EA201792444A1 - 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 - Google Patents

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

Info

Publication number
EA201792444A1
EA201792444A1 EA201792444A EA201792444A EA201792444A1 EA 201792444 A1 EA201792444 A1 EA 201792444A1 EA 201792444 A EA201792444 A EA 201792444A EA 201792444 A EA201792444 A EA 201792444A EA 201792444 A1 EA201792444 A1 EA 201792444A1
Authority
EA
Eurasian Patent Office
Prior art keywords
factor
gene
kng1
klkb1
kininogen
Prior art date
Application number
EA201792444A
Other languages
English (en)
Inventor
Акин Акинк
Грегори Хинкл
Мартин Майер
Джеймс Батлер
Дзингсюань Лю
Original Assignee
Элнилэм Фармасьютикалз, Инк.
Priority date (The priority date 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 date listed.)
Filing date
Publication date
Application filed by Элнилэм Фармасьютикалз, Инк. filed Critical Элнилэм Фармасьютикалз, Инк.
Publication of EA201792444A1 publication Critical patent/EA201792444A1/ru

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/713Double-stranded nucleic acids or oligonucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549Sugars, nucleosides, nucleotides or nucleic acids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/02Antithrombotic agents; Anticoagulants; Platelet aggregation inhibitors
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/10Antioedematous agents; Diuretics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/12Antihypertensives
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12QMEASURING OR TESTING PROCESSES INVOLVING ENZYMES, NUCLEIC ACIDS OR MICROORGANISMS; COMPOSITIONS OR TEST PAPERS THEREFOR; PROCESSES OF PREPARING SUCH COMPOSITIONS; CONDITION-RESPONSIVE CONTROL IN MICROBIOLOGICAL OR ENZYMOLOGICAL PROCESSES
    • C12Q1/00Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions
    • C12Q1/68Measuring or testing processes involving enzymes, nucleic acids or microorganisms; Compositions therefor; Processes of preparing such compositions involving nucleic acids
    • C12Q1/6876Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes
    • C12Q1/6883Nucleic acid products used in the analysis of nucleic acids, e.g. primers or probes for diseases caused by alterations of genetic material
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/14Type of nucleic acid interfering N.A.
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3222'-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/344Position-specific modifications, e.g. on every purine, at the 3'-end
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/352Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521Methyl
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/353Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533Halogen

Abstract

Настоящее изобретение относится к средствам для RNAi, например к двухнитевым средствам для RNAi, целенаправленно воздействующим на ген плазменного калликреина В (фактора Флетчера) 1 (KLKB1), ген фактора XII (фактора Хагемана (F12) или ген кининогена 1 (KNG1), и к способам применения таких средств для RNAi для ингибирования экспрессии гена KLKB1, гена F12 и/или гена KNG1, и к способам лечения субъектов с наследственным ангионевротическим отеком (HAE) и/или нарушением, ассоциированным с контактным путем активации свертывания крови.
EA201792444A 2015-05-06 2016-05-05 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 EA201792444A1 (ru)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201562157890P 2015-05-06 2015-05-06
US201562260887P 2015-11-30 2015-11-30
US201562266958P 2015-12-14 2015-12-14
PCT/US2016/030876 WO2016179342A2 (en) 2015-05-06 2016-05-05 Factor xii (hageman factor) (f12), kallikrein b, plasma (fletcher factor) 1 (klkb1), and kininogen 1 (kng1) irna compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
EA201792444A1 true EA201792444A1 (ru) 2018-06-29

Family

ID=55971215

Family Applications (1)

Application Number Title Priority Date Filing Date
EA201792444A EA201792444A1 (ru) 2015-05-06 2016-05-05 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

Country Status (13)

Country Link
US (4) US20180100150A1 (ru)
EP (1) EP3292201A2 (ru)
JP (4) JP6695902B2 (ru)
KR (1) KR20180002688A (ru)
CN (2) CN108271386B (ru)
AU (3) AU2016257996A1 (ru)
BR (1) BR112017021967A2 (ru)
CA (1) CA2984636A1 (ru)
EA (1) EA201792444A1 (ru)
HK (1) HK1249920A1 (ru)
TW (3) TW202336231A (ru)
UY (1) UY36671A (ru)
WO (1) WO2016179342A2 (ru)

Families Citing this family (18)

* 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
WO2016179342A2 (en) 2015-05-06 2016-11-10 Alnylam Pharmaceuticals, Inc. Factor xii (hageman factor) (f12), kallikrein b, plasma (fletcher factor) 1 (klkb1), and kininogen 1 (kng1) irna compositions and methods of use thereof
US10883107B2 (en) 2016-01-08 2021-01-05 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting factor XII (hageman factor) (F12) and methods of use thereof
EP3573623A4 (en) * 2017-01-30 2020-11-25 Arrowhead Pharmaceuticals, Inc. COMPOSITIONS AND METHODS OF INHIBITION OF GENE EXPRESSION OF FACTOR XII
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)
WO2019105419A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 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
AU2018374219C1 (en) 2017-12-01 2023-05-11 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof
WO2019105435A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 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
JP7261494B2 (ja) 2017-12-01 2023-04-20 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、当該核酸を含む組成物及び複合体ならびに調製方法と使用
AU2018394875B2 (en) 2017-12-29 2023-08-03 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
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
EP3862024A4 (en) 2018-09-30 2022-08-17 Suzhou Ribo Life Science Co., Ltd. SHORT INTERFERENT RNA CONJUGATE, METHOD FOR PREPARATION AND USE THEREOF
CN113330117A (zh) * 2019-01-18 2021-08-31 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
JP2022533421A (ja) * 2019-05-22 2022-07-22 スーチョウ リボ ライフ サイエンス カンパニー、リミテッド 核酸、薬物組成物及び複合体ならびに調製方法と使用
US20220315929A1 (en) * 2019-05-24 2022-10-06 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate, preparation method therefor and use thereof
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
IL310407A (en) * 2021-08-13 2024-03-01 Alnylam Pharmaceuticals Inc Factor XII (F12) IRNA compositions and methods of using them
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

Family Cites Families (59)

* 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
EP0646178A1 (en) 1992-06-04 1995-04-05 The Regents Of The University Of California expression cassette with regularoty regions functional in the mammmlian host
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
JPH08503855A (ja) 1992-12-03 1996-04-30 ジェンザイム・コーポレイション 嚢胞性線維症に対する遺伝子治療
CA2156289C (en) 1993-02-19 2006-01-03 Junichi Yano Drug composition containing nucleic acid copolymer
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
JP4335310B2 (ja) 1995-06-07 2009-09-30 ザ ユニバーシティ オブ ブリティッシュ コロンビア 疎水性脂質−核酸複合中間体を通して調製される脂質−核酸粒子、及び遺伝子移送のための使用
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
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
IL136940A0 (en) 1997-12-24 2001-06-14 Vertex Pharma Sulphonamide derivatives and pharmaceutical compositions containing the same
US6436989B1 (en) 1997-12-24 2002-08-20 Vertex Pharmaceuticals, Incorporated Prodrugs of aspartyl protease inhibitors
CA2335393C (en) 1998-07-20 2008-09-23 Inex Pharmaceuticals Corporation Liposomal encapsulated nucleic acid-complexes
BR9914773A (pt) 1998-10-09 2002-02-05 Ingene Inc Conjunto de elementos genéricos, método para a produção de dna de cordão único, transcrição de mrna, construção de ácido nucléico, transcrição de ssdna, vetor, sistema vetor, célula hospedeira, conjunto para a produção de uma sequência de ácido nucléico de cordão único, método para a produção in vivo ou in vitro de uma sequência de ácido nucléico de cordão único, transcrição de cdna de cordão único, ácido nucléico inibidor, molécula heteroduplex, e composição farmacêutica
AU6430599A (en) 1998-10-09 2000-05-01 Cytogenix, Inc. Enzymatic synthesis of ssdna
US6319946B1 (en) 1999-02-12 2001-11-20 Vertex Pharmaceuticals Incorporated Inhibitors of aspartyl protease
IT1318539B1 (it) 2000-05-26 2003-08-27 Italfarmaco Spa Composizioni farmaceutiche a rilascio prolungato per lasomministrazione parenterale di sostanze idrofile biologicamente
DK1401853T3 (da) * 2001-05-25 2010-11-01 Univ Duke Modulatorer af farmakologiske midler
US7063860B2 (en) 2001-08-13 2006-06-20 University Of Pittsburgh Application of lipid vehicles and use for drug delivery
CA2566286A1 (en) 2004-05-11 2005-12-08 Rnai Co., Ltd. Polynucleotide causing rna interfere and method of regulating gene expression with the use of the same
CN101133074B (zh) * 2004-09-24 2012-05-30 阿尔尼拉姆医药品有限公司 APOB的RNAi调节及其用途
EP1954822A1 (en) * 2005-11-23 2008-08-13 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
EP2423306A1 (en) * 2006-06-19 2012-02-29 Catalyst Biosciences, Inc. Modified coagulation factor IX polypeptides and use thereof for treatment
EP2215256A1 (en) * 2007-10-22 2010-08-11 Georg Dewald Disorders of vasoregulation and methods of diagnosing them
CA2743249A1 (en) * 2008-11-17 2010-05-20 F. Hoffmann-La Roche Ag Compositions and methods for inhibiting expression of factor vii genes
CN104873464B (zh) 2009-06-10 2018-06-22 阿布特斯生物制药公司 改进的脂质制剂
US20110269665A1 (en) 2009-06-26 2011-11-03 Avi Biopharma, Inc. Compound and method for treating myotonic dystrophy
US9512164B2 (en) 2009-07-07 2016-12-06 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
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
RU2649367C2 (ru) 2013-01-30 2018-04-02 Ф. Хоффманн-Ля Рош Аг Конъюгаты углевода и lna-олигонуклеотида
HUE034987T2 (en) * 2013-03-14 2018-05-02 Alnylam Pharmaceuticals Inc C5 complementary component IRNS preparations and methods for their use
BR112015029276B1 (pt) * 2013-05-22 2022-07-12 Alnylam Pharmaceuticals, Inc Agente de irna fita dupla capaz de inibir a expressão de tmprss6, composição farmacêutica e uso dos mesmos
EP3271482A4 (en) 2015-03-17 2019-02-13 Arrowhead Pharmaceuticals, Inc. COMPOSITIONS AND METHODS FOR INHIBITING THE GENE EXPRESSION OF FACTOR XII
US20160272970A1 (en) 2015-03-17 2016-09-22 Arrowhead Madison Inc. RNA Interference Agents
WO2016179342A2 (en) 2015-05-06 2016-11-10 Alnylam Pharmaceuticals, Inc. Factor xii (hageman factor) (f12), kallikrein b, plasma (fletcher factor) 1 (klkb1), and kininogen 1 (kng1) irna compositions and methods of use thereof
WO2018098328A1 (en) 2016-11-23 2018-05-31 Alnylam Pharmaceuticals, Inc. Modified rna agents with reduced off-target effect
EP3573623A4 (en) 2017-01-30 2020-11-25 Arrowhead Pharmaceuticals, Inc. COMPOSITIONS AND METHODS OF INHIBITION OF GENE EXPRESSION OF FACTOR XII

Also Published As

Publication number Publication date
JP2018519797A (ja) 2018-07-26
UY36671A (es) 2016-12-30
CA2984636A1 (en) 2016-11-10
US20180100150A1 (en) 2018-04-12
WO2016179342A9 (en) 2016-12-08
WO2016179342A3 (en) 2017-01-12
US20240076664A1 (en) 2024-03-07
US20210246447A1 (en) 2021-08-12
TWI806034B (zh) 2023-06-21
AU2021258003A1 (en) 2021-11-25
CN115322991A (zh) 2022-11-11
TWI727948B (zh) 2021-05-21
AU2024200717A1 (en) 2024-04-18
CN108271386A (zh) 2018-07-10
CN108271386B (zh) 2022-07-15
US10934544B2 (en) 2021-03-02
BR112017021967A2 (pt) 2018-07-31
JP2022141707A (ja) 2022-09-29
KR20180002688A (ko) 2018-01-08
JP2020188756A (ja) 2020-11-26
HK1249920A1 (zh) 2018-11-16
JP2024037849A (ja) 2024-03-19
EP3292201A2 (en) 2018-03-14
JP6695902B2 (ja) 2020-05-20
TW201704472A (zh) 2017-02-01
WO2016179342A2 (en) 2016-11-10
TW202336231A (zh) 2023-09-16
AU2016257996A1 (en) 2017-10-26
TW202130811A (zh) 2021-08-16
US20200131513A1 (en) 2020-04-30

Similar Documents

Publication Publication Date Title
EA201792444A1 (ru) 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
MX2021006635A (es) Composiciones de acido ribonucleico de interferencia (arni) contra el virus de la hepatitis b (hbv) y metodos de uso de estas.
PH12017500669A1 (en) Compositions and methods for inhibition of hao1 (hydroxyacid oxidase 1 (glycolate oxidase)) gene expression
CY1122159T1 (el) Συνθεσεις irna του γονιδιου serpina1 και μεθοδοι χρησης τους
EA201792263A1 (ru) КОМПОЗИЦИИ НА ОСНОВЕ iRNA ПРОТИВ АНГИОПОЭТИН-ПОДОБНОГО БЕЛКА 3 (ANGPTL3) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
PH12020500622A1 (en) 17á-HYDROXYSTEROID DEHYDROGENASE TYPE 13 (HSD17B13) IRNA COMPOSITIONS AND METHODS OF USE THEREOF
EA201592225A1 (ru) КОМПОЗИЦИИ НА ОСНОВЕ iRNA TMPRSS6 И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
MX2021006053A (es) Composiciones de angiotensinogeno (agt) arni y metodos de uso de las mismas.
PH12019550076A1 (en) SERPINA1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
EA201890571A1 (ru) Способы и композиции для лечения нарушения, ассоциированного с геном пропротеинконвертазы субтилизин/кексинового типа (pcsk9)
CO2018006590A2 (es) Métodos y composiciones para el tratamiento de un trastorno asociado a serpinc1
EA201691215A1 (ru) КОМПОЗИЦИИ НА ОСНОВЕ iRNA К КОМПОНЕНТУ КОМПЛЕМЕНТА И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
EA201891539A1 (ru) Соединения, ингибирующие металлоферменты
MX2021010716A (es) Composiciones y metodos para inhibir la expresion del gen lect2.
EA201690685A1 (ru) Композиции и способы ингибирования экспрессии гена alas1
EA201791014A1 (ru) Композиции на основе irna против вируса гепатита в (hbv) и способы их применения
CL2022000539A1 (es) Composiciones y métodos para inhibir la expresión del gen lect2
EA202092726A1 (ru) КОМПОЗИЦИИ НА ОСНОВЕ иРНК ПРОТИВ АНГИОТЕНЗИНОГЕНА (AGT) И СПОСОБЫ ИХ ПРИМЕНЕНИЯ
AR104542A1 (es) COMPOSICIONES DE ARNi CONTRA FACTOR XII (FACTOR DE HAGEMAN) (F12), CALICREÍNA B, PLASMÁTICA (FACTOR DE FLETCHER) 1 (KLKB1), Y QUININÓGENO 1 (KNG1) Y MÉTODOS DE USO DE LOS MISMOS
EA202090893A3 (ru) Композиции с ирнк к pcsk9 и способы их применения