JP2014530602A5 - - Google Patents

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Publication number
JP2014530602A5
JP2014530602A5 JP2014534724A JP2014534724A JP2014530602A5 JP 2014530602 A5 JP2014530602 A5 JP 2014530602A5 JP 2014534724 A JP2014534724 A JP 2014534724A JP 2014534724 A JP2014534724 A JP 2014534724A JP 2014530602 A5 JP2014530602 A5 JP 2014530602A5
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JP
Japan
Prior art keywords
composition
lipid
nucleic acid
particle
item
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.)
Pending
Application number
JP2014534724A
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English (en)
Japanese (ja)
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JP2014530602A (ja
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Publication date
Application filed filed Critical
Publication of JP2014530602A publication Critical patent/JP2014530602A/ja
Publication of JP2014530602A5 publication Critical patent/JP2014530602A5/ja
Pending legal-status Critical Current

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JP2014534724A 2011-10-05 2012-10-04 アルデヒドデヒドロゲナーゼをサイレンシングするための組成物および方法 Pending JP2014530602A (ja)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201161543700P 2011-10-05 2011-10-05
US61/543,700 2011-10-05
US201261599238P 2012-02-15 2012-02-15
US61/599,238 2012-02-15

Publications (2)

Publication Number Publication Date
JP2014530602A JP2014530602A (ja) 2014-11-20
JP2014530602A5 true JP2014530602A5 (https=) 2015-09-10

Family

ID=48044157

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2014534724A Pending JP2014530602A (ja) 2011-10-05 2012-10-04 アルデヒドデヒドロゲナーゼをサイレンシングするための組成物および方法

Country Status (10)

Country Link
US (1) US8999950B2 (https=)
EP (1) EP2764007A4 (https=)
JP (1) JP2014530602A (https=)
KR (1) KR20140097143A (https=)
CN (1) CN104114571A (https=)
AU (2) AU2012318249B2 (https=)
CA (1) CA2850792A1 (https=)
HK (1) HK1200837A1 (https=)
RU (1) RU2014117690A (https=)
WO (1) WO2013052677A1 (https=)

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AU2009238175C1 (en) 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
ES2613498T3 (es) 2009-07-01 2017-05-24 Protiva Biotherapeutics Inc. Nuevas formulaciones de lípidos para el suministro de agentes terapéuticos a tumores sólidos
HUE042177T2 (hu) 2009-12-01 2019-06-28 Translate Bio Inc Szteroidszármazék mRNS szállítására humán genetikai betegségekben
WO2012000104A1 (en) 2010-06-30 2012-01-05 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
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
HK1206644A1 (en) 2012-03-29 2016-01-15 Shire Human Genetic Therapies, Inc. Lipid-derived neutral nanoparticles
EP3536787A1 (en) 2012-06-08 2019-09-11 Translate Bio, Inc. Nuclease resistant polynucleotides and uses thereof
US10245229B2 (en) 2012-06-08 2019-04-02 Translate Bio, Inc. Pulmonary delivery of mRNA to non-lung target cells
PL3467108T3 (pl) 2013-03-14 2024-09-30 Translate Bio, Inc. Sposoby oczyszczania informacyjnego rna
KR20210122917A (ko) 2013-03-14 2021-10-12 샤이어 휴먼 지네틱 테라피즈 인크. Cftr mrna 조성물 및 관련 방법 및 사용
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
EA034103B1 (ru) 2013-10-22 2019-12-27 Транслейт Био, Инк. СПОСОБ ЛЕЧЕНИЯ ФЕНИЛКЕТОНУРИИ С ПРИМЕНЕНИЕМ мРНК
MX2016005238A (es) 2013-10-22 2016-08-12 Shire Human Genetic Therapies Formulaciones de lipidos para la administracion de acido ribonucleico mensajero.
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
EP3160959B1 (en) 2014-06-24 2023-08-30 Translate Bio, Inc. Stereochemically enriched compositions for delivery of nucleic acids
BR112016030852A2 (pt) 2014-07-02 2018-01-16 Shire Human Genetic Therapies encapsulação de rna mensageiro
WO2016090262A1 (en) 2014-12-05 2016-06-09 Shire Human Genetic Therapies, Inc. Messenger rna therapy for treatment of articular disease
US10172924B2 (en) 2015-03-19 2019-01-08 Translate Bio, Inc. MRNA therapy for pompe disease
WO2016183366A2 (en) * 2015-05-12 2016-11-17 Protiva Biotherapeutics, Inc. Compositions and methods for silencing expression of hepatitis d virus rna
CN114686548A (zh) 2015-10-14 2022-07-01 川斯勒佰尔公司 用于增强生产的rna相关酶的修饰
JP6457704B2 (ja) 2015-12-13 2019-01-23 日東電工株式会社 高活性及びオフターゲット削減のためのsiRNA構造
EA201991747A1 (ru) 2017-02-27 2020-06-04 Транслейт Био, Инк. НОВАЯ КОДОН-ОПТИМИЗИРОВАННАЯ мРНК CFTR
EP3483269A1 (en) * 2017-11-13 2019-05-15 Silence Therapeutics GmbH Products and compositions
MX2019013752A (es) 2017-05-16 2020-07-20 Translate Bio Inc Tratamiento de la fibrosis quística mediante el suministro de arnm que codifica cftr optimizado en codones.
CA3118327A1 (en) * 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of aldh2 in a cell
WO2019143621A1 (en) * 2018-01-16 2019-07-25 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting aldh2 expression
AU2019227510B2 (en) * 2018-03-02 2026-02-12 Novo Nordisk A/S Compositions and methods for inhibiting GYS2 expression
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
CN113166783B (zh) 2018-10-09 2024-10-11 不列颠哥伦比亚大学 包含无有机溶剂和去污剂的转染活性囊泡的组合物和系统以及与之相关的方法
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.
CA3135402A1 (en) * 2019-04-04 2020-10-08 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression in the central nervous system

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ES2559828T3 (es) * 2003-07-16 2016-02-16 Protiva Biotherapeutics Inc. ARN de interferencia encapsulado en lípidos
US7404969B2 (en) * 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
US8101741B2 (en) * 2005-11-02 2012-01-24 Protiva Biotherapeutics, Inc. Modified siRNA molecules and uses thereof
US20100015218A1 (en) * 2007-02-16 2010-01-21 Vasant Jadhav Compositions and methods for potentiated activity of biologically active molecules
AU2009238175C1 (en) * 2008-04-15 2023-11-30 Arbutus Biopharma Corporation Novel lipid formulations for nucleic acid delivery
US20110071208A1 (en) * 2009-06-05 2011-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated dicer-substrate interfering rna
NZ597504A (en) * 2009-06-15 2013-10-25 Alnylam Pharmaceuticals Inc Lipid formulated dsrna targeting the pcsk9 gene
US8236943B2 (en) * 2009-07-01 2012-08-07 Protiva Biotherapeutics, Inc. Compositions and methods for silencing apolipoprotein B
WO2011011447A1 (en) * 2009-07-20 2011-01-27 Protiva Biotherapeutics, Inc. Compositions and methods for silencing ebola virus gene expression

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