JP2024545572A5 - - Google Patents

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Publication number
JP2024545572A5
JP2024545572A5 JP2024527340A JP2024527340A JP2024545572A5 JP 2024545572 A5 JP2024545572 A5 JP 2024545572A5 JP 2024527340 A JP2024527340 A JP 2024527340A JP 2024527340 A JP2024527340 A JP 2024527340A JP 2024545572 A5 JP2024545572 A5 JP 2024545572A5
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JP
Japan
Application number
JP2024527340A
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Japanese (ja)
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JP2024545572A (ja
JPWO2023086893A5 (https=
Filing date
Publication date
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Priority claimed from PCT/US2022/079650 external-priority patent/WO2023086893A1/en
Publication of JP2024545572A publication Critical patent/JP2024545572A/ja
Publication of JPWO2023086893A5 publication Critical patent/JPWO2023086893A5/ja
Publication of JP2024545572A5 publication Critical patent/JP2024545572A5/ja
Pending legal-status Critical Current

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JP2024527340A 2021-11-10 2022-11-10 原発性線毛機能不全の治療のための組成物及び方法 Pending JP2024545572A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202163278007P 2021-11-10 2021-11-10
US63/278,007 2021-11-10
PCT/US2022/079650 WO2023086893A1 (en) 2021-11-10 2022-11-10 Composition and methods for treatment of primary ciliary dyskinesia

Publications (3)

Publication Number Publication Date
JP2024545572A JP2024545572A (ja) 2024-12-10
JPWO2023086893A5 JPWO2023086893A5 (https=) 2025-11-17
JP2024545572A5 true JP2024545572A5 (https=) 2025-11-17

Family

ID=84829874

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2024527340A Pending JP2024545572A (ja) 2021-11-10 2022-11-10 原発性線毛機能不全の治療のための組成物及び方法

Country Status (6)

Country Link
US (1) US20240415866A1 (https=)
EP (1) EP4429713A1 (https=)
JP (1) JP2024545572A (https=)
CN (1) CN118591398A (https=)
AU (1) AU2022388747A1 (https=)
WO (1) WO2023086893A1 (https=)

Family Cites Families (53)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
US4458066A (en) 1980-02-29 1984-07-03 University Patents, Inc. Process for preparing polynucleotides
US4500707A (en) 1980-02-29 1985-02-19 University Patents, Inc. Nucleosides useful in the preparation of polynucleotides
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4668777A (en) 1981-03-27 1987-05-26 University Patents, Inc. Phosphoramidite nucleoside compounds
US4415732A (en) 1981-03-27 1983-11-15 University Patents, Inc. Phosphoramidite compounds and processes
US4401796A (en) 1981-04-30 1983-08-30 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4737323A (en) 1986-02-13 1988-04-12 Liposome Technology, Inc. Liposome extrusion method
US5153319A (en) 1986-03-31 1992-10-06 University Patents, Inc. Process for preparing polynucleotides
US5047524A (en) 1988-12-21 1991-09-10 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
US5334761A (en) 1992-08-28 1994-08-02 Life Technologies, Inc. Cationic lipids
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
US5700642A (en) 1995-05-22 1997-12-23 Sri International Oligonucleotide sizing using immobilized cleavable primers
US5744335A (en) 1995-09-19 1998-04-28 Mirus Corporation Process of transfecting a cell with a polynucleotide mixed with an amphipathic compound and a DNA-binding protein
US7799565B2 (en) 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
EP3611266B1 (en) 2005-08-23 2022-11-09 The Trustees of the University of Pennsylvania Rna containing modified nucleosides and methods of use thereof
CN104119242B (zh) 2008-10-09 2017-07-07 泰米拉制药公司 改善的氨基脂质和递送核酸的方法
ES2646630T3 (es) 2008-11-07 2017-12-14 Massachusetts Institute Of Technology Lipidoides aminoalcohólicos y usos de los mismos
MX342785B (es) 2009-06-10 2016-10-12 Alnylam Pharmaceuticals Inc Formulacion mejorada de lipido.
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"
HUE042177T2 (hu) 2009-12-01 2019-06-28 Translate Bio Inc Szteroidszármazék mRNS szállítására humán genetikai betegségekben
EP3354644A1 (en) 2011-06-08 2018-08-01 Translate Bio, Inc. Cleavable lipids
KR102451116B1 (ko) 2011-10-27 2022-10-06 메사추세츠 인스티튜트 오브 테크놀로지 약물 캡슐화 마이크로스피어를 형성할 수 있는, n-말단 상에 관능화된 아미노산 유도체
ES2762873T3 (es) 2012-03-29 2020-05-26 Translate Bio Inc Lípidos catiónicos ionizables
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
PL3467108T3 (pl) 2013-03-14 2024-09-30 Translate Bio, Inc. Sposoby oczyszczania informacyjnego rna
ES2895651T3 (es) 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
PT3134506T (pt) 2014-04-25 2019-10-31 Translate Bio Inc Métodos de purificação de rna mensageiro
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
SMT202200502T1 (it) 2014-06-25 2023-01-13 Acuitas Therapeutics Inc Nuovi lipidi e formulazioni di nanoparticelle lipidiche per l'erogazione di acidi nucleici
US9840479B2 (en) 2014-07-02 2017-12-12 Massachusetts Institute Of Technology Polyamine-fatty acid derived lipidoids and uses thereof
BR112016030852A2 (pt) 2014-07-02 2018-01-16 Shire Human Genetic Therapies encapsulação de rna mensageiro
US20180000953A1 (en) 2015-01-21 2018-01-04 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
WO2016118725A1 (en) 2015-01-23 2016-07-28 Moderna Therapeutics, Inc. Lipid nanoparticle compositions
ES2949540T3 (es) 2015-06-19 2023-09-29 Massachusetts Inst Technology 2,5-piperazinadionas sustituidas con alquenilo y su uso en composiciones para suministrar un agente a un sujeto o una célula
LT3313829T (lt) 2015-06-29 2024-08-12 Acuitas Therapeutics Inc. Lipidai ir lipidų nanodalelių sudėtys, skirtos nukleorūgščių tiekimui
EP4286012A3 (en) 2015-09-17 2024-05-29 ModernaTX, Inc. Compounds and compositions for intracellular delivery of therapeutic agents
IL307179A (en) 2015-10-28 2023-11-01 Acuitas Therapeutics Inc Novel lipids and lipid nanoparticle formulations for delivery of nucleic acids
EP3397613A1 (en) 2015-12-30 2018-11-07 Acuitas Therapeutics Inc. Lipids and lipid nanoparticle formulations for delivery of nucleic acids
TWI773666B (zh) 2016-03-30 2022-08-11 美商英特利亞醫療公司 Crispr/cas 組分之脂質奈米粒子調配物
EP4316588A3 (en) * 2016-05-27 2024-05-15 Transcriptx, Inc. Treatment of primary ciliary dyskinesia with synthetic messenger rna
WO2018089801A1 (en) 2016-11-10 2018-05-17 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
MX2019005470A (es) 2016-11-10 2019-11-21 Translate Bio Inc Formulación de nanopartículas lipídicas basadas en ice mejorada para el suministro de arnm.
CA3053814A1 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Large scale synthesis of messenger rna
EP3585892B8 (en) 2017-02-27 2022-07-13 Translate Bio, Inc. Methods for purification of messenger rna
WO2018157133A1 (en) 2017-02-27 2018-08-30 Translate Bio, Inc. Methods for purification 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
WO2021226463A1 (en) * 2020-05-07 2021-11-11 Translate Bio, Inc. Composition and methods for treatment of primary ciliary dyskinesia
KR20230175204A (ko) * 2021-03-22 2023-12-29 리코드 테라퓨틱스, 인크. 폴리뉴클레오티드 조성물, 관련된 제제, 및 이의 사용 방법

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