RU2619453C2 - КОНЪЮГАТЫ ДЛЯ ДОСТАВКИ ПОЛИНУКЛЕОТИДОВ in vivo, СОДЕРЖАЩИЕ ЧУВСТВИТЕЛЬНЫЕ К ФЕРМЕНТАТИВНОМУ РАСЩЕПЛЕНИЮ СВЯЗИ - Google Patents

КОНЪЮГАТЫ ДЛЯ ДОСТАВКИ ПОЛИНУКЛЕОТИДОВ in vivo, СОДЕРЖАЩИЕ ЧУВСТВИТЕЛЬНЫЕ К ФЕРМЕНТАТИВНОМУ РАСЩЕПЛЕНИЮ СВЯЗИ Download PDF

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RU2619453C2
RU2619453C2 RU2013117286A RU2013117286A RU2619453C2 RU 2619453 C2 RU2619453 C2 RU 2619453C2 RU 2013117286 A RU2013117286 A RU 2013117286A RU 2013117286 A RU2013117286 A RU 2013117286A RU 2619453 C2 RU2619453 C2 RU 2619453C2
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polymer
mmol
polynucleotide
product
compound
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RU2013117286A
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RU2013117286A (ru
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Дэвид Б. Розема
Дэвид Л. Льюис
Даррен Х. Вэйкфилд
Эрик А. Китас
Филипп ХАДВИГЕР
Джон А. ВОЛФ
Инго РОЭЛЬ
Питер МОР
Торстен ХОФМАНН
Керстин ЖАН-ХОФФМАН
Ханс Мартин МЮЛЛЕР
Гюнтер ОТТ
Андрей В. БЛОХИН
Джеффри С. КАРЛСОН
Джонатан Д. БЕНСОН
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Эрроухэд Фармасьютикалз, Инк.
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    • C07C233/04—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having nitrogen atoms of carboxamide groups bound to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals with carbon atoms of carboxamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton
    • C07C233/05—Carboxylic acid amides having carbon atoms of carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms having nitrogen atoms of carboxamide groups bound to hydrogen atoms or to carbon atoms of unsubstituted hydrocarbon radicals with carbon atoms of carboxamide groups bound to acyclic carbon atoms of an acyclic saturated carbon skeleton having the nitrogen atoms of the carboxamide groups bound to hydrogen atoms or to acyclic carbon atoms
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RU2013117286A 2010-12-29 2011-12-28 КОНЪЮГАТЫ ДЛЯ ДОСТАВКИ ПОЛИНУКЛЕОТИДОВ in vivo, СОДЕРЖАЩИЕ ЧУВСТВИТЕЛЬНЫЕ К ФЕРМЕНТАТИВНОМУ РАСЩЕПЛЕНИЮ СВЯЗИ RU2619453C2 (ru)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201061427936P 2010-12-29 2010-12-29
US61/427,936 2010-12-29
US13/336,028 US8426554B2 (en) 2010-12-29 2011-12-23 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages
US13/336,028 2011-12-23
PCT/US2011/067588 WO2012092373A2 (en) 2010-12-29 2011-12-28 In vivo polynucleotide delivery conjugates having enzyme sensitive linkages

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RU2013117286A RU2013117286A (ru) 2015-03-10
RU2619453C2 true RU2619453C2 (ru) 2017-05-16

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EP (1) EP2658579A4 (enrdf_load_stackoverflow)
JP (1) JP5941926B2 (enrdf_load_stackoverflow)
KR (1) KR20130136494A (enrdf_load_stackoverflow)
CN (1) CN103491982B (enrdf_load_stackoverflow)
AU (1) AU2011352204B2 (enrdf_load_stackoverflow)
BR (1) BR112013014115A2 (enrdf_load_stackoverflow)
CA (1) CA2816041C (enrdf_load_stackoverflow)
CL (2) CL2013001876A1 (enrdf_load_stackoverflow)
MX (2) MX347298B (enrdf_load_stackoverflow)
NZ (1) NZ611656A (enrdf_load_stackoverflow)
PE (1) PE20140198A1 (enrdf_load_stackoverflow)
RU (1) RU2619453C2 (enrdf_load_stackoverflow)
SG (1) SG189942A1 (enrdf_load_stackoverflow)
WO (1) WO2012092373A2 (enrdf_load_stackoverflow)
ZA (1) ZA201302896B (enrdf_load_stackoverflow)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2819894C1 (ru) * 2021-01-20 2024-05-28 Аргорна Фармасьютикалз Ко., Лтд. Лигандные соединения, конъюгаты и их применение

Families Citing this family (15)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2653438C2 (ru) 2012-11-15 2018-05-08 Рош Инновейшен Сентер Копенгаген А/С Конъюгаты олигонуклеотидов
EA201690051A1 (ru) * 2013-08-07 2016-06-30 Эрроухэд Рисерч Корпорейшн Поликонъюгаты для доставки триггеров рнк интерференции в клетки опухоли in vivo
JOP20210043A1 (ar) 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
MA45328A (fr) * 2016-04-01 2019-02-06 Avidity Biosciences Llc Compositions acide nucléique-polypeptide et utilisations de celles-ci
US12173350B2 (en) 2017-05-24 2024-12-24 Ramot At Tel-Aviv University Ltd. Chemiluminescent probes for imaging/detection of proteases
SG11202005022YA (en) 2017-12-06 2020-06-29 Avidity Biosciences Inc Compositions and methods of treating muscle atrophy and myotonic dystrophy
JP2023501020A (ja) * 2018-12-28 2023-01-18 サーナオミクス インコーポレイテッド 治療用分子の標的化送達
US20200399660A1 (en) * 2019-06-24 2020-12-24 Promega Corporation Modified polyamine polymers for delivery of biomolecules into cells
IL296387B2 (en) 2020-03-19 2024-08-01 Avidity Biosciences Inc Preparations and methods for the treatment of facial, back and arm muscle atrophy
US20230390411A1 (en) * 2021-01-28 2023-12-07 Nanjing Chempion Biotechnology Co., Ltd. Conjugate and use thereof
CA3219184A1 (en) * 2021-06-24 2022-12-29 Toru Nakazawa Fluorescent probe
KR102712976B1 (ko) * 2021-07-27 2024-10-07 국립안동대학교 산학협력단 등검은말벌 추출물을 유효성분으로 함유하는 혈전증의 예방 또는 치료용 약학적 조성물 및 건강 기능 식품
CN113621003B (zh) * 2021-09-10 2023-02-03 国科温州研究院(温州生物材料与工程研究所) 一种制备有机分子共价修饰的功能化核酸材料的方法及其应用
CA3231330A1 (en) 2021-09-16 2023-03-23 Avidity Biosciences, Inc. Compositions and methods of treating facioscapulohumeral muscular dystrophy
WO2024237099A1 (ja) * 2023-05-12 2024-11-21 五稜化薬株式会社 凍結乾燥製剤、凍結乾燥溶液、及びこれらの製造方法、並びに液剤

Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6180095B1 (en) * 1997-12-17 2001-01-30 Enzon, Inc. Polymeric prodrugs of amino- and hydroxyl-containing bioactive agents
WO2002083180A1 (en) * 2001-03-23 2002-10-24 Syntarga B.V. Elongated and multiple spacers in activatible prodrugs
US7091186B2 (en) * 2001-09-24 2006-08-15 Seattle Genetics, Inc. p-Amidobenzylethers in drug delivery agents
US20080293800A1 (en) * 2005-11-10 2008-11-27 Medarex, Inc. Cytotoxic Compounds and Conjugates
WO2009141240A1 (en) * 2008-05-20 2009-11-26 Sigma-Tau Industrie Farmaceutiche Riunite S.P.A. Novel dual targeting antitumoural conjugates
RU2430740C2 (ru) * 2006-08-18 2011-10-10 Ф.Хоффманн-Ля Рош Аг Поликонъюгаты для введения in vivo полинуклеотидов

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6214345B1 (en) * 1993-05-14 2001-04-10 Bristol-Myers Squibb Co. Lysosomal enzyme-cleavable antitumor drug conjugates
EP1242609A2 (en) * 1999-12-30 2002-09-25 Novartis AG Novel colloid synthetic vectors for gene therapy
PL358187A1 (en) * 2000-02-24 2004-08-09 Genentech, Inc. Caspase activated prodrugs therapy
US7541330B2 (en) * 2004-06-15 2009-06-02 Kosan Biosciences Incorporated Conjugates with reduced adverse systemic effects
CN102614528B (zh) * 2006-08-18 2014-02-26 箭头研究公司 用于体内递送多核苷酸的多缀合物
CA2916671C (en) * 2007-01-17 2018-01-09 Immunomedics, Inc. Polymeric carriers of therapeutic agents and recognition moieties for antibody-based targeting of disease sites
US8501930B2 (en) * 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system

Patent Citations (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6180095B1 (en) * 1997-12-17 2001-01-30 Enzon, Inc. Polymeric prodrugs of amino- and hydroxyl-containing bioactive agents
WO2002083180A1 (en) * 2001-03-23 2002-10-24 Syntarga B.V. Elongated and multiple spacers in activatible prodrugs
US7091186B2 (en) * 2001-09-24 2006-08-15 Seattle Genetics, Inc. p-Amidobenzylethers in drug delivery agents
US20080293800A1 (en) * 2005-11-10 2008-11-27 Medarex, Inc. Cytotoxic Compounds and Conjugates
RU2430740C2 (ru) * 2006-08-18 2011-10-10 Ф.Хоффманн-Ля Рош Аг Поликонъюгаты для введения in vivo полинуклеотидов
WO2009141240A1 (en) * 2008-05-20 2009-11-26 Sigma-Tau Industrie Farmaceutiche Riunite S.P.A. Novel dual targeting antitumoural conjugates

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2819894C1 (ru) * 2021-01-20 2024-05-28 Аргорна Фармасьютикалз Ко., Лтд. Лигандные соединения, конъюгаты и их применение

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MX341118B (es) 2016-08-09
JP5941926B2 (ja) 2016-06-29
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EP2658579A2 (en) 2013-11-06
WO2012092373A2 (en) 2012-07-05
RU2013117286A (ru) 2015-03-10
KR20130136494A (ko) 2013-12-12
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BR112013014115A2 (pt) 2019-09-24
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MX347298B (es) 2017-04-21
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