JP2021522248A5 - - Google Patents
Info
- Publication number
- JP2021522248A5 JP2021522248A5 JP2020559465A JP2020559465A JP2021522248A5 JP 2021522248 A5 JP2021522248 A5 JP 2021522248A5 JP 2020559465 A JP2020559465 A JP 2020559465A JP 2020559465 A JP2020559465 A JP 2020559465A JP 2021522248 A5 JP2021522248 A5 JP 2021522248A5
- Authority
- JP
- Japan
- Prior art keywords
- composition
- cationic
- nucleic acid
- additive
- nano
- 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
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| EP18169325.0 | 2018-04-25 | ||
| EP18169325 | 2018-04-25 | ||
| EP18189010.4 | 2018-08-14 | ||
| EP18189010 | 2018-08-14 | ||
| PCT/EP2019/060646 WO2019207061A1 (en) | 2018-04-25 | 2019-04-25 | Cryoprotective agents for particulate formulations |
Publications (3)
| Publication Number | Publication Date |
|---|---|
| JP2021522248A JP2021522248A (ja) | 2021-08-30 |
| JP2021522248A5 true JP2021522248A5 (enExample) | 2022-04-28 |
| JP7256824B2 JP7256824B2 (ja) | 2023-04-12 |
Family
ID=66240151
Family Applications (2)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020559465A Active JP7256824B2 (ja) | 2018-04-25 | 2019-04-25 | 粒子状製剤用の凍結保護剤 |
| JP2020558887A Pending JP2021522228A (ja) | 2018-04-25 | 2019-04-25 | Rnaの送達用の脂質に基づく製剤 |
Family Applications After (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2020558887A Pending JP2021522228A (ja) | 2018-04-25 | 2019-04-25 | Rnaの送達用の脂質に基づく製剤 |
Country Status (13)
| Country | Link |
|---|---|
| US (2) | US20210137840A1 (enExample) |
| EP (3) | EP4311541A3 (enExample) |
| JP (2) | JP7256824B2 (enExample) |
| KR (2) | KR20210022535A (enExample) |
| CN (2) | CN112153986B (enExample) |
| AU (2) | AU2019258679B2 (enExample) |
| BR (2) | BR112020021054A2 (enExample) |
| CA (1) | CA3098259C (enExample) |
| ES (2) | ES2960936T3 (enExample) |
| MX (2) | MX2020011167A (enExample) |
| PL (1) | PL3784285T3 (enExample) |
| WO (2) | WO2019207060A1 (enExample) |
| ZA (2) | ZA202006025B (enExample) |
Families Citing this family (50)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| ES2795110T3 (es) | 2011-06-08 | 2020-11-20 | Translate Bio Inc | Lípidos escindibles |
| RU2013154295A (ru) | 2011-06-08 | 2015-07-20 | Шир Хьюман Дженетик Терапис, Инк. | КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК |
| BR112014024131A2 (pt) | 2012-03-29 | 2017-07-25 | Shire Human Genetic Therapies | lipídios catiônicos ionizáveis |
| RS57739B1 (sr) | 2013-03-14 | 2018-12-31 | Translate Bio Inc | Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima |
| BR112015022505A2 (pt) | 2013-03-14 | 2017-10-24 | Shire Human Genetic Therapies | avaliação quantitativa para eficiência do cap de rna mensageiro |
| ES2708562T3 (es) | 2013-03-14 | 2019-04-10 | Translate Bio Inc | Evaluación cuantitativa de la eficacidad de tapa de ARN mensajero |
| EP3750903A1 (en) | 2013-03-14 | 2020-12-16 | Translate Bio, Inc. | Ribonucleic acids with 4'-thio-modified nucleotides and related methods |
| EP3388834B1 (en) | 2013-03-15 | 2020-04-15 | Translate Bio, Inc. | Synergistic enhancement of the delivery of nucleic acids via blended formulations |
| JP6506749B2 (ja) | 2013-10-22 | 2019-04-24 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | フェニルケトン尿症のためのmRNA療法 |
| CN106413811A (zh) | 2013-10-22 | 2017-02-15 | 夏尔人类遗传性治疗公司 | 精氨基琥珀酸合成酶缺乏症的mrna疗法 |
| ES3032935T3 (en) | 2013-10-22 | 2025-07-29 | Translate Bio Inc | Lipid formulations for delivery of messenger rna |
| AU2014340149B2 (en) | 2013-10-22 | 2020-12-24 | Shire Human Genetic Therapies, Inc. | CNS delivery of mRNA and uses thereof |
| JP6557722B2 (ja) | 2014-05-30 | 2019-08-07 | シャイアー ヒューマン ジェネティック セラピーズ インコーポレイテッド | 核酸の送達のための生分解性脂質 |
| JP6767976B2 (ja) | 2014-12-05 | 2020-10-14 | トランスレイト バイオ, インコーポレイテッド | 関節疾患の治療のためのメッセンジャーrna治療法 |
| EP3469074B1 (en) | 2016-06-13 | 2020-12-09 | Translate Bio, Inc. | Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency |
| CA3043033A1 (en) | 2016-11-10 | 2018-05-17 | Translate Bio, Inc. | Improved ice-based lipid nanoparticle formulation for delivery of mrna |
| CA3054321A1 (en) | 2017-02-27 | 2018-08-30 | Translate Bio, Inc. | Methods for purification of messenger rna |
| EP3638716B1 (en) | 2017-06-12 | 2022-01-05 | Translate Bio, Inc. | Poly(phosphoesters) for delivery of nucleic acids |
| EP3641834B1 (en) | 2017-06-19 | 2023-10-04 | Translate Bio, Inc. | Messenger rna therapy for the treatment of friedreich's ataxia |
| US11167043B2 (en) | 2017-12-20 | 2021-11-09 | Translate Bio, Inc. | Composition and methods for treatment of ornithine transcarbamylase deficiency |
| US20210137840A1 (en) | 2018-04-25 | 2021-05-13 | Ethris Gmbh | Lipid-based formulations for the delivery of rna |
| US11964051B2 (en) | 2018-05-16 | 2024-04-23 | Translate Bio, Inc. | Ribose cationic lipids |
| JP7488193B2 (ja) | 2018-05-24 | 2024-05-21 | トランスレイト バイオ, インコーポレイテッド | チオエステルカチオン性脂質 |
| US12240824B2 (en) | 2018-05-30 | 2025-03-04 | Translate Bio, Inc. | Vitamin cationic lipids |
| WO2019232208A1 (en) | 2018-05-30 | 2019-12-05 | Translate Bio, Inc. | Cationic lipids comprising a steroidal moiety |
| CA3101454A1 (en) | 2018-05-30 | 2019-12-05 | Translate Bio, Inc. | Messenger rna vaccines and uses thereof |
| JP7422977B2 (ja) | 2018-07-23 | 2024-01-29 | トランスレイト バイオ, インコーポレイテッド | メッセンジャーrna用乾燥粉末製剤 |
| WO2020035460A1 (en) * | 2018-08-14 | 2020-02-20 | Ethris Gmbh | Lipid-based formulations containing salts for the delivery of rna |
| KR102950141B1 (ko) | 2018-08-24 | 2026-04-07 | 트랜슬레이트 바이오 인코포레이티드 | 전령 rna의 정제 방법 |
| EP3849617A1 (en) | 2018-09-14 | 2021-07-21 | Translate Bio, Inc. | Composition and methods for treatment of methylmalonic acidemia |
| KR20210089648A (ko) | 2018-11-08 | 2021-07-16 | 트랜슬레이트 바이오 인코포레이티드 | 메신저 rna 정제를 위한 방법 및 조성물 |
| CA3119976A1 (en) | 2018-11-21 | 2020-05-28 | Translate Bio, Inc. | Cationic lipid compounds |
| WO2020106946A1 (en) | 2018-11-21 | 2020-05-28 | Translate Bio, Inc. | TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR |
| JP2022516356A (ja) | 2019-01-07 | 2022-02-25 | トランスレイト バイオ, インコーポレイテッド | 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 |
| AU2020275885B2 (en) | 2019-05-15 | 2026-02-05 | Translate Bio, Inc. | Methods for purification of messenger RNA |
| CA3146675A1 (en) | 2019-07-23 | 2021-01-28 | Translate Bio, Inc. | Stable compositions of mrna-loaded lipid nanoparticles and processes of making |
| EP4031662A1 (en) | 2019-09-20 | 2022-07-27 | Translate Bio, Inc. | Mrna encoding engineered cftr |
| WO2021072172A1 (en) | 2019-10-09 | 2021-04-15 | Translate Bio, Inc. | Compositions, methods and uses of messenger rna |
| US20230399390A1 (en) | 2019-10-24 | 2023-12-14 | Novago Therapeutics Ag | Novel anti-nogo-a antibodies |
| JP2023508881A (ja) | 2019-12-20 | 2023-03-06 | トランスレイト バイオ, インコーポレイテッド | Mrna担持脂質ナノ粒子を調製する改善されたプロセス |
| CN115427021A (zh) | 2020-02-25 | 2022-12-02 | 翻译生物公司 | 制备负载mrna的脂质纳米颗粒的改进方法 |
| WO2021226468A1 (en) | 2020-05-07 | 2021-11-11 | Translate Bio, Inc. | Improved compositions for cftr mrna therapy |
| US12514932B2 (en) | 2020-05-14 | 2026-01-06 | Translate Bio, Inc. | PEG lipidoid compounds |
| US20230242624A1 (en) | 2020-06-02 | 2023-08-03 | Neurimmune Ag | HUMAN ANTIBODIES AGAINST SEVERE ACUTE RESPIRATORY SYNDROME CORONAVIRUS-2 (SARS-CoV-2) |
| WO2025056938A1 (en) * | 2023-09-11 | 2025-03-20 | BioNTech SE | Rna compositions for delivery of incretin agents |
| WO2022082001A1 (en) | 2020-10-15 | 2022-04-21 | Translate Bio, Inc. | Large scale synthesis of messenger rna |
| AU2021373892A1 (en) | 2020-11-09 | 2023-07-06 | Translate Bio, Inc. | Improved compositions for delivery of codon-optimized mrna |
| WO2023009422A1 (en) * | 2021-07-26 | 2023-02-02 | Modernatx, Inc. | Processes for preparing lipid nanoparticle compositions for the delivery of payload molecules to airway epithelium |
| WO2025057088A1 (en) * | 2023-09-11 | 2025-03-20 | BioNTech SE | Rna compositions for delivery of incretin agents |
| WO2026006617A1 (en) * | 2024-06-27 | 2026-01-02 | Genentech, Inc. | Lyophilized and frozen mrna-lnp formulations |
Family Cites Families (40)
| 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 |
| CA1223831A (en) | 1982-06-23 | 1987-07-07 | Dean Engelhardt | Modified nucleotides, methods of preparing and utilizing and compositions containing the same |
| JPS607932A (ja) * | 1983-06-29 | 1985-01-16 | Dai Ichi Seiyaku Co Ltd | リポソーム懸濁液およびその製法 |
| 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 |
| US5840710A (en) | 1994-12-09 | 1998-11-24 | Genzyme Corporation | Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules |
| JPH11504631A (ja) | 1995-04-25 | 1999-04-27 | バイカル インコーポレイテッド | Dna/脂質複合体の単一バイアル製剤 |
| US6017700A (en) | 1995-08-04 | 2000-01-25 | Bayer Corporation | Cationic oligonucleotides, and related methods of synthesis and use |
| PT1023050E (pt) * | 1997-06-27 | 2013-12-04 | Abraxis Bioscience Llc | Novas formulações de agentes farmacológicos, métodos para a sua preparação e métodos para a sua utilização |
| TW520294B (en) | 1998-01-08 | 2003-02-11 | Res Dev Foundation | Stabilization of lipid: DNA formulations during nebulization |
| JP2008501336A (ja) * | 2004-06-02 | 2008-01-24 | ソースフアーム・インコーポレイテツド | Rnaを含有する微小胞およびそのための方法 |
| CN1981044B (zh) * | 2004-06-07 | 2010-05-05 | 普洛体维生物治疗公司 | 包封干扰rna的脂质 |
| PL2578685T3 (pl) | 2005-08-23 | 2020-01-31 | The Trustees Of The University Of Pennsylvania | Rna zawierający zmodyfikowane nukleozydy i sposoby jego zastosowania |
| CA2631714C (en) | 2005-12-02 | 2014-09-16 | Novartis Ag | Nanoparticles for use in immunogenic compositions |
| NZ569138A (en) | 2005-12-15 | 2012-06-29 | Centre Nat Rech Scient | Cationic oligonucleotides automated methods for preparing same and their uses |
| CN101686939B (zh) | 2007-04-17 | 2013-03-27 | 巴克斯特国际公司 | 用于肺部投送的核酸微粒 |
| GB0801876D0 (en) * | 2008-02-01 | 2008-03-12 | Vectura Group Plc | Suspension formulations |
| JP2012505913A (ja) * | 2008-10-16 | 2012-03-08 | マリーナ バイオテック,インコーポレイテッド | 遺伝子発現を抑制する治療におけるリポソームによる効率的な送達のプロセスおよび組成物 |
| EP3269395A1 (en) | 2008-11-07 | 2018-01-17 | Massachusetts Institute Of Technology | Aminoalcohol lipidoids and uses thereof |
| CA2769670C (en) | 2009-07-31 | 2018-10-02 | Ethris Gmbh | Rna with a combination of unmodified and modified nucleotides for protein expression |
| EP3296398A1 (en) | 2009-12-07 | 2018-03-21 | Arbutus Biopharma Corporation | Compositions for nucleic acid delivery |
| EP2338520A1 (de) | 2009-12-21 | 2011-06-29 | Ludwig Maximilians Universität | Konjugat mit Zielfindungsligand und dessen Verwendung |
| AU2012255194B2 (en) * | 2011-05-16 | 2017-01-05 | Danmarks Tekniske Universitet | Charge triggering of self-organized nanoparticles |
| DE19216461T1 (de) | 2011-10-03 | 2021-10-07 | Modernatx, Inc. | Modifizierte nukleoside, nukleotide und nukleinsäuren und verwendungen davon |
| GB201118704D0 (en) * | 2011-10-28 | 2011-12-14 | Univ Oxford | Cystic fibrosis treatment |
| CA3018046A1 (en) | 2011-12-16 | 2013-06-20 | Moderna Therapeutics, Inc. | Modified nucleoside, nucleotide, and nucleic acid compositions |
| US20150126589A1 (en) | 2012-06-08 | 2015-05-07 | Ethris Gmbh | Pulmonary Delivery of Messenger RNA |
| EP3388834B1 (en) * | 2013-03-15 | 2020-04-15 | Translate Bio, Inc. | Synergistic enhancement of the delivery of nucleic acids via blended formulations |
| US12064484B2 (en) | 2013-06-28 | 2024-08-20 | Ethris Gmbh | Compositions for introducing RNA into cells |
| AU2014348212C1 (en) * | 2013-11-18 | 2018-11-29 | Arcturus Therapeutics, Inc. | Ionizable cationic lipid for RNA delivery |
| JP2017507946A (ja) * | 2014-02-26 | 2017-03-23 | エスリス ゲーエムベーハーethris GmbH | Rnaを胃腸内投与するための組成物 |
| JP6637988B2 (ja) | 2014-11-18 | 2020-01-29 | アークトゥルス セラピューティクス, インコーポレイテッド | Rna送達のためのイオン化可能カチオン性脂質 |
| EP3034539A1 (en) | 2014-12-19 | 2016-06-22 | Ethris GmbH | Compositions for introducing nucleic acid into cells |
| JP2018509387A (ja) | 2015-01-21 | 2018-04-05 | フェーズアールエックス インコーポレイテッド | 細胞に治療および診断剤を送達するための方法、組成物、ならびにシステム |
| EP3247363A4 (en) * | 2015-01-21 | 2018-10-03 | Moderna Therapeutics, Inc. | Lipid nanoparticle compositions |
| WO2018010815A1 (en) | 2016-07-15 | 2018-01-18 | Biontech Rna Pharmaceuticals Gmbh | Formulation for administration of rna |
| US11583504B2 (en) * | 2016-11-08 | 2023-02-21 | Modernatx, Inc. | Stabilized formulations of lipid nanoparticles |
| AU2017357758B2 (en) | 2016-11-10 | 2023-11-16 | Translate Bio, Inc. | Improved process of preparing mRNA-loaded lipid nanoparticles |
| US20210137840A1 (en) * | 2018-04-25 | 2021-05-13 | Ethris Gmbh | Lipid-based formulations for the delivery of rna |
| WO2020035460A1 (en) | 2018-08-14 | 2020-02-20 | Ethris Gmbh | Lipid-based formulations containing salts for the delivery of rna |
-
2019
- 2019-04-25 US US17/050,441 patent/US20210137840A1/en not_active Abandoned
- 2019-04-25 EP EP23187109.6A patent/EP4311541A3/en not_active Withdrawn
- 2019-04-25 KR KR1020207030470A patent/KR20210022535A/ko not_active Ceased
- 2019-04-25 ES ES19718759T patent/ES2960936T3/es active Active
- 2019-04-25 PL PL19718760.2T patent/PL3784285T3/pl unknown
- 2019-04-25 WO PCT/EP2019/060644 patent/WO2019207060A1/en not_active Ceased
- 2019-04-25 CA CA3098259A patent/CA3098259C/en active Active
- 2019-04-25 CN CN201980034487.6A patent/CN112153986B/zh not_active Expired - Fee Related
- 2019-04-25 US US17/050,440 patent/US12465568B2/en active Active
- 2019-04-25 BR BR112020021054-4A patent/BR112020021054A2/pt unknown
- 2019-04-25 BR BR112020021037-4A patent/BR112020021037A2/pt unknown
- 2019-04-25 MX MX2020011167A patent/MX2020011167A/es unknown
- 2019-04-25 AU AU2019258679A patent/AU2019258679B2/en active Active
- 2019-04-25 EP EP19718760.2A patent/EP3784285B1/en active Active
- 2019-04-25 JP JP2020559465A patent/JP7256824B2/ja active Active
- 2019-04-25 ES ES19718760T patent/ES2960939T3/es active Active
- 2019-04-25 CN CN201980034447.1A patent/CN112153985B/zh active Active
- 2019-04-25 EP EP19718759.4A patent/EP3784284B1/en active Active
- 2019-04-25 JP JP2020558887A patent/JP2021522228A/ja active Pending
- 2019-04-25 WO PCT/EP2019/060646 patent/WO2019207061A1/en not_active Ceased
- 2019-04-25 MX MX2020011166A patent/MX2020011166A/es unknown
- 2019-04-25 KR KR1020207030471A patent/KR20210021943A/ko active Pending
- 2019-04-25 AU AU2019258678A patent/AU2019258678B2/en not_active Ceased
-
2020
- 2020-09-29 ZA ZA2020/06025A patent/ZA202006025B/en unknown
- 2020-09-29 ZA ZA2020/06026A patent/ZA202006026B/en unknown
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