JP2022516356A5 - - Google Patents

Info

Publication number
JP2022516356A5
JP2022516356A5 JP2021539435A JP2021539435A JP2022516356A5 JP 2022516356 A5 JP2022516356 A5 JP 2022516356A5 JP 2021539435 A JP2021539435 A JP 2021539435A JP 2021539435 A JP2021539435 A JP 2021539435A JP 2022516356 A5 JP2022516356 A5 JP 2022516356A5
Authority
JP
Japan
Application number
JP2021539435A
Other languages
Japanese (ja)
Other versions
JPWO2020146344A5 (https=
JP2022516356A (ja
Filing date
Publication date
Application filed filed Critical
Priority claimed from PCT/US2020/012529 external-priority patent/WO2020146344A1/en
Publication of JP2022516356A publication Critical patent/JP2022516356A/ja
Publication of JPWO2020146344A5 publication Critical patent/JPWO2020146344A5/ja
Publication of JP2022516356A5 publication Critical patent/JP2022516356A5/ja
Priority to JP2024010105A priority Critical patent/JP7789103B2/ja
Pending legal-status Critical Current

Links

JP2021539435A 2019-01-07 2020-01-07 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 Pending JP2022516356A (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2024010105A JP7789103B2 (ja) 2019-01-07 2024-01-26 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

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962789414P 2019-01-07 2019-01-07
US62/789,414 2019-01-07
PCT/US2020/012529 WO2020146344A1 (en) 2019-01-07 2020-01-07 Composition and methods for treatment of primary ciliary dyskinesia

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2024010105A Division JP7789103B2 (ja) 2019-01-07 2024-01-26 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

Publications (3)

Publication Number Publication Date
JP2022516356A JP2022516356A (ja) 2022-02-25
JPWO2020146344A5 JPWO2020146344A5 (https=) 2022-12-05
JP2022516356A5 true JP2022516356A5 (https=) 2022-12-05

Family

ID=69423424

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2021539435A Pending JP2022516356A (ja) 2019-01-07 2020-01-07 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
JP2024010105A Active JP7789103B2 (ja) 2019-01-07 2024-01-26 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

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2024010105A Active JP7789103B2 (ja) 2019-01-07 2024-01-26 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

Country Status (8)

Country Link
US (3) US11559561B2 (https=)
EP (1) EP3908597A1 (https=)
JP (2) JP2022516356A (https=)
CN (1) CN113412273B (https=)
AU (2) AU2020206109B2 (https=)
CA (1) CA3125588A1 (https=)
IL (1) IL284480A (https=)
WO (1) WO2020146344A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP4316588A3 (en) 2016-05-27 2024-05-15 Transcriptx, Inc. Treatment of primary ciliary dyskinesia with synthetic 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
US20240123087A1 (en) * 2021-03-19 2024-04-18 Recode Therapeutics, Inc. Polynucleotide compositions, related formulations, and methods of use thereof
JP2024511463A (ja) 2021-03-22 2024-03-13 リコード セラピューティクス,インク. 細胞への標的送達のための組成物および方法
JP2024545572A (ja) * 2021-11-10 2024-12-10 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の治療のための組成物及び方法

Family Cites Families (49)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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
US5132418A (en) 1980-02-29 1992-07-21 University Patents, Inc. Process for preparing polynucleotides
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
US4973679A (en) 1981-03-27 1990-11-27 University Patents, Inc. Process for oligonucleo tide synthesis using phosphormidite intermediates
US4373071A (en) 1981-04-30 1983-02-08 City Of Hope Research Institute Solid-phase synthesis of polynucleotides
US4401796A (en) 1981-04-30 1983-08-30 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
US5262530A (en) 1988-12-21 1993-11-16 Applied Biosystems, Inc. Automated system for polynucleotide synthesis and purification
US5047524A (en) 1988-12-21 1991-09-10 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
EP1327684A1 (en) * 2002-01-14 2003-07-16 Universitätsklinikum Freiburg DNAH5 and its use in diagnosis
US7799565B2 (en) 2004-06-07 2010-09-21 Protiva Biotherapeutics, Inc. Lipid encapsulated interfering RNA
EP2049665A2 (en) 2006-07-28 2009-04-22 Applera Corporation Dinucleotide mrna cap analogs
US8252757B2 (en) * 2006-11-09 2012-08-28 Dynavax Technologies Corporation Long term disease modification using immunostimulatory oligonucleotides
WO2009058911A2 (en) 2007-10-31 2009-05-07 Applied Biosystems Inc. Preparation and isolation of 5' capped mrna
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.
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
ES2895651T3 (es) 2013-12-19 2022-02-22 Novartis Ag Lípidos y composiciones lipídicas para la administración de agentes activos
US20170143848A1 (en) * 2014-03-24 2017-05-25 Shire Human Genetic Therapies, Inc. Mrna therapy for the treatment of ocular diseases
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
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
US10172924B2 (en) * 2015-03-19 2019-01-08 Translate Bio, Inc. MRNA therapy for pompe disease
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
MX2019005470A (es) 2016-11-10 2019-11-21 Translate Bio Inc Formulación de nanopartículas lipídicas basadas en ice mejorada para el suministro de arnm.
WO2018089801A1 (en) 2016-11-10 2018-05-17 Translate Bio, Inc. Improved process of preparing mrna-loaded lipid nanoparticles
MA47824A (fr) * 2017-03-07 2020-01-15 Translate Bio Inc Administration polyanionique d'acides nucléiques
MX2020011166A (es) 2018-04-25 2022-10-19 Ethris Gmbh Agentes crioprotectores para formulaciones en forma de particulas.
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

Similar Documents

Publication Publication Date Title
CN306039902S (https=)
CN305857584S (https=)
CN305540769S (https=)
CN305541912S (https=)
CN305649652S (https=)
CN305650238S (https=)
CN305651081S (https=)
CN305652139S (https=)
CN305667235S (https=)
CN305667363S (https=)
CN305687932S (https=)
CN306042114S (https=)
CN305717979S (https=)
CN305730338S (https=)
CN305731015S (https=)
CN305747558S (https=)
CN305751205S (https=)
CN305751700S (https=)
CN305751861S (https=)
CN305752749S (https=)
CN305816545S (https=)
CN305817471S (https=)
CN305818070S (https=)
CN305818594S (https=)
CN305819511S (https=)