JP2019147811A5 - - Google Patents
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- JP2019147811A5 JP2019147811A5 JP2019078998A JP2019078998A JP2019147811A5 JP 2019147811 A5 JP2019147811 A5 JP 2019147811A5 JP 2019078998 A JP2019078998 A JP 2019078998A JP 2019078998 A JP2019078998 A JP 2019078998A JP 2019147811 A5 JP2019147811 A5 JP 2019147811A5
- Authority
- JP
- Japan
- Prior art keywords
- rna molecule
- modified rna
- synthetic modified
- use according
- synthetic
- 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
- 108091032973 (ribonucleotides)n+m Proteins 0.000 claims 28
- 102000009524 Vascular Endothelial Growth Factor A Human genes 0.000 claims 6
- 108010073929 Vascular Endothelial Growth Factor A Proteins 0.000 claims 6
- 230000004064 dysfunction Effects 0.000 claims 5
- 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 4
- 210000004351 coronary vessel Anatomy 0.000 claims 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 claims 3
- 239000000203 mixture Substances 0.000 claims 3
- SXUXMRMBWZCMEN-UHFFFAOYSA-N 2'-O-methyl uridine Natural products COC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 SXUXMRMBWZCMEN-UHFFFAOYSA-N 0.000 claims 2
- GJTBSTBJLVYKAU-XVFCMESISA-N 2-thiouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=S)NC(=O)C=C1 GJTBSTBJLVYKAU-XVFCMESISA-N 0.000 claims 2
- ZAYHVCMSTBRABG-UHFFFAOYSA-N 5-Methylcytidine Natural products O=C1N=C(N)C(C)=CN1C1C(O)C(O)C(CO)O1 ZAYHVCMSTBRABG-UHFFFAOYSA-N 0.000 claims 2
- 208000031229 Cardiomyopathies Diseases 0.000 claims 2
- 208000002330 Congenital Heart Defects Diseases 0.000 claims 2
- 206010020772 Hypertension Diseases 0.000 claims 2
- 108091026898 Leader sequence (mRNA) Proteins 0.000 claims 2
- 108091036407 Polyadenylation Proteins 0.000 claims 2
- 229930185560 Pseudouridine Natural products 0.000 claims 2
- PTJWIQPHWPFNBW-UHFFFAOYSA-N Pseudouridine C Natural products OC1C(O)C(CO)OC1C1=CNC(=O)NC1=O PTJWIQPHWPFNBW-UHFFFAOYSA-N 0.000 claims 2
- 108091036066 Three prime untranslated region Proteins 0.000 claims 2
- DRTQHJPVMGBUCF-XVFCMESISA-N Uridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-XVFCMESISA-N 0.000 claims 2
- 206010003119 arrhythmia Diseases 0.000 claims 2
- WGDUUQDYDIIBKT-UHFFFAOYSA-N beta-Pseudouridine Natural products OC1OC(CN2C=CC(=O)NC2=O)C(O)C1O WGDUUQDYDIIBKT-UHFFFAOYSA-N 0.000 claims 2
- 230000005540 biological transmission Effects 0.000 claims 2
- 208000028831 congenital heart disease Diseases 0.000 claims 2
- FPUGCISOLXNPPC-IOSLPCCCSA-N cordysinin B Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C2=NC=NC(N)=C2N=C1 FPUGCISOLXNPPC-IOSLPCCCSA-N 0.000 claims 2
- 208000035475 disorder Diseases 0.000 claims 2
- 208000019622 heart disease Diseases 0.000 claims 2
- 208000031225 myocardial ischemia Diseases 0.000 claims 2
- 125000003835 nucleoside group Chemical group 0.000 claims 2
- 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 2
- GRYSXUXXBDSYRT-WOUKDFQISA-N (2r,3r,4r,5r)-2-(hydroxymethyl)-4-methoxy-5-[6-(methylamino)purin-9-yl]oxolan-3-ol Chemical compound C1=NC=2C(NC)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1OC GRYSXUXXBDSYRT-WOUKDFQISA-N 0.000 claims 1
- OTFGHFBGGZEXEU-PEBGCTIMSA-N 1-[(2r,3r,4r,5r)-4-hydroxy-5-(hydroxymethyl)-3-methoxyoxolan-2-yl]-3-methylpyrimidine-2,4-dione Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)N(C)C(=O)C=C1 OTFGHFBGGZEXEU-PEBGCTIMSA-N 0.000 claims 1
- QPHRQMAYYMYWFW-FJGDRVTGSA-N 1-[(2r,3s,4r,5r)-3-fluoro-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]pyrimidine-2,4-dione Chemical compound O[C@]1(F)[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 QPHRQMAYYMYWFW-FJGDRVTGSA-N 0.000 claims 1
- FPUGCISOLXNPPC-UHFFFAOYSA-N 2'-O-Methyladenosine Natural products COC1C(O)C(CO)OC1N1C2=NC=NC(N)=C2N=C1 FPUGCISOLXNPPC-UHFFFAOYSA-N 0.000 claims 1
- RFCQJGFZUQFYRF-UHFFFAOYSA-N 2'-O-Methylcytidine Natural products COC1C(O)C(CO)OC1N1C(=O)N=C(N)C=C1 RFCQJGFZUQFYRF-UHFFFAOYSA-N 0.000 claims 1
- OVYNGSFVYRPRCG-UHFFFAOYSA-N 2'-O-Methylguanosine Natural products COC1C(O)C(CO)OC1N1C(NC(N)=NC2=O)=C2N=C1 OVYNGSFVYRPRCG-UHFFFAOYSA-N 0.000 claims 1
- RFCQJGFZUQFYRF-ZOQUXTDFSA-N 2'-O-methylcytidine Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)N=C(N)C=C1 RFCQJGFZUQFYRF-ZOQUXTDFSA-N 0.000 claims 1
- OVYNGSFVYRPRCG-KQYNXXCUSA-N 2'-O-methylguanosine Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(N=C(N)NC2=O)=C2N=C1 OVYNGSFVYRPRCG-KQYNXXCUSA-N 0.000 claims 1
- SXUXMRMBWZCMEN-ZOQUXTDFSA-N 2'-O-methyluridine Chemical compound CO[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=O)C=C1 SXUXMRMBWZCMEN-ZOQUXTDFSA-N 0.000 claims 1
- ZLOIGESWDJYCTF-UHFFFAOYSA-N 4-Thiouridine Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=S)C=C1 ZLOIGESWDJYCTF-UHFFFAOYSA-N 0.000 claims 1
- ZLOIGESWDJYCTF-XVFCMESISA-N 4-thiouridine Chemical compound O[C@@H]1[C@H](O)[C@@H](CO)O[C@H]1N1C(=O)NC(=S)C=C1 ZLOIGESWDJYCTF-XVFCMESISA-N 0.000 claims 1
- OGHAROSJZRTIOK-KQYNXXCUSA-O 7-methylguanosine Chemical compound C1=2N=C(N)NC(=O)C=2[N+](C)=CN1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O OGHAROSJZRTIOK-KQYNXXCUSA-O 0.000 claims 1
- 201000001320 Atherosclerosis Diseases 0.000 claims 1
- DWRXFEITVBNRMK-UHFFFAOYSA-N Beta-D-1-Arabinofuranosylthymine Natural products O=C1NC(=O)C(C)=CN1C1C(O)C(O)C(CO)O1 DWRXFEITVBNRMK-UHFFFAOYSA-N 0.000 claims 1
- 206010007559 Cardiac failure congestive Diseases 0.000 claims 1
- 206010069729 Collateral circulation Diseases 0.000 claims 1
- 206010019280 Heart failures Diseases 0.000 claims 1
- 101000808011 Homo sapiens Vascular endothelial growth factor A Proteins 0.000 claims 1
- 206010020751 Hypersensitivity Diseases 0.000 claims 1
- 206010052768 Infectious myocarditis Diseases 0.000 claims 1
- 229930010555 Inosine Natural products 0.000 claims 1
- 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 1
- 206010028289 Muscle atrophy Diseases 0.000 claims 1
- 208000021642 Muscular disease Diseases 0.000 claims 1
- 208000009525 Myocarditis Diseases 0.000 claims 1
- 201000009623 Myopathy Diseases 0.000 claims 1
- IYYIBFCJILKPCO-WOUKDFQISA-O N(2),N(2),N(7)-trimethylguanosine Chemical compound C1=2NC(N(C)C)=NC(=O)C=2N(C)C=[N+]1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O IYYIBFCJILKPCO-WOUKDFQISA-O 0.000 claims 1
- ZBYRSRLCXTUFLJ-IOSLPCCCSA-O N(2),N(7)-dimethylguanosine Chemical compound CNC=1NC(C=2[N+](=CN([C@H]3[C@H](O)[C@H](O)[C@@H](CO)O3)C=2N=1)C)=O ZBYRSRLCXTUFLJ-IOSLPCCCSA-O 0.000 claims 1
- VQAYFKKCNSOZKM-IOSLPCCCSA-N N(6)-methyladenosine Chemical compound C1=NC=2C(NC)=NC=NC=2N1[C@@H]1O[C@H](CO)[C@@H](O)[C@H]1O VQAYFKKCNSOZKM-IOSLPCCCSA-N 0.000 claims 1
- 230000005856 abnormality Effects 0.000 claims 1
- 208000026935 allergic disease Diseases 0.000 claims 1
- 230000006793 arrhythmia Effects 0.000 claims 1
- 230000001363 autoimmune Effects 0.000 claims 1
- DRTQHJPVMGBUCF-PSQAKQOGSA-N beta-L-uridine Natural products O[C@H]1[C@@H](O)[C@H](CO)O[C@@H]1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-PSQAKQOGSA-N 0.000 claims 1
- 230000017531 blood circulation Effects 0.000 claims 1
- 208000029078 coronary artery disease Diseases 0.000 claims 1
- 201000010099 disease Diseases 0.000 claims 1
- 239000003814 drug Substances 0.000 claims 1
- 229940079593 drug Drugs 0.000 claims 1
- 238000012377 drug delivery Methods 0.000 claims 1
- 206010014665 endocarditis Diseases 0.000 claims 1
- 102000058223 human VEGFA Human genes 0.000 claims 1
- 230000009610 hypersensitivity Effects 0.000 claims 1
- 208000022368 idiopathic cardiomyopathy Diseases 0.000 claims 1
- 238000002347 injection Methods 0.000 claims 1
- 239000007924 injection Substances 0.000 claims 1
- 229960003786 inosine Drugs 0.000 claims 1
- 208000028867 ischemia Diseases 0.000 claims 1
- 150000002632 lipids Chemical class 0.000 claims 1
- 210000003205 muscle Anatomy 0.000 claims 1
- 201000006938 muscular dystrophy Diseases 0.000 claims 1
- 208000010125 myocardial infarction Diseases 0.000 claims 1
- 230000002685 pulmonary effect Effects 0.000 claims 1
- 208000002815 pulmonary hypertension Diseases 0.000 claims 1
- DWRXFEITVBNRMK-JXOAFFINSA-N ribothymidine Chemical compound O=C1NC(=O)C(C)=CN1[C@H]1[C@H](O)[C@H](O)[C@@H](CO)O1 DWRXFEITVBNRMK-JXOAFFINSA-N 0.000 claims 1
- 210000001519 tissue Anatomy 0.000 claims 1
- 239000003053 toxin Substances 0.000 claims 1
- 231100000765 toxin Toxicity 0.000 claims 1
- 108700012359 toxins Proteins 0.000 claims 1
- 235000011178 triphosphate Nutrition 0.000 claims 1
- 239000001226 triphosphate Substances 0.000 claims 1
- UNXRWKVEANCORM-UHFFFAOYSA-N triphosphoric acid Chemical compound OP(O)(=O)OP(O)(=O)OP(O)(O)=O UNXRWKVEANCORM-UHFFFAOYSA-N 0.000 claims 1
- DRTQHJPVMGBUCF-UHFFFAOYSA-N uracil arabinoside Natural products OC1C(O)C(CO)OC1N1C(=O)NC(=O)C=C1 DRTQHJPVMGBUCF-UHFFFAOYSA-N 0.000 claims 1
- 229940045145 uridine Drugs 0.000 claims 1
- 208000019553 vascular disease Diseases 0.000 claims 1
Priority Applications (1)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| JP2021102103A JP7439019B2 (ja) | 2011-04-03 | 2021-06-21 | 修飾型ｒｎａ（ｍｏｄ－ｒｎａ）を使用する効率的なインビボタンパク質発現 |
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201161471166P | 2011-04-03 | 2011-04-03 | |
| US61/471,166 | 2011-04-03 | ||
| US201161471584P | 2011-04-04 | 2011-04-04 | |
| US61/471,584 | 2011-04-04 | ||
| JP2017012661A JP2017128570A (ja) | 2011-04-03 | 2017-01-27 | 修飾型ｒｎａ（ｍｏｄ−ｒｎａ）を使用する効率的なインビボタンパク質発現 |
Related Parent Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017012661A Division JP2017128570A (ja) | 2011-04-03 | 2017-01-27 | 修飾型ｒｎａ（ｍｏｄ−ｒｎａ）を使用する効率的なインビボタンパク質発現 |
Related Child Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021102103A Division JP7439019B2 (ja) | 2011-04-03 | 2021-06-21 | 修飾型ｒｎａ（ｍｏｄ－ｒｎａ）を使用する効率的なインビボタンパク質発現 |
Publications (4)
| Publication Number | Publication Date |
|---|---|
| JP2019147811A JP2019147811A (ja) | 2019-09-05 |
| JP2019147811A5 true JP2019147811A5 (enExample) | 2019-10-17 |
| JP6902570B2 JP6902570B2 (ja) | 2021-07-14 |
| JP6902570B6 JP6902570B6 (ja) | 2021-08-18 |
Family
ID=46969503
Family Applications (4)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014503668A Withdrawn JP2014511694A (ja) | 2011-04-03 | 2012-03-12 | 修飾型ｒｎａ（ｍｏｄ−ｒｎａ）を使用する効率的なインビボタンパク質発現 |
| JP2017012661A Withdrawn JP2017128570A (ja) | 2011-04-03 | 2017-01-27 | 修飾型ｒｎａ（ｍｏｄ−ｒｎａ）を使用する効率的なインビボタンパク質発現 |
| JP2019078998A Active JP6902570B6 (ja) | 2011-04-03 | 2019-04-18 | 修飾型ｒｎａ（ｍｏｄ−ｒｎａ）を使用する効率的なインビボタンパク質発現 |
| JP2021102103A Active JP7439019B2 (ja) | 2011-04-03 | 2021-06-21 | 修飾型ｒｎａ（ｍｏｄ－ｒｎａ）を使用する効率的なインビボタンパク質発現 |
Family Applications Before (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2014503668A Withdrawn JP2014511694A (ja) | 2011-04-03 | 2012-03-12 | 修飾型ｒｎａ（ｍｏｄ−ｒｎａ）を使用する効率的なインビボタンパク質発現 |
| JP2017012661A Withdrawn JP2017128570A (ja) | 2011-04-03 | 2017-01-27 | 修飾型ｒｎａ（ｍｏｄ−ｒｎａ）を使用する効率的なインビボタンパク質発現 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021102103A Active JP7439019B2 (ja) | 2011-04-03 | 2021-06-21 | 修飾型ｒｎａ（ｍｏｄ－ｒｎａ）を使用する効率的なインビボタンパク質発現 |
Country Status (4)
| Country | Link |
|---|---|
| US (3) | US10086043B2 (enExample) |
| EP (2) | EP2694660B1 (enExample) |
| JP (4) | JP2014511694A (enExample) |
| WO (1) | WO2012138453A1 (enExample) |
Families Citing this family (89)
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| EP3578205A1 (en) | 2010-08-06 | 2019-12-11 | ModernaTX, Inc. | A pharmaceutical formulation comprising engineered nucleic acids and medical use thereof |
| CN103429606A (zh) | 2010-10-01 | 2013-12-04 | 现代治疗公司 | 设计核酸及其使用方法 |
| CA2831613A1 (en) | 2011-03-31 | 2012-10-04 | Moderna Therapeutics, Inc. | Delivery and formulation of engineered nucleic acids |
| US10086043B2 (en) * | 2011-04-03 | 2018-10-02 | The General Hospital Corporation | Efficient protein expression in vivo using modified RNA (MOD-RNA) |
| US9464124B2 (en) | 2011-09-12 | 2016-10-11 | Moderna Therapeutics, Inc. | Engineered nucleic acids and methods of use thereof |
| KR20190099538A (ko) | 2011-10-03 | 2019-08-27 | 모더나 세라퓨틱스, 인코포레이티드 | 변형된 뉴클레오사이드, 뉴클레오타이드, 및 핵산, 및 이들의 용도 |
| BR112014013664A2 (pt) | 2011-12-05 | 2020-11-03 | Factor Bioscience Inc. | métodos e produtos para transfectar células |
| US8497124B2 (en) | 2011-12-05 | 2013-07-30 | Factor Bioscience Inc. | Methods and products for reprogramming cells to a less differentiated state |
| EP4144378A1 (en) | 2011-12-16 | 2023-03-08 | ModernaTX, Inc. | Modified nucleoside, nucleotide, and nucleic acid compositions |
| US9572897B2 (en) | 2012-04-02 | 2017-02-21 | Modernatx, Inc. | Modified polynucleotides for the production of cytoplasmic and cytoskeletal proteins |
| AU2013243954A1 (en) | 2012-04-02 | 2014-10-30 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of cosmetic proteins and peptides |
| US9254311B2 (en) | 2012-04-02 | 2016-02-09 | Moderna Therapeutics, Inc. | Modified polynucleotides for the production of proteins |
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| BR122019025681B1 (pt) | 2012-11-01 | 2023-04-18 | Factor Bioscience Inc | Método para inserir uma sequência de ácido nucleico em uma localização segura de um genoma de uma célula |
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| 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 |
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| EP3708184A1 (en) | 2013-03-27 | 2020-09-16 | The General Hospital Corporation | Methods and agents for treating alzheimer s disease |
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| PT3981437T (pt) | 2014-04-23 | 2025-01-15 | Modernatx Inc | Vacinas de ácidos nucleicos |
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2012
- 2012-03-12 US US14/009,351 patent/US10086043B2/en active Active
- 2012-03-12 WO PCT/US2012/028802 patent/WO2012138453A1/en not_active Ceased
- 2012-03-12 EP EP12767300.2A patent/EP2694660B1/en active Active
- 2012-03-12 JP JP2014503668A patent/JP2014511694A/ja not_active Withdrawn
- 2012-03-12 EP EP18183738.6A patent/EP3460064B8/en active Active
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2017
- 2017-01-27 JP JP2017012661A patent/JP2017128570A/ja not_active Withdrawn
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2018
- 2018-06-22 US US16/015,460 patent/US20190117733A1/en not_active Abandoned
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2019
- 2019-04-18 JP JP2019078998A patent/JP6902570B6/ja active Active
- 2019-07-23 US US16/519,607 patent/US12357675B2/en active Active
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2021
- 2021-06-21 JP JP2021102103A patent/JP7439019B2/ja active Active
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