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 (enExample) 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 (enExample)
EP (1) EP2764007A4 (enExample)
JP (1) JP2014530602A (enExample)
KR (1) KR20140097143A (enExample)
CN (1) CN104114571A (enExample)
AU (2) AU2012318249B2 (enExample)
CA (1) CA2850792A1 (enExample)
HK (1) HK1200837A1 (enExample)
RU (1) RU2014117690A (enExample)
WO (1) WO2013052677A1 (enExample)

Families Citing this family (36)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
PL2279254T3 (pl) 2008-04-15 2017-11-30 Protiva Biotherapeutics Inc. Nowe preparaty lipidowe do dostarczania kwasów nukleinowych
EP2449114B9 (en) 2009-07-01 2017-04-19 Protiva Biotherapeutics Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
NZ700688A (en) 2009-12-01 2016-02-26 Shire Human Genetic Therapies Delivery of mrna for the augmentation of proteins and enzymes in human genetic diseases
US9006417B2 (en) 2010-06-30 2015-04-14 Protiva Biotherapeutics, Inc. Non-liposomal systems for nucleic acid delivery
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
EP3336082B1 (en) 2011-06-08 2020-04-15 Translate Bio, Inc. Cleavable lipids
CN103906527B (zh) 2011-06-08 2020-07-10 川斯勒佰尔公司 Mrna递送的脂质纳米颗粒组合物和方法
JP6211054B2 (ja) 2012-03-29 2017-10-11 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 脂質誘導される中性ナノ粒子
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
EP3884949A1 (en) 2012-06-08 2021-09-29 Translate Bio, Inc. Pulmonary delivery of mrna to non-lung target cells
US20150267192A1 (en) 2012-06-08 2015-09-24 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
ES2708561T3 (es) 2013-03-14 2019-04-10 Translate Bio Inc Métodos para la purificación de ARN mensajero
EP2968586B1 (en) 2013-03-14 2018-07-25 Translate Bio, Inc. Cftr mrna compositions and related methods and uses
EP3060671B1 (en) 2013-10-22 2021-12-29 Translate Bio, Inc. Cns delivery of mrna and uses thereof
EP3060258A1 (en) 2013-10-22 2016-08-31 Shire Human Genetic Therapies, Inc. Mrna therapy for phenylketonuria
EP4276176A3 (en) 2013-10-22 2024-01-10 Translate Bio, Inc. Mrna therapy for argininosuccinate synthetase deficiency
KR101988552B1 (ko) 2013-10-22 2019-09-25 샤이어 휴먼 지네틱 테라피즈 인크. 메신저 rna의 전달을 위한 지질 제형
CA2944800A1 (en) 2014-04-25 2015-10-29 Shire Human Genetic Therapies, Inc. Methods for purification of messenger rna
EP3148552B1 (en) 2014-05-30 2019-07-31 Translate Bio, Inc. Biodegradable lipids for delivery of nucleic acids
KR102511554B1 (ko) 2014-06-24 2023-03-16 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
CA2953265C (en) 2014-07-02 2023-09-26 Shire Human Genetic Therapies, Inc. Encapsulation of messenger rna
US9943595B2 (en) 2014-12-05 2018-04-17 Translate Bio, Inc. Messenger RNA therapy for treatment of articular disease
JP6895892B2 (ja) 2015-03-19 2021-06-30 トランスレイト バイオ, インコーポレイテッド ポンペ病のmRNA治療
WO2016183366A2 (en) * 2015-05-12 2016-11-17 Protiva Biotherapeutics, Inc. Compositions and methods for silencing expression of hepatitis d virus rna
AU2016338559B2 (en) 2015-10-14 2022-11-24 Translate Bio, Inc. Modification of RNA-related enzymes for enhanced production
PT3386519T (pt) 2015-12-13 2021-04-27 Nitto Denko Corp Estruturas de sirna para atividade elevada e redução de alvos extra
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
WO2018213476A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
WO2019092282A1 (en) * 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of aldh2 in a cell
EP3710588A4 (en) * 2018-01-16 2021-08-18 Dicerna Pharmaceuticals, Inc. COMPOSITIONS AND METHODS TO INHIBIT ALDH2 EXPRESSION
US11572562B2 (en) * 2018-03-02 2023-02-07 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting GYS2 expression
WO2020041793A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
EP3864163B1 (en) 2018-10-09 2024-03-20 The University of British Columbia Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto
EP3883592A1 (en) 2018-11-21 2021-09-29 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mrna encoding cftr
US20220170025A1 (en) * 2019-04-04 2022-06-02 Dicerna Pharmaceuticals Inc. Compositions and methods for inhibiting gene expression in the central nervous system

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
SG190613A1 (en) * 2003-07-16 2013-06-28 Protiva Biotherapeutics Inc Lipid encapsulated interfering rna
US7404969B2 (en) * 2005-02-14 2008-07-29 Sirna Therapeutics, Inc. Lipid nanoparticle based compositions and methods for the delivery of biologically active molecules
AU2006308765B2 (en) * 2005-11-02 2013-09-05 Arbutus Biopharma Corporation Modified siRNA molecules and uses thereof
EP2131848A4 (en) * 2007-02-16 2012-06-27 Merck Sharp & Dohme COMPOSITIONS AND METHODS FOR REINFORCED ACTIVITY OF BIOLOGICAL ACTIVE MOLECULES
PL2279254T3 (pl) * 2008-04-15 2017-11-30 Protiva Biotherapeutics Inc. Nowe preparaty lipidowe do dostarczania kwasów nukleinowych
US20110071208A1 (en) * 2009-06-05 2011-03-24 Protiva Biotherapeutics, Inc. Lipid encapsulated dicer-substrate interfering rna
CN104651408A (zh) * 2009-06-15 2015-05-27 阿尔尼拉姆医药品有限公司 靶向pcsk9基因的脂质配制的dsrna
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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