JP2018502956A5 - - Google Patents
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- JP2018502956A5 JP2018502956A5 JP2017533008A JP2017533008A JP2018502956A5 JP 2018502956 A5 JP2018502956 A5 JP 2018502956A5 JP 2017533008 A JP2017533008 A JP 2017533008A JP 2017533008 A JP2017533008 A JP 2017533008A JP 2018502956 A5 JP2018502956 A5 JP 2018502956A5
- Authority
- JP
- Japan
- Prior art keywords
- copolymer
- repeating units
- composition
- nucleic acid
- repeating
- 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.)
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- 229920001577 copolymer Polymers 0.000 claims 17
- 239000000203 mixture Substances 0.000 claims 8
- 108020004707 nucleic acids Proteins 0.000 claims 5
- 150000007523 nucleic acids Chemical class 0.000 claims 5
- 229920003118 cationic copolymer Polymers 0.000 claims 3
- 239000008194 pharmaceutical composition Substances 0.000 claims 3
- 201000010099 disease Diseases 0.000 claims 2
- RWRDLPDLKQPQOW-UHFFFAOYSA-N pyrrolidine Chemical compound C1CCNC1 RWRDLPDLKQPQOW-UHFFFAOYSA-N 0.000 claims 2
- HONIICLYMWZJFZ-UHFFFAOYSA-N Azetidine Chemical compound C1CNC1 HONIICLYMWZJFZ-UHFFFAOYSA-N 0.000 claims 1
- 108020004999 Messenger RNA Proteins 0.000 claims 1
- NOWKCMXCCJGMRR-UHFFFAOYSA-N aziridine Chemical compound C1CN1 NOWKCMXCCJGMRR-UHFFFAOYSA-N 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 229920005684 linear copolymer Polymers 0.000 claims 1
- 229920002106 messenger RNA Polymers 0.000 claims 1
- 239000000178 monomer Substances 0.000 claims 1
- 125000004433 nitrogen atoms Chemical group N* 0.000 claims 1
- 210000000056 organs Anatomy 0.000 claims 1
- 230000000379 polymerizing Effects 0.000 claims 1
- 210000001519 tissues Anatomy 0.000 claims 1
Claims (14)
- 下記式（ａ１）および（ａ２）の繰り返し単位から独立して選択される複数の繰り返し単位（ａ）と、
コポリマー中に含まれる繰り返し単位（ａ）および／または（ｂ）の窒素原子の１または２以上がプロトン化されてカチオン性コポリマーとされてもよい、統計コポリマー。 - 繰り返し単位（ａ２）、（ｂ２）および（ｂ４）から選択される繰り返し単位の１または２種以上を含む分岐または樹状コポリマーである、請求項１に記載のコポリマー。
- 繰り返し単位（ａ１）および（ｂ１）を含む直鎖コポリマーである、請求項１に記載のコポリマー。
- 繰り返し単位（ａ）および（ｂ）が、コポリマー中の全ての繰り返し単位の８０モル％以上を占める、請求項１〜３のいずれか一項に記載のコポリマー。
- 繰り返し単位（ａ１）および（ａ２）から選択される繰り返し単位（ａ）と、繰り返し単位（ｂ１）および（ｂ２）から選択される繰り返し単位（ｂ）とが、コポリマー中の全ての繰り返し単位の８０モル％以上を占める、請求項１〜３のいずれか一項に記載のコポリマー。
- コポリマーの末端基が、下記式（ｃ１）〜（ｃ３）の基から独立して選択される１または２種以上の基（ｃ）を含む、請求項１〜５のいずれか一項に記載のコポリマー。
- 繰り返し単位（ｂ）に対する繰り返し単位（ａ）のモル比が、０．８／１．０〜１．０／０．８の範囲である、請求項１〜６のいずれか一項に記載のコポリマー。
- 核酸と、請求項１〜７のいずれか一項に記載のコポリマーとを含む、組成物。
- 核酸がｍＲＮＡである、請求項８に記載の組成物。
- コポリマーがカチオン性コポリマーであり、カチオン性コポリマーが核酸と複合体を形成する、請求項８または９に記載の組成物。
- 請求項８〜１０のいずれか一項に記載の組成物と、任意で、追加の薬学的に許容される担体および／または希釈剤とを含む、医薬組成物。
- 細胞（ヒトを除く）内へ核酸を送達するための、請求項１〜７のいずれか一項に記載のコポリマー、または、請求項８〜１１のいずれか一項に記載の組成物または医薬組成物の使用。
- 組成物に含まれる核酸を、疾患を有する患者の体内の組織または器官へ提供することにより治療可能な疾患を治療するための医薬組成物を調製するための、請求項８〜１０のいずれか一項に記載の組成物の使用。
- アジリジン、アゼチジン、および、任意でピロリジンを含むモノマー混合物を重合させるステップを含む、請求項１〜７のいずれか１項に記載のコポリマーの製造方法。
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP14199439.2A EP3034539A1 (en) | 2014-12-19 | 2014-12-19 | Compositions for introducing nucleic acid into cells |
| EP14199439.2 | 2014-12-19 | ||
| PCT/EP2015/080669 WO2016097377A1 (en) | 2014-12-19 | 2015-12-18 | Compositions for introducing nucleic acid into cells |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2018502956A JP2018502956A (ja) | 2018-02-01 |
| JP2018502956A5 true JP2018502956A5 (ja) | 2019-02-07 |
| JP6745272B2 JP6745272B2 (ja) | 2020-08-26 |
Family
ID=52292658
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2017533008A Active JP6745272B2 (ja) | 2014-12-19 | 2015-12-18 | 核酸を細胞内へ導入するための組成物 |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US11020487B2 (ja) |
| EP (2) | EP3034539A1 (ja) |
| JP (1) | JP6745272B2 (ja) |
| KR (1) | KR102142180B1 (ja) |
| CN (1) | CN107001627B (ja) |
| AU (1) | AU2015366220B2 (ja) |
| BR (1) | BR112017012482B1 (ja) |
| CA (1) | CA2971284C (ja) |
| DK (1) | DK3242903T3 (ja) |
| ES (1) | ES2887404T3 (ja) |
| RU (1) | RU2715227C2 (ja) |
| WO (1) | WO2016097377A1 (ja) |
Families Citing this family (13)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP3034539A1 (en) | 2014-12-19 | 2016-06-22 | Ethris GmbH | Compositions for introducing nucleic acid into cells |
| EP3317411B1 (en) | 2015-06-30 | 2020-12-30 | ethris GmbH | Atp-binding cassette family coding polyribonucleotides and formulations thereof |
| WO2018010815A1 (en) * | 2016-07-15 | 2018-01-18 | Biontech Rna Pharmaceuticals Gmbh | Formulation for administration of rna |
| WO2019137999A1 (en) * | 2018-01-11 | 2019-07-18 | Biontech Rna Pharmaceuticals Gmbh | Formulation for administration of rna |
| CA3098259C (en) | 2018-04-25 | 2024-02-20 | Ethris Gmbh | Lipid-based formulations for the delivery of rna |
| IL293911A (en) * | 2019-12-17 | 2022-08-01 | Univ Gent | Non-viral vectors consisting of polypropylene |
| CN111249476B (zh) * | 2020-02-19 | 2023-09-26 | 深圳厚存纳米药业有限公司 | 泊洛沙姆和/或泊洛沙胺与脂质组合中性复合物纳米粒 |
| EP4297722A1 (en) | 2021-02-26 | 2024-01-03 | Ethris GmbH | Formulations for aerosol formation and aerosols for the delivery of nucleic acid |
| WO2023049814A2 (en) * | 2021-09-22 | 2023-03-30 | Sirnaomics, Inc. | Nanoparticle pharmaceutical compositions with reduced nanoparticle size and improved polydispersity index |
| CN116549626A (zh) * | 2022-01-27 | 2023-08-08 | 深圳瑞吉生物科技有限公司 | 一种载核酸脂质纳米颗粒冻干制剂及其制备方法与应用 |
| WO2024042236A1 (en) | 2022-08-26 | 2024-02-29 | Ethris Gmbh | Stable lipid or lipidoid nanoparticle suspensions |
| EP4327829A1 (en) | 2022-08-26 | 2024-02-28 | Ethris GmbH | Stabilization of lipid or lipidoid nanoparticle suspensions |
| CN116478410B (zh) * | 2023-06-20 | 2023-09-12 | 觅投克(北京)生物医学技术有限公司 | 一种菊糖修饰的聚乙烯亚胺衍生物及其制备方法和应用 |
Family Cites Families (35)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US4711955A (en) | 1981-04-17 | 1987-12-08 | Yale University | Modified nucleotides and methods of preparing and using same |
| JPS5821590A (ja) | 1981-07-31 | 1983-02-08 | 株式会社東芝 | 制御棒駆動水圧装置 |
| JPS58215490A (ja) * | 1982-06-08 | 1983-12-14 | Nippon Shokubai Kagaku Kogyo Co Ltd | 石炭−水スラリ−用分散剤 |
| CA1223831A (en) | 1982-06-23 | 1987-07-07 | Dean Engelhardt | Modified nucleotides, methods of preparing and utilizing and compositions containing the same |
| US5792608A (en) | 1991-12-12 | 1998-08-11 | Gilead Sciences, Inc. | Nuclease stable and binding competent oligomers and methods for their use |
| US5525711A (en) | 1994-05-18 | 1996-06-11 | The United States Of America As Represented By The Secretary Of The Department Of Health And Human Services | Pteridine nucleotide analogs as fluorescent DNA probes |
| FR2722506B1 (fr) | 1994-07-13 | 1996-08-14 | Rhone Poulenc Rorer Sa | Composition contenant des acides nucleiques, preparation et utilisations |
| US5656611A (en) * | 1994-11-18 | 1997-08-12 | Supratek Pharma Inc. | Polynucleotide compositions |
| DE19527102A1 (de) * | 1995-07-25 | 1997-01-30 | Bayer Ag | Neue Polyamine und ihre Verwendung in Lacken und Beschichtungen |
| US6017700A (en) | 1995-08-04 | 2000-01-25 | Bayer Corporation | Cationic oligonucleotides, and related methods of synthesis and use |
| FR2739292B1 (fr) * | 1995-09-28 | 1997-10-31 | Rhone Poulenc Rorer Sa | Composition pharmaceutique utile pour la transfection d'acides nucleiques et ses utilisations |
| CA2298061A1 (en) * | 1997-08-01 | 1999-02-11 | Supratek Pharma, Inc. | Polynucleotide compositions |
| WO2001000708A1 (de) | 1999-06-25 | 2001-01-04 | Christian Plank | Kombinationen zur einführung von nucleinsäuren in zellen |
| DE19933024C2 (de) * | 1999-07-15 | 2003-03-13 | Medinnova Ges Med Innovationen | Kationische Blockcopolymere |
| EP1297169B1 (en) | 2000-06-26 | 2012-08-08 | Ethris Gmbh | Method for transfecting cells using a magnetic field |
| US20030215395A1 (en) * | 2002-05-14 | 2003-11-20 | Lei Yu | Controllably degradable polymeric biomolecule or drug carrier and method of synthesizing said carrier |
| US20070269891A9 (en) | 2003-01-13 | 2007-11-22 | Yasunobu Tanaka | Solid surface with immobilized degradable cationic polymer for transfecting eukaryotic cells |
| JP4535229B2 (ja) | 2003-05-08 | 2010-09-01 | 国立大学法人 東京大学 | ポリエチレングリコール−ポリカチオンブロック共重合体 |
| EP1859812B1 (en) | 2005-02-10 | 2014-01-01 | The University of Tokyo | Polycation chargeable polymer and use as carrier of nucleic acid |
| AU2006259415B2 (en) | 2005-06-15 | 2012-08-30 | Massachusetts Institute Of Technology | Amine-containing lipids and uses thereof |
| LT2578685T (lt) | 2005-08-23 | 2019-06-10 | The Trustees Of The University Of Pennsylvania | Rnr, apimančios modifikuotus nukleozidus ir jų panaudojimo būdai |
| JP5346585B2 (ja) | 2005-12-15 | 2013-11-20 | サーントゥル ナシオナル ドゥ ラ ルシェルシュ シャーンティフィク （セ エン エール エス） | カチオン性オリゴヌクレオチド、同ヌクレオチドの自動調製法およびそれらの使用 |
| US7700541B2 (en) * | 2006-04-06 | 2010-04-20 | Nitto Denko Corporation | Biodegradable cationic polymers |
| GB0613753D0 (en) * | 2006-07-11 | 2006-08-23 | Norwegian Radium Hospital Res | Method |
| AU2007285782B2 (en) * | 2006-08-18 | 2010-06-24 | Arrowhead Research Corporation | Polyconjugates for in vivo delivery of polynucleotides |
| US20090042825A1 (en) * | 2007-08-06 | 2009-02-12 | Majed Matar | Composition, method of preparation & application of concentrated formulations of condensed nucleic acids with a cationic lipopolymer |
| AU2009223313A1 (en) * | 2008-03-14 | 2009-09-17 | Egen, Inc. | Biodegradable cross-linked branched poly (alkylene imines) |
| EP2365962B1 (en) | 2008-11-07 | 2017-07-05 | Massachusetts Institute of Technology | Aminoalcohol lipidoids and uses thereof |
| US20110263025A1 (en) | 2008-12-02 | 2011-10-27 | University Of Utah Research Foundation | Biodegradable polydisulfide amines for gene delivery |
| CN105255881A (zh) | 2009-07-31 | 2016-01-20 | 埃泽瑞斯公司 | 用于蛋白质表达的具有未修饰和修饰核苷酸的组合的rna |
| WO2011154331A1 (en) | 2010-06-10 | 2011-12-15 | F. Hoffmann-La Roche Ag | Polymers for delivery of nucleic acids |
| EP3682905B1 (en) | 2011-10-03 | 2021-12-01 | ModernaTX, Inc. | Modified nucleosides, nucleotides, and nucleic acids, and uses thereof |
| WO2014025795A1 (en) | 2012-08-07 | 2014-02-13 | Northeastern University | Compositions for the delivery of rna and drugs into cells |
| US10258698B2 (en) | 2013-03-14 | 2019-04-16 | Modernatx, Inc. | Formulation and delivery of modified nucleoside, nucleotide, and nucleic acid compositions |
| EP3034539A1 (en) | 2014-12-19 | 2016-06-22 | Ethris GmbH | Compositions for introducing nucleic acid into cells |
-
2014
- 2014-12-19 EP EP14199439.2A patent/EP3034539A1/en not_active Withdrawn
-
2015
- 2015-12-18 EP EP15823330.4A patent/EP3242903B1/en active Active
- 2015-12-18 AU AU2015366220A patent/AU2015366220B2/en active Active
- 2015-12-18 CA CA2971284A patent/CA2971284C/en active Active
- 2015-12-18 ES ES15823330T patent/ES2887404T3/es active Active
- 2015-12-18 KR KR1020177019543A patent/KR102142180B1/ko active IP Right Grant
- 2015-12-18 CN CN201580068845.7A patent/CN107001627B/zh active Active
- 2015-12-18 US US15/537,096 patent/US11020487B2/en active Active
- 2015-12-18 BR BR112017012482-3A patent/BR112017012482B1/pt active IP Right Grant
- 2015-12-18 WO PCT/EP2015/080669 patent/WO2016097377A1/en active Application Filing
- 2015-12-18 DK DK15823330.4T patent/DK3242903T3/da active
- 2015-12-18 JP JP2017533008A patent/JP6745272B2/ja active Active
- 2015-12-18 RU RU2017122022A patent/RU2715227C2/ru active
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