TW202536180A - 第十二因子（哈格曼因子）（Ｆ１２）、激肽釋放素Ｂ、血漿（夫列契因子）１（ＫＬＫＢ１）及激肽原１（ＫＮＧ１）ｉＲＮＡ組成物及其使用方法 - Google Patents

第十二因子（哈格曼因子）（Ｆ１２）、激肽釋放素Ｂ、血漿（夫列契因子）１（ＫＬＫＢ１）及激肽原１（ＫＮＧ１）ｉＲＮＡ組成物及其使用方法

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TW202536180A
TW202536180A TW114118710A TW114118710A TW202536180A TW 202536180 A TW202536180 A TW 202536180A TW 114118710 A TW114118710 A TW 114118710A TW 114118710 A TW114118710 A TW 114118710A TW 202536180 A TW202536180 A TW 202536180A
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nucleotides
dsrna
subject
dsrna agent
agent
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TW114118710A
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Chinese (zh)
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阿奇 阿奇尼
喬治 亥克爾
馬丁 馬爾
詹姆士 巴特爾
敬玄 劉
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美商阿尼拉製藥公司
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TW114118710A 2015-05-06 2016-05-05 第十二因子（哈格曼因子）（Ｆ１２）、激肽釋放素Ｂ、血漿（夫列契因子）１（ＫＬＫＢ１）及激肽原１（ＫＮＧ１）ｉＲＮＡ組成物及其使用方法 TW202536180A (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201562157890P 2015-05-06 2015-05-06
US62/157,890 2015-05-06
US201562260887P 2015-11-30 2015-11-30
US62/260,887 2015-11-30
US201562266958P 2015-12-14 2015-12-14
US62/266,958 2015-12-14

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TW202536180A true TW202536180A (zh) 2025-09-16

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TW114118710A TW202536180A (zh) 2015-05-06 2016-05-05 第十二因子（哈格曼因子）（Ｆ１２）、激肽釋放素Ｂ、血漿（夫列契因子）１（ＫＬＫＢ１）及激肽原１（ＫＮＧ１）ｉＲＮＡ組成物及其使用方法
TW105113928A TWI727948B (zh) 2015-05-06 2016-05-05 第十二因子（哈格曼因子）（Ｆ１２）、激肽釋放素Ｂ、血漿（夫列契因子）１（ＫＬＫＢ１）及激肽原１（ＫＮＧ１）ｉＲＮＡ組成物及其使用方法
TW112118563A TWI887656B (zh) 2015-05-06 2016-05-05 第十二因子（哈格曼因子）（Ｆ１２）、激肽釋放素Ｂ、血漿（夫列契因子）１（ＫＬＫＢ１）及激肽原１（ＫＮＧ１）ｉＲＮＡ組成物及其使用方法
TW110113584A TWI806034B (zh) 2015-05-06 2016-05-05 第十二因子（哈格曼因子）（Ｆ１２）、激肽釋放素Ｂ、血漿（夫列契因子）１（ＫＬＫＢ１）及激肽原１（ＫＮＧ１）ｉＲＮＡ組成物及其使用方法

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TW105113928A TWI727948B (zh) 2015-05-06 2016-05-05 第十二因子（哈格曼因子）（Ｆ１２）、激肽釋放素Ｂ、血漿（夫列契因子）１（ＫＬＫＢ１）及激肽原１（ＫＮＧ１）ｉＲＮＡ組成物及其使用方法
TW112118563A TWI887656B (zh) 2015-05-06 2016-05-05 第十二因子（哈格曼因子）（Ｆ１２）、激肽釋放素Ｂ、血漿（夫列契因子）１（ＫＬＫＢ１）及激肽原１（ＫＮＧ１）ｉＲＮＡ組成物及其使用方法
TW110113584A TWI806034B (zh) 2015-05-06 2016-05-05 第十二因子（哈格曼因子）（Ｆ１２）、激肽釋放素Ｂ、血漿（夫列契因子）１（ＫＬＫＢ１）及激肽原１（ＫＮＧ１）ｉＲＮＡ組成物及其使用方法

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US (4) US20180100150A1 (enExample)
EP (1) EP3292201A2 (enExample)
JP (4) JP6695902B2 (enExample)
KR (2) KR102827366B1 (enExample)
CN (2) CN115322991A (enExample)
AU (3) AU2016257996A1 (enExample)
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TW (4) TW202536180A (enExample)
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Families Citing this family (25)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP3271482A4 (en) 2015-03-17 2019-02-13 Arrowhead Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR INHIBITING THE GENE EXPRESSION OF FACTOR XII
EA201792444A1 (ru) 2015-05-06 2018-06-29 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ ФАКТОРА XII (ФАКТОРА ХАГЕМАНА) (F12), ПЛАЗМЕННОГО КАЛЛИКРЕИНА B (ФАКТОРА ФЛЕТЧЕРА) 1 (KLKB1) И КИНИНОГЕНА 1 (KNG1) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
US10883107B2 (en) 2016-01-08 2021-01-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting factor XII (hageman factor) (F12) and methods of use thereof
CA3048661A1 (en) * 2017-01-30 2018-08-02 Arrowhead Pharmaceuticals Inc. Compositions and methods for inhibition of factor xii gene expression
KR102656438B1 (ko) * 2017-04-11 2024-04-12 아뷰터스 바이오파마 코포레이션 표적화 조성물
EP3649243A1 (en) * 2017-07-07 2020-05-13 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing contact-activation pathway-associated diseases using irna compositions targeting factor xii (hageman factor) (f12)
CN110944675B9 (zh) 2017-12-01 2024-08-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
HRP20250192T1 (hr) 2017-12-01 2025-04-11 Suzhou Ribo Life Science Co., Ltd. Nukleinska kiselina, sastav i konjugat koji je sadrži, te metoda pripreme i uporaba
CN110997919B (zh) 2017-12-01 2024-04-02 苏州瑞博生物技术股份有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
CN110997917B (zh) 2017-12-01 2024-04-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN110945130B (zh) 2017-12-01 2024-04-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN116375774B (zh) 2017-12-29 2025-09-23 苏州瑞博生物技术股份有限公司 缀合物及其制备方法和用途
WO2020038377A1 (zh) 2018-08-21 2020-02-27 苏州瑞博生物技术有限公司 一种核酸、含有该核酸的药物组合物和缀合物及其用途
CN111655297A (zh) 2018-09-30 2020-09-11 苏州瑞博生物技术有限公司 一种siRNA缀合物及其制备方法和用途
US12496347B2 (en) 2018-12-28 2025-12-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
CN113330117B (zh) * 2019-01-18 2024-05-28 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CN113227376B (zh) 2019-05-22 2024-04-09 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN113811613B (zh) * 2019-05-22 2024-04-05 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN113891939B (zh) * 2019-05-24 2024-04-02 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
JP2023512758A (ja) 2020-02-07 2023-03-29 インテリア セラピューティクス，インコーポレイテッド カリクレイン（ｋｌｋｂ１）遺伝子編集のための組成物および方法
US20230126881A1 (en) * 2020-03-27 2023-04-27 Alnylam Pharmaceuticals, Inc. Fc FRAGMENT OF IgG RECEPTOR AND TRANSPORTER (FCGRT) iRNA COMPOSITIONS AND METHODS OF USE THEREOF
WO2022133278A2 (en) 2020-12-18 2022-06-23 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating factor xii
EP4384617A1 (en) * 2021-08-13 2024-06-19 Alnylam Pharmaceuticals, Inc. Factor xii (f12) irna compositions and methods of use thereof
TW202346586A (zh) * 2022-01-28 2023-12-01 大陸商上海舶望製藥有限公司 用於抑制前激肽釋放酶（pkk）蛋白表達的組合物和方法
WO2024169577A1 (zh) * 2023-02-14 2024-08-22 北京福元医药股份有限公司 用于抑制fxii基因表达的双链核糖核酸及其修饰物、缀合物和用途

Family Cites Families (68)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US1861608A (en) 1929-12-21 1932-06-07 Emerson Electric Mfg Co Fan and means for directing the air current therethrough
US1861108A (en) 1930-01-24 1932-05-31 Eugene O Brace Integral clutch and transmission control
US3974808A (en) 1975-07-02 1976-08-17 Ford Motor Company Air intake duct assembly
US4708708A (en) 1982-12-06 1987-11-24 International Paper Company Method and apparatus for skiving and hemming
US4897355A (en) 1985-01-07 1990-01-30 Syntex (U.S.A.) Inc. N[ω,(ω-1)-dialkyloxy]- and N-[ω,(ω-1)-dialkenyloxy]-alk-1-yl-N,N,N-tetrasubstituted ammonium lipids and uses therefor
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4920016A (en) 1986-12-24 1990-04-24 Linear Technology, Inc. Liposomes with enhanced circulation time
US4837028A (en) 1986-12-24 1989-06-06 Liposome Technology, Inc. Liposomes with enhanced circulation time
FR2645866B1 (fr) 1989-04-17 1991-07-05 Centre Nat Rech Scient Nouvelles lipopolyamines, leur preparation et leur emploi
NL8901881A (nl) 1989-07-20 1991-02-18 Rockwool Grodan Bv Drainagekoppelelement.
US5436146A (en) 1989-09-07 1995-07-25 The Trustees Of Princeton University Helper-free stocks of recombinant adeno-associated virus vectors
US5264618A (en) 1990-04-19 1993-11-23 Vical, Inc. Cationic lipids for intracellular delivery of biologically active molecules
US5283185A (en) 1991-08-28 1994-02-01 University Of Tennessee Research Corporation Method for delivering nucleic acids into cells
WO1993025673A1 (en) 1992-06-04 1993-12-23 The Regents Of The University Of California In vivo gene therapy with intron-free sequence of interest
EP0648265A4 (en) 1992-06-18 1996-12-04 Genpharm Int PROCESS FOR THE PRODUCTION OF NON-HUMAN TRANSGENIC ANIMALS HAVING AN ARTIFICIAL YEAST CHROMOSOME.
US5783701A (en) 1992-09-08 1998-07-21 Vertex Pharmaceuticals, Incorporated Sulfonamide inhibitors of aspartyl protease
EP0673431A1 (en) 1992-12-03 1995-09-27 Genzyme Corporation Gene therapy for cystic fibrosis
RU2123492C1 (ru) 1993-02-19 1998-12-20 Ниппон Синяку Ко., Лтд Производные глицерина, средство для доставки физиологически активного вещества и фармацевтическая композиция
US6054299A (en) 1994-04-29 2000-04-25 Conrad; Charles A. Stem-loop cloning vector and method
US5543152A (en) 1994-06-20 1996-08-06 Inex Pharmaceuticals Corporation Sphingosomes for enhanced drug delivery
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
US7422902B1 (en) 1995-06-07 2008-09-09 The University Of British Columbia Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
ES2231819T3 (es) 1995-06-07 2005-05-16 Inex Pharmaceuticals Corp Particulas de lipido-acido nucleico preparadas a traves de un intermedio complejo de lipido-acido nucleico hidrofobo y uso para transferir genes.
US5858397A (en) 1995-10-11 1999-01-12 University Of British Columbia Liposomal formulations of mitoxantrone
US5854033A (en) 1995-11-21 1998-12-29 Yale University Rolling circle replication reporter systems
US5858401A (en) 1996-01-22 1999-01-12 Sidmak Laboratories, Inc. Pharmaceutical composition for cyclosporines
US5994316A (en) 1996-02-21 1999-11-30 The Immune Response Corporation Method of preparing polynucleotide-carrier complexes for delivery to cells
US6034135A (en) 1997-03-06 2000-03-07 Promega Biosciences, Inc. Dimeric cationic lipids
US6436989B1 (en) 1997-12-24 2002-08-20 Vertex Pharmaceuticals, Incorporated Prodrugs of aspartyl protease inhibitors
CN1110492C (zh) 1997-12-24 2003-06-04 沃泰克斯药物股份有限公司 天冬氨酰蛋白酶抑制剂的前药
AU747597B2 (en) 1998-07-20 2002-05-16 Inex Pharmaceuticals Corporation Liposomal encapsulated nucleic acid-complexes
BR9914772A (pt) 1998-10-09 2001-12-11 Ingene Inc Conjunto de elementos genéticos, vetor, célulahospedeira, conjunto para a produção de umasequência de ácido nucléico, método para aprodução in vivo ou in vitro de uma sequência deácido nucléico, transcrição de cdna, molécula deácido nucléico inibidor, transcrição de mrna,molécula heteroduplex e composiçãofarmacêutica
MXPA01003643A (es) 1998-10-09 2003-07-21 Ingene Inc Produccion de adnss in vivo.
CO5261510A1 (es) 1999-02-12 2003-03-31 Vertex Pharma Inhibidores de aspartil proteasa
IT1318539B1 (it) 2000-05-26 2003-08-27 Italfarmaco Spa Composizioni farmaceutiche a rilascio prolungato per lasomministrazione parenterale di sostanze idrofile biologicamente
SG2011071982A (en) * 2001-05-25 2016-09-29 Univ Duke Modulators of pharmacological agents
US7063860B2 (en) 2001-08-13 2006-06-20 University Of Pittsburgh Application of lipid vehicles and use for drug delivery
US20040142325A1 (en) 2001-09-14 2004-07-22 Liat Mintz Methods and systems for annotating biomolecular sequences
AU2005248147A1 (en) * 2004-05-11 2005-12-08 Alphagen Co., Ltd. Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same
EP1802644B1 (en) * 2004-09-24 2012-11-07 Alnylam Pharmaceuticals Inc. Rnai modulation of apob and uses thereof
CA2630838A1 (en) * 2005-11-23 2007-05-31 Georg Dewald Detection and treatment of drug associated angioedema
US8178503B2 (en) * 2006-03-03 2012-05-15 International Business Machines Corporation Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes
US20070258952A1 (en) * 2006-05-04 2007-11-08 Baylor Research Institute Anti-Tumor Activity of an Oncolytic Adenovirus-Delivered Oncogene siRNA
TW200804416A (en) * 2006-06-19 2008-01-16 Nautilus Technology Llc Modified coagulation factor IX polypeptides and use thereof for treatment
WO2008011431A2 (en) * 2006-07-17 2008-01-24 Sirna Therapeutics Inc. Rna interference mediated inhibition of proprotein convertase subtilisin kexin 9 (pcsk9) gene expression using short interfering nucleic acid (sina)
CA2703264A1 (en) * 2007-10-22 2009-04-30 Georg Dewald Disorders of vasoregulation and methods of diagnosing them
PE20120115A1 (es) * 2008-11-17 2012-02-20 Hoffmann La Roche Composiciones y metodos para inhibir la expresion de genes del factor vii
NZ622843A (en) 2009-06-10 2015-10-30 Tekmira Pharmaceuticals Corp Improved lipid formulation
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
WO2011005861A1 (en) 2009-07-07 2011-01-13 Alnylam Pharmaceuticals, Inc. Oligonucleotide end caps
US9150864B2 (en) * 2010-11-08 2015-10-06 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
US9187749B2 (en) 2011-06-10 2015-11-17 Isis Pharmaceuticals, Inc. Methods for modulating factor 12 expression
MX345095B (es) * 2011-06-21 2017-01-17 Alnylam Pharmaceuticals Inc Composiciones de arni similares a angiopoyetina 3 (angptl3) y metodos para su uso.
SI3366775T2 (sl) * 2011-11-18 2025-12-31 Alnylam Pharmaceuticals, Inc. Modificirana sredstva RNAI
CN102600471A (zh) * 2012-02-15 2012-07-25 中国人民解放军军事医学科学院微生物流行病研究所 一种败血症治疗的靶点
WO2014089493A1 (en) * 2012-12-07 2014-06-12 Vanderbilt University Antibodies against factor xii and uses thereof
HK1215718A1 (zh) 2013-01-30 2016-09-09 F. Hoffmann-La Roche Ag Lna寡核苷酸碳水化合物缀合物
CN105324485B (zh) * 2013-03-14 2021-11-23 阿尔尼拉姆医药品有限公司 补体组分C5 iRNA组合物及其使用方法
MY181888A (en) * 2013-05-22 2021-01-12 Alnylam Pharmaceuticals Inc Tmprss6 irna compositions and methods of use thereof
KR102295648B1 (ko) * 2013-05-22 2021-08-31 알닐람 파마슈티칼스 인코포레이티드 ＳＥＲＰＩＮＡ1 ｉＲＮＡ 조성물 및 이의 사용 방법
EP3271482A4 (en) 2015-03-17 2019-02-13 Arrowhead Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR INHIBITING THE GENE EXPRESSION OF FACTOR XII
WO2016149020A1 (en) 2015-03-17 2016-09-22 Arrowhead Research Corporation Rna interference agents
EA201792444A1 (ru) 2015-05-06 2018-06-29 Элнилэм Фармасьютикалз, Инк. КОМПОЗИЦИИ НА ОСНОВЕ iRNA ДЛЯ ФАКТОРА XII (ФАКТОРА ХАГЕМАНА) (F12), ПЛАЗМЕННОГО КАЛЛИКРЕИНА B (ФАКТОРА ФЛЕТЧЕРА) 1 (KLKB1) И КИНИНОГЕНА 1 (KNG1) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
US10883107B2 (en) 2016-01-08 2021-01-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting factor XII (hageman factor) (F12) and methods of use thereof
KR102645243B1 (ko) 2016-11-23 2024-03-11 알닐람 파마슈티칼스 인코포레이티드 표적외 효과가 감소된 변형 rna 작용제
CA3048661A1 (en) 2017-01-30 2018-08-02 Arrowhead Pharmaceuticals Inc. Compositions and methods for inhibition of factor xii gene expression
EP3649243A1 (en) 2017-07-07 2020-05-13 Alnylam Pharmaceuticals, Inc. Methods for treating or preventing contact-activation pathway-associated diseases using irna compositions targeting factor xii (hageman factor) (f12)
EP4384617A1 (en) 2021-08-13 2024-06-19 Alnylam Pharmaceuticals, Inc. Factor xii (f12) irna compositions and methods of use thereof

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