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
Application number
JP2020559465A
Other languages
English (en)
Japanese (ja)
Other versions
JP7256824B2 (ja
JPWO2019207061A5 (https=
JP2021522248A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/EP2019/060646 external-priority patent/WO2019207061A1/en
Publication of JP2021522248A publication Critical patent/JP2021522248A/ja
Publication of JP2021522248A5 publication Critical patent/JP2021522248A5/ja
Publication of JPWO2019207061A5 publication Critical patent/JPWO2019207061A5/ja
Application granted granted Critical
Publication of JP7256824B2 publication Critical patent/JP7256824B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

JP2020559465A 2018-04-25 2019-04-25 粒子状製剤用の凍結保護剤 Active JP7256824B2 (ja)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
EP18169325 2018-04-25
EP18169325.0 2018-04-25
EP18189010 2018-08-14
EP18189010.4 2018-08-14
PCT/EP2019/060646 WO2019207061A1 (en) 2018-04-25 2019-04-25 Cryoprotective agents for particulate formulations

Publications (4)

Publication Number Publication Date
JP2021522248A JP2021522248A (ja) 2021-08-30
JP2021522248A5 true JP2021522248A5 (https=) 2022-04-28
JPWO2019207061A5 JPWO2019207061A5 (https=) 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 (https=)
EP (3) EP3784285B1 (https=)
JP (2) JP7256824B2 (https=)
KR (2) KR20210021943A (https=)
CN (2) CN112153986B (https=)
AU (2) AU2019258678B2 (https=)
BR (2) BR112020021037A2 (https=)
CA (1) CA3098259C (https=)
ES (2) ES2960939T3 (https=)
MX (2) MX2020011166A (https=)
PL (1) PL3784285T3 (https=)
WO (2) WO2019207061A1 (https=)
ZA (2) ZA202006026B (https=)

Families Citing this family (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL3586861T3 (pl) 2011-06-08 2022-05-23 Translate Bio, Inc. Kompozycje nanocząstek lipidowych i sposoby dostarczania mrna
EP3354644A1 (en) 2011-06-08 2018-08-01 Translate Bio, Inc. Cleavable lipids
ES2762873T3 (es) 2012-03-29 2020-05-26 Translate Bio Inc Lípidos catiónicos ionizables
SG11201507474QA (en) 2013-03-14 2015-10-29 Shire Human Genetic Therapies RIBONUCLEIC ACIDs WITH 4'-THIO-MODIFIED NUCLEOTIDES AND RELATED METHODS
DK2971098T3 (en) 2013-03-14 2019-02-18 Translate Bio Inc QUANTITATIVE ASSESSMENT FOR CAPE EFFECTIVENESS OF MESSENGER RNA
CN105051213A (zh) 2013-03-14 2015-11-11 夏尔人类遗传性治疗公司 信使rna加帽效率的定量评估
KR20210122917A (ko) 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
US10130649B2 (en) 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
AU2014340149B2 (en) 2013-10-22 2020-12-24 Shire Human Genetic Therapies, Inc. CNS delivery of mRNA and uses thereof
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
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
WO2016090262A1 (en) 2014-12-05 2016-06-09 Shire Human Genetic Therapies, Inc. Messenger rna therapy for treatment of articular disease
CA3027312A1 (en) 2016-06-13 2017-12-21 Translate Bio, Inc. Messenger rna therapy for the treatment of ornithine transcarbamylase deficiency
MX2019005470A (es) 2016-11-10 2019-11-21 Translate Bio Inc Formulación de nanopartículas lipídicas basadas en ice mejorada para el suministro de arnm.
WO2018157133A1 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Methods for purification of messenger rna
EP4047039A1 (en) 2017-06-12 2022-08-24 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
EP3727428A1 (en) 2017-12-20 2020-10-28 Translate Bio, Inc. Improved composition and methods for treatment of ornithine transcarbamylase deficiency
MX2020011166A (es) 2018-04-25 2022-10-19 Ethris Gmbh Agentes crioprotectores para formulaciones en forma de particulas.
JP7384832B2 (ja) 2018-05-16 2023-11-21 トランスレイト バイオ, インコーポレイテッド リボースカチオン性脂質
CN112437767B (zh) 2018-05-24 2023-10-27 川斯勒佰尔公司 硫酯阳离子脂质
JP7463006B2 (ja) 2018-05-30 2024-04-08 トランスレイト バイオ, インコーポレイテッド ステロイド性部分を含むカチオン性脂質
WO2019232095A1 (en) 2018-05-30 2019-12-05 Translate Bio, Inc. Vitamin cationic lipids
CA3101454A1 (en) 2018-05-30 2019-12-05 Translate Bio, Inc. Messenger rna vaccines and uses thereof
KR20210056331A (ko) 2018-07-23 2021-05-18 트랜슬레이트 바이오 인코포레이티드 전령 rna의 건조 분말 제형
EP3836903B1 (en) * 2018-08-14 2024-05-01 Ethris GmbH Lipid-based formulations containing salts for the delivery of rna
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
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
MX2021005969A (es) 2018-11-21 2021-09-14 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr nebulizado.
WO2020106903A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. Cationic lipid compounds and compositions thereof for use in the delivery of messenger rna
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
EP4003311A1 (en) 2019-07-23 2022-06-01 Translate Bio, Inc. Stable compositions of mrna-loaded lipid nanoparticles and processes of making
WO2021072172A1 (en) 2019-10-09 2021-04-15 Translate Bio, Inc. Compositions, methods and uses of messenger rna
WO2021079002A2 (en) 2019-10-24 2021-04-29 Novago Therapeutics Ag Novel anti-nogo-a antibodies
US12053551B2 (en) 2019-12-20 2024-08-06 Translate Bio, Inc. Process of preparing mRNA-loaded lipid nanoparticles
JP7744354B2 (ja) 2020-02-25 2025-09-25 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方法
US12605399B2 (en) 2020-05-07 2026-04-21 Translate Bio, Inc. Compositions for CFTR MRNA therapy
WO2021231697A1 (en) 2020-05-14 2021-11-18 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
EP4229191A1 (en) 2020-10-15 2023-08-23 Translate Bio, Inc. Large scale synthesis of messenger rna
JP2023548587A (ja) 2020-11-09 2023-11-17 トランスレイト バイオ, インコーポレイテッド コドン最適化したmRNAの送達のための改善された組成物
JP2024527940A (ja) * 2021-07-26 2024-07-26 モデルナティエックス インコーポレイテッド ペイロード分子を気道上皮に送達するための脂質ナノ粒子組成物を調製するプロセス
CN121844046A (zh) * 2023-09-11 2026-04-10 C·米库尔卡 用于递送肠促胰岛素剂的rna组合物
WO2026006617A1 (en) * 2024-06-27 2026-01-02 Genentech, Inc. Lyophilized and frozen mrna-lnp formulations

Family Cites Families (40)

* Cited by examiner, † Cited by third party
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
CA2214029A1 (en) * 1995-04-25 1996-10-31 Magda Marquet Single-vial formulations of dna/lipid complexes
US6017700A (en) 1995-08-04 2000-01-25 Bayer Corporation Cationic oligonucleotides, and related methods of synthesis and use
BRPI9810945B8 (pt) * 1997-06-27 2021-05-25 Abraxis Bioscience Inc formulações de agentes farmacológicos, métodos para sua preparação e métodos para uso das mesmas
TW520294B (en) * 1998-01-08 2003-02-11 Res Dev Foundation Stabilization of lipid: DNA formulations during nebulization
WO2005121369A2 (en) * 2004-06-02 2005-12-22 Sourcepharm, Inc. Rna-containing microvesicles and methods therefor
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
EP3611266B1 (en) 2005-08-23 2022-11-09 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
WO2008051245A2 (en) * 2005-12-02 2008-05-02 Novartis Ag Nanoparticles for use in immunogenic compositions
RU2451022C2 (ru) 2005-12-15 2012-05-20 Сантр Насьональ Де Ля Решерш Сьентифик (Снрс) Катионные олигонуклеотиды, автоматизированные способы их получения и их применение
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
CN102231979B (zh) * 2008-10-16 2014-10-29 玛瑞纳生物技术有限公司 基因沉默治疗剂的脂质体有效递送方法和组合物
ES2646630T3 (es) 2008-11-07 2017-12-14 Massachusetts Institute Of Technology Lipidoides aminoalcohólicos y usos de los mismos
BR112012002291A2 (pt) 2009-07-31 2016-11-29 Ethris Gmbh "polirribonucleotídeo com uma sequência que codifica uma proteína ou fragmento de proteína, implante, e, processo para a seleção de sequências de nucleotídeos"
WO2011071860A2 (en) 2009-12-07 2011-06-16 Alnylam Pharmaceuticals, Inc. Compositions for nucleic acid delivery
EP2338520A1 (de) 2009-12-21 2011-06-29 Ludwig Maximilians Universität Konjugat mit Zielfindungsligand und dessen Verwendung
CN103687588B (zh) * 2011-05-16 2017-04-12 丹麦科技大学 电荷触发的自组织纳米颗粒
RU2648950C2 (ru) 2011-10-03 2018-04-02 Модерна Терапьютикс, Инк. Модифицированные нуклеозиды, нуклеотиды и нуклеиновые кислоты и их применение
GB201118704D0 (en) * 2011-10-28 2011-12-14 Univ Oxford Cystic fibrosis treatment
CN104114572A (zh) * 2011-12-16 2014-10-22 现代治疗公司 经修饰的核苷、核苷酸和核酸组合物
EA037448B1 (ru) 2012-06-08 2021-03-30 Этрис Гмбх Способ доставки в легкие терапевтической мрнк с pei и фармацевтическая композиция
US10130649B2 (en) * 2013-03-15 2018-11-20 Translate Bio, Inc. Synergistic enhancement of the delivery of nucleic acids via blended formulations
EP3013964B1 (en) 2013-06-28 2020-05-06 ethris GmbH Compositions for introducing rna into cells
US9593077B2 (en) 2013-11-18 2017-03-14 Arcturus Therapeutics, Inc. Ionizable cationic lipid for RNA delivery
WO2015128030A1 (en) * 2014-02-26 2015-09-03 Ethris Gmbh Compositions for gastrointestinal administration of rna
KR102380363B1 (ko) 2014-11-18 2022-03-29 아크투루스 쎄라퓨틱스, 인크. Rna 전달을 위한 이온성 양이온 지질
EP3034539A1 (en) 2014-12-19 2016-06-22 Ethris GmbH Compositions for introducing nucleic acid into cells
US11219634B2 (en) 2015-01-21 2022-01-11 Genevant Sciences Gmbh Methods, compositions, and systems for delivering therapeutic and diagnostic agents into cells
US20180000953A1 (en) * 2015-01-21 2018-01-04 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
WO2018089801A1 (en) 2016-11-10 2018-05-17 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
MX2020011166A (es) 2018-04-25 2022-10-19 Ethris Gmbh Agentes crioprotectores para formulaciones en forma de particulas.
EP3836903B1 (en) 2018-08-14 2024-05-01 Ethris GmbH Lipid-based formulations containing salts for the delivery of rna

Similar Documents

Publication Publication Date Title
JP2021522248A5 (https=)
JPWO2019207061A5 (https=)
ES2314408T3 (es) Formulaciones de hfc en solucion que contienen un anticolinergico.
Myrdal et al. Advances in metered dose inhaler technology: formulation development
JP3056784B2 (ja) エアゾール医薬製剤
JP3026841B2 (ja) 医 薬
JP3210012B2 (ja) 医薬品
ES2199739T5 (es) Composiciones de aerosol.
JP3468767B2 (ja) 医薬品
JP3280974B2 (ja) 医 薬
JP2021522228A5 (https=)
JPH10510521A (ja) エアゾール処方物用噴射剤混合物
JPH06501710A (ja) ベクロメタゾン17,21ジプロピオネートを含んで成るエアロゾル製剤
JP2003521459A (ja) 医薬用エーロゾル製剤
JPH07503476A (ja) 医薬
JP2002541183A (ja) 医薬用エアゾール製剤
JP2016534142A5 (https=)
HUP0001339A2 (hu) Aeroszol gyógyszerkészítmény
JP2014224147A5 (https=)
JP2019524648A (ja) ニコチン粒子および組成物
CN102238939B (zh) 药物气溶胶组合物
BR102012026195B1 (pt) método de preparação de uma formulação de nicotina adequada para inalação e método para a realização de uma terapia de substituição ou retirada de tabaco
CN102905694B (zh) 吸入溶液
JP5938476B2 (ja) 呼吸器疾患を治療するための定量噴霧式吸入剤を調製する方法
JP2005529115A5 (https=)