2020-01-07 | Composition and methods for treatment of primary ciliary dyskinesia |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024010105A Division JP2024028651A (ja) | 2019-01-07 | 2024-01-26 | 原発性線毛機能不全症の治療のための組成物および方法 |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022516356A JP2022516356A (ja) | 2022-02-25 |
| JPWO2020146344A5 true JPWO2020146344A5 (de) | 2022-12-05 |
Family
ID=69423424
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021539435A Pending JP2022516356A (ja) | 2019-01-07 | 2020-01-07 | 原発性線毛機能不全症の治療のための組成物および方法 |
| JP2024010105A Pending JP2024028651A (ja) | 2019-01-07 | 2024-01-26 | 原発性線毛機能不全症の治療のための組成物および方法 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2024010105A Pending JP2024028651A (ja) | 2019-01-07 | 2024-01-26 | 原発性線毛機能不全症の治療のための組成物および方法 |
Country Status (8)
| Country | Link |
|---|---|
| US (3) | US20220087935A1 (de) |
| EP (1) | EP3908597A1 (de) |
| JP (2) | JP2022516356A (de) |
| CN (1) | CN113412273A (de) |
| AU (1) | AU2020206109A1 (de) |
| CA (1) | CA3125588A1 (de) |
| IL (1) | IL284480A (de) |
| WO (1) | WO2020146344A1 (de) |
Families Citing this family (4)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| DK3463483T3 (da) * | 2016-05-27 | 2024-03-04 | Transcriptx Inc | Behandling af primær ciliedyskinesi med syntetisk messenger-RNA |
| EP3908597A1 (de) * | 2019-01-07 | 2021-11-17 | Translate Bio, Inc. | Zusammensetzung und verfahren zur behandlung der primären ciliären dyskinesie |
| EP4308703A1 (de) * | 2021-03-19 | 2024-01-24 | Recode Therapeutics, Inc. | Polynukleotidzusammensetzungen, zugehörige formulierungen und verfahren zur verwendung davon |
| WO2023086893A1 (en) * | 2021-11-10 | 2023-05-19 | Translate Bio, Inc. | Composition and methods for treatment of primary ciliary dyskinesia |
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2020
- 2020-01-07 EP EP20703340.8A patent/EP3908597A1/de active Pending
- 2020-01-07 CN CN202080012787.7A patent/CN113412273A/zh active Pending
- 2020-01-07 US US17/420,346 patent/US20220087935A1/en not_active Abandoned
- 2020-01-07 US US16/736,417 patent/US11559561B2/en active Active
- 2020-01-07 AU AU2020206109A patent/AU2020206109A1/en active Pending
- 2020-01-07 WO PCT/US2020/012529 patent/WO2020146344A1/en unknown
- 2020-01-07 JP JP2021539435A patent/JP2022516356A/ja active Pending
- 2020-01-07 CA CA3125588A patent/CA3125588A1/en active Pending
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2021
- 2021-06-29 IL IL284480A patent/IL284480A/en unknown
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2022
- 2022-12-06 US US18/062,435 patent/US20230226147A1/en active Pending
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2024
- 2024-01-26 JP JP2024010105A patent/JP2024028651A/ja active Pending
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