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
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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 (cg-RX-API-DMAC7.html) 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 (cg-RX-API-DMAC7.html)
EP (1) EP2764007A4 (cg-RX-API-DMAC7.html)
JP (1) JP2014530602A (cg-RX-API-DMAC7.html)
KR (1) KR20140097143A (cg-RX-API-DMAC7.html)
CN (1) CN104114571A (cg-RX-API-DMAC7.html)
AU (2) AU2012318249B2 (cg-RX-API-DMAC7.html)
CA (1) CA2850792A1 (cg-RX-API-DMAC7.html)
HK (1) HK1200837A1 (cg-RX-API-DMAC7.html)
RU (1) RU2014117690A (cg-RX-API-DMAC7.html)
WO (1) WO2013052677A1 (cg-RX-API-DMAC7.html)

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JP5475753B2 (ja) 2008-04-15 2014-04-16 プロチバ バイオセラピューティクス インコーポレイティッド 核酸送達用の脂質製剤
CA2767127A1 (en) 2009-07-01 2011-01-06 Protiva Biotherapeutics, Inc. Novel lipid formulations for delivery of therapeutic agents to solid tumors
EP3318248B1 (en) 2009-12-01 2019-04-10 Translate Bio, Inc. 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
WO2012075040A2 (en) 2010-11-30 2012-06-07 Shire Human Genetic Therapies, Inc. mRNA FOR USE IN TREATMENT OF HUMAN GENETIC DISEASES
CA3198966A1 (en) 2011-06-08 2012-12-13 Translate Bio, Inc. Cleavable lipids
EP2717893B1 (en) 2011-06-08 2019-05-08 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mrna delivery
CA2868034C (en) 2012-03-29 2021-07-27 Shire Human Genetic Therapies, Inc. Ionizable cationic lipids
HK1206644A1 (en) 2012-03-29 2016-01-15 Shire Human Genetic Therapies, Inc. Lipid-derived neutral nanoparticles
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
WO2013185069A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Pulmonary delivery of mrna to non-lung target cells
TR201901310T4 (tr) 2013-03-14 2019-02-21 Translate Bio Inc Mesajcı RNA'nın saflaştırılması yöntemleri.
LT2968586T (lt) 2013-03-14 2018-11-26 Translate Bio, Inc. Cft mrnr kompozicijos bei su jomis susiję būdai ir panaudojimai
BR112016009014B1 (pt) 2013-10-22 2024-02-06 Translate Bio, Inc USO DE COMPOSIÇÃO COMPREENDENDO mRNA PARA DEFICIÊNCIA DE ARGININOSSUCINATO SINTETASE
CN112618732A (zh) 2013-10-22 2021-04-09 夏尔人类遗传性治疗公司 用于递送信使rna的脂质制剂
EP3574923A1 (en) 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
CN105658800A (zh) 2013-10-22 2016-06-08 夏尔人类遗传性治疗公司 Mrna的cns递送及其用途
KR102739894B1 (ko) 2014-04-25 2024-12-05 샤이어 휴먼 지네틱 테라피즈 인크. 메신저 rna 의 정제 방법
US10022455B2 (en) 2014-05-30 2018-07-17 Translate Bio, Inc. Biodegradable lipids for delivery of nucleic acids
CN111588695A (zh) 2014-06-24 2020-08-28 川斯勒佰尔公司 用于递送核酸的立体化学富集组合物
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
AU2016233135B2 (en) 2015-03-19 2021-07-08 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
CA3001852A1 (en) 2015-10-14 2017-04-20 Translate Bio, Inc. Modification of rna-related enzymes for enhanced production
US10358647B2 (en) 2015-12-13 2019-07-23 Nitto Denko Corporation siRNA structures for high activity and reduced off target
EP3585417B1 (en) 2017-02-27 2023-02-22 Translate Bio, Inc. Method of making a codon-optimized cftr mrna
EP3483269A1 (en) * 2017-11-13 2019-05-15 Silence Therapeutics GmbH Products and compositions
CA3063531A1 (en) 2017-05-16 2018-11-22 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of codon-optimized mrna encoding cftr
EP3735461A1 (en) * 2017-11-13 2020-11-11 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of aldh2 in a cell
AU2019209324A1 (en) * 2018-01-16 2020-07-09 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting ALDH2 expression
MX2020009072A (es) * 2018-03-02 2020-10-08 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de gys2.
CA3108544A1 (en) 2018-08-24 2020-02-27 Translate Bio, Inc. Methods for purification of messenger rna
EP4379053A3 (en) 2018-10-09 2024-09-18 The University of British Columbia Compositions and systems comprising transfection-competent vesicles free of organic-solvents and detergents and methods related thereto
CA3120647A1 (en) 2018-11-21 2020-05-28 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
JP4951338B2 (ja) * 2003-07-16 2012-06-13 プロチバ バイオセラピューティクス インコーポレイティッド 脂質に封入された干渉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
EP1948674A4 (en) * 2005-11-02 2009-02-04 Protiva Biotherapeutics Inc MODIFIED SIRNA MOLECULES AND APPLICATIONS THEREOF
AU2008219165A1 (en) * 2007-02-16 2008-08-28 Merck Sharp & Dohme Corp. Compositions and methods for potentiated activity of biologicaly active molecules
JP5475753B2 (ja) * 2008-04-15 2014-04-16 プロチバ バイオセラピューティクス インコーポレイティッド 核酸送達用の脂質製剤
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
EP2449106B1 (en) * 2009-07-01 2015-04-08 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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