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 (38)

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EP2279254B1 (en) 2008-04-15 2017-07-05 Protiva Biotherapeutics Inc. 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
SI2506857T1 (en) 2009-12-01 2018-08-31 Translate Bio, Inc. Delivery of mRNA for the enrichment 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
KR20220025112A (ko) 2011-06-08 2022-03-03 샤이어 휴먼 지네틱 테라피즈 인크. Mrna 전달을 위한 지질 나노입자 조성물 및 방법
EP4074693A1 (en) 2011-06-08 2022-10-19 Translate Bio, Inc. Cleavable lipids
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
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
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
EA201591229A1 (ru) 2013-03-14 2016-01-29 Шир Хьюман Дженетик Терапис, Инк. Способы очистки матричной рнк
KR102311614B1 (ko) 2013-03-14 2021-10-08 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
EP3574923A1 (en) 2013-10-22 2019-12-04 Translate Bio, Inc. Mrna therapy for phenylketonuria
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
EP4729528A3 (en) 2014-04-25 2026-06-24 Translate Bio, Inc. Methods for purification of messenger rna
WO2015184256A2 (en) 2014-05-30 2015-12-03 Shire Human Genetic Therapies, Inc. Biodegradable lipids for delivery of nucleic acids
KR102559979B1 (ko) 2014-06-24 2023-07-25 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
WO2016004318A1 (en) 2014-07-02 2016-01-07 Shire Human Genetic Therapies, Inc. Encapsulation of messenger rna
AU2015357562B2 (en) 2014-12-05 2021-10-21 Translate Bio, Inc. Messenger RNA therapy for treatment of articular disease
EP3270894B1 (en) 2015-03-19 2021-02-24 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
ES2862412T3 (es) 2015-10-14 2021-10-07 Translate Bio Inc Modificación de enzimas relacionados con el ARN para producción mejorada
AU2016371624B2 (en) 2015-12-13 2020-08-27 Nitto Denko Corporation siRNA structures for high activity and reduced off target
MX2019010155A (es) 2017-02-27 2020-12-10 Translate Bio Inc Arnm de cftr optimizado por codón novedoso.
EP3483269A1 (en) * 2017-11-13 2019-05-15 Silence Therapeutics GmbH Products and compositions
IL270631B2 (en) 2017-05-16 2024-03-01 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
MX2020009072A (es) 2018-03-02 2020-10-08 Dicerna Pharmaceuticals Inc Composiciones y metodos para inhibir la expresion de gys2.
US11174500B2 (en) 2018-08-24 2021-11-16 Translate Bio, Inc. Methods for purification of messenger RNA
DK3864163T3 (da) 2018-10-09 2024-05-06 Univ British Columbia Sammensætninger og systemer omfattende transfektionskompetente vesikler fri for organiske opløsningsmidler og rengøringsmidler og fremgangsmåder forbundet dermed
AU2019384557B2 (en) 2018-11-21 2025-07-17 Translate Bio, Inc. Treatment of cystic fibrosis by delivery of nebulized mRNA encoding CFTR
AU2020252560A1 (en) * 2019-04-04 2021-10-28 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting gene expression in the central nervous system
CA3155003A1 (en) 2019-09-20 2021-03-25 Translate Bio, Inc. Mrna encoding engineered cftr
US12678406B2 (en) 2020-10-12 2026-07-14 Translate Bio Inc. Process of preparing ice-based lipid nanoparticles

Family Cites Families (9)

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Publication number Priority date Publication date Assignee Title
AU2004257373B2 (en) * 2003-07-16 2011-03-24 Arbutus Biopharma Corporation 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
EP1948674A4 (en) * 2005-11-02 2009-02-04 Protiva Biotherapeutics Inc MODIFIED SIRNA MOLECULES AND APPLICATIONS THEREOF
WO2008103276A2 (en) * 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions and methods for potentiated activity of biologicaly active molecules
EP2279254B1 (en) * 2008-04-15 2017-07-05 Protiva Biotherapeutics Inc. 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
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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