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 of JP2014530602A publication Critical patent/JP2014530602A/ja
Publication of JP2014530602A5 publication Critical patent/JP2014530602A5/ja
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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)

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CA2721333C (en) 2008-04-15 2020-12-01 Protiva Biotherapeutics, Inc. Novel lipid formulations for nucleic acid delivery
WO2011000107A1 (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
US8853377B2 (en) 2010-11-30 2014-10-07 Shire Human Genetic Therapies, Inc. mRNA for use in treatment of human genetic diseases
RU2013154295A (ru) 2011-06-08 2015-07-20 Шир Хьюман Дженетик Терапис, Инк. КОМПОЗИЦИИ ЛИПИДНЫХ НАНОЧАСТИЦ И СПОСОБЫ ДЛЯ ДОСТАВКИ мРНК
ES2795110T3 (es) 2011-06-08 2020-11-20 Translate Bio Inc Lípidos escindibles
BR112014024131A2 (pt) 2012-03-29 2017-07-25 Shire Human Genetic Therapies lipídios catiônicos ionizáveis
AU2013237874B2 (en) 2012-03-29 2018-01-18 Translate Bio, Inc. Lipid-derived neutral nanoparticles
WO2013185067A1 (en) 2012-06-08 2013-12-12 Shire Human Genetic Therapies, Inc. Nuclease resistant polynucleotides and uses thereof
EP2858679B2 (en) 2012-06-08 2024-06-05 Translate Bio, Inc. Pulmonary delivery of mrna to non-lung target cells
JP6586075B2 (ja) 2013-03-14 2019-10-02 トランスレイト バイオ, インコーポレイテッド メッセンジャーrnaの精製方法
RS57739B1 (sr) 2013-03-14 2018-12-31 Translate Bio Inc Kompozicije cftr irnk i postupci i upotrebe u vezi sa njima
JP6506749B2 (ja) 2013-10-22 2019-04-24 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 フェニルケトン尿症のためのmRNA療法
CN106413811A (zh) 2013-10-22 2017-02-15 夏尔人类遗传性治疗公司 精氨基琥珀酸合成酶缺乏症的mrna疗法
AU2014340149B2 (en) 2013-10-22 2020-12-24 Shire Human Genetic Therapies, Inc. CNS delivery of mRNA and uses thereof
ES3032935T3 (en) 2013-10-22 2025-07-29 Translate Bio Inc Lipid formulations for delivery of messenger rna
US9850269B2 (en) 2014-04-25 2017-12-26 Translate Bio, Inc. Methods for purification of messenger RNA
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
KR102559979B1 (ko) 2014-06-24 2023-07-25 샤이어 휴먼 지네틱 테라피즈 인크. 핵산의 전달용 입체화학적으로 풍부한 조성물
BR112016030852A2 (pt) 2014-07-02 2018-01-16 Shire Human Genetic Therapies encapsulação de rna mensageiro
JP6767976B2 (ja) 2014-12-05 2020-10-14 トランスレイト バイオ, インコーポレイテッド 関節疾患の治療のためのメッセンジャーrna治療法
WO2016149508A1 (en) 2015-03-19 2016-09-22 Shire Human Genetic Therapies, 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
JP6997704B2 (ja) 2015-10-14 2022-02-04 トランスレイト バイオ, インコーポレイテッド 生産性向上のためのrna関連酵素の修飾
BR112018008344A2 (pt) 2015-12-13 2018-10-30 Nitto Denko Corp molécula de ácido nucleico, composição farmacêutica, vetor ou célula, método para prevenir, tratar ou melhorar uma doença, e, uso de uma composição
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.
EP3735461A1 (en) * 2017-11-13 2020-11-11 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of aldh2 in a cell
KR20200109311A (ko) * 2018-01-16 2020-09-22 다이서나 파마수이티컬, 인크. Aldh2 발현을 억제하기 위한 조성물 및 방법
CA3092092A1 (en) * 2018-03-02 2019-09-06 Dicerna Pharmaceuticals, Inc. Compositions and methods for inhibiting gys2 expression
KR102950141B1 (ko) 2018-08-24 2026-04-07 트랜슬레이트 바이오 인코포레이티드 전령 rna의 정제 방법
HRP20240586T1 (hr) 2018-10-09 2024-07-19 The University Of British Columbia Sastavi i sustavi koji sadrže transfekcijske sposobne vezikle bez organskih otapala i deterdženta i s njim povezanim postupcima
WO2020106946A1 (en) 2018-11-21 2020-05-28 Translate Bio, Inc. TREATMENT OF CYSTIC FIBROSIS BY DELIVERY OF NEBULIZED mRNA ENCODING CFTR
BR112021019793A2 (pt) * 2019-04-04 2021-12-07 Dicerna Pharmaceuticals Inc Composições e métodos para inibir expressão de gene no sistema nervoso central
EP4031662A1 (en) 2019-09-20 2022-07-27 Translate Bio, Inc. Mrna encoding engineered cftr

Family Cites Families (9)

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Publication number Priority date Publication date Assignee Title
CA2532228C (en) * 2003-07-16 2017-02-14 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
CN101346393B (zh) * 2005-11-02 2015-07-22 普洛体维生物治疗公司 修饰的siRNA分子及其应用
CA2689042A1 (en) * 2007-02-16 2008-08-28 Merck & Co., Inc. Compositions and methods for potentiated activity of biologicaly active molecules
CA2721333C (en) * 2008-04-15 2020-12-01 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
JP5894913B2 (ja) * 2009-06-15 2016-03-30 アルナイラム ファーマシューティカルズ, インコーポレイテッドAlnylam Pharmaceuticals, Inc. Pcsk9遺伝子を標的とする、脂質で製剤化されたdsrna
EP2449106B1 (en) * 2009-07-01 2015-04-08 Protiva Biotherapeutics Inc. Compositions and methods for silencing apolipoprotein b
US8716464B2 (en) 2009-07-20 2014-05-06 Thomas W. Geisbert Compositions and methods for silencing Ebola virus gene expression

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