TW201823461A - 抑制ａｓｇｒ１表現之ｒｎａｉ構築體及其使用方法 - Google Patents

抑制ａｓｇｒ１表現之ｒｎａｉ構築體及其使用方法 Download PDF

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TW201823461A
TW201823461A TW106129226A TW106129226A TW201823461A TW 201823461 A TW201823461 A TW 201823461A TW 106129226 A TW106129226 A TW 106129226A TW 106129226 A TW106129226 A TW 106129226A TW 201823461 A TW201823461 A TW 201823461A
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seq
rnai construct
sequence
asgr1
sense strand
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TW106129226A
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Chinese (zh)
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麥克 歐爾曼
李洋
竣 張
考斯達夫 畢斯瓦茲
奧莉佛 霍曼
來斯利 P 米蘭達
賈斯汀 K 穆瑞
吳斌
尹歐奎
約翰 高登 艾倫
恰薇塔 奈提羅真那庫爾
元 鄭
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美商安美基公司
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-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
    • C12N15/1138—Non-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 receptors or cell surface proteins
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/06—Antihyperlipidemics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/10—Drugs 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
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    • C07K16/00—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2851—Immunoglobulins [IGs], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against the lectin superfamily, e.g. CD23, CD72
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2317/00—Immunoglobulins specific features
    • C07K2317/50—Immunoglobulins specific features characterized by immunoglobulin fragments
    • C07K2317/52—Constant or Fc region; Isotype
    • C07K2317/526—CH3 domain
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/317—Chemical structure of the backbone with an inverted bond, e.g. a cap structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide

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  • Biochemistry (AREA)
  • Biophysics (AREA)
  • Obesity (AREA)
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  • Hematology (AREA)
  • Physics & Mathematics (AREA)
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  • Plant Pathology (AREA)
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  • Heart & Thoracic Surgery (AREA)
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  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
TW106129226A 2016-08-26 2017-08-28 抑制ａｓｇｒ１表現之ｒｎａｉ構築體及其使用方法 TW201823461A (zh)

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US201662380216P 2016-08-26 2016-08-26
US62/380,216 2016-08-26

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TW201823461A true TW201823461A (zh) 2018-07-01

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US (2) US10870856B2 (cg-RX-API-DMAC7.html)
EP (1) EP3504333A1 (cg-RX-API-DMAC7.html)
JP (3) JP7082610B2 (cg-RX-API-DMAC7.html)
AR (1) AR110629A1 (cg-RX-API-DMAC7.html)
AU (2) AU2017315952B2 (cg-RX-API-DMAC7.html)
CA (1) CA3034463C (cg-RX-API-DMAC7.html)
MX (2) MX2019002271A (cg-RX-API-DMAC7.html)
TW (1) TW201823461A (cg-RX-API-DMAC7.html)
UY (1) UY37376A (cg-RX-API-DMAC7.html)
WO (1) WO2018039647A1 (cg-RX-API-DMAC7.html)

Families Citing this family (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2016332900C1 (en) 2015-09-29 2024-07-04 Amgen Inc. ASGR inhibitors
SI3661562T1 (sl) 2017-08-04 2025-01-31 Amgen Inc. Postopek konjugiranja CYS-MABS
WO2019079294A1 (en) * 2017-10-17 2019-04-25 Arrowhead Pharmaceuticals, Inc. RNAI-BASED AGENTS AND COMPOSITIONS FOR INHIBITING ASIALOGLYCOPROTEIN RECEPTOR 1 RECEPTOR 1
AU2018360697A1 (en) * 2017-11-01 2020-05-14 Alnylam Pharmaceuticals, Inc. Complement component C3 iRNA compositions and methods of use thereof
KR102798620B1 (ko) * 2018-06-07 2025-04-22 암젠 인크 단백질-폴리뉴클레오티드 접합체에 대한 검출 검정
EP3894554A1 (en) * 2018-12-10 2021-10-20 Amgen Inc. Chemically-modified rnai constructs and uses thereof
SG11202113112WA (en) * 2019-05-30 2021-12-30 Amgen Inc Rnai constructs for inhibiting scap expression and methods of use thereof
EP3990465A1 (en) 2019-06-25 2022-05-04 Amgen Inc. Purification methods for carbohydrate-linked oligonucleotides
US20230314417A1 (en) 2020-03-23 2023-10-05 Amgen Inc. Monoclonal antibodies to chemically-modified nucleic acids and uses thereof
CN111705063B (zh) * 2020-06-02 2023-08-08 成都中科奥格生物科技有限公司 Asgr1突变基因及其在制备哺乳动物肝损伤敏感模型中的应用
WO2024046297A1 (zh) * 2022-09-02 2024-03-07 北京福元医药股份有限公司 抑制去唾液酸糖蛋白受体基因表达的siRNA、其缀合物和药物组合物及用途

Family Cites Families (43)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
US5981505A (en) 1993-01-26 1999-11-09 The Trustees Of The University Of Pennsylvania Compositions and methods for delivery of genetic material
US5837533A (en) 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US5840710A (en) 1994-12-09 1998-11-24 Genzyme Corporation Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules
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
US5981501A (en) 1995-06-07 1999-11-09 Inex Pharmaceuticals Corp. Methods for encapsulating plasmids in lipid bilayers
AU723163B2 (en) 1995-06-07 2000-08-17 Tekmira Pharmaceuticals Corporation Lipid-nucleic acid particles prepared via a hydrophobic lipid-nucleic acid complex intermediate and use for gene transfer
US6217900B1 (en) 1997-04-30 2001-04-17 American Home Products Corporation Vesicular complexes and methods of making and using the same
DE69834038D1 (de) 1997-07-01 2006-05-18 Isis Pharmaceutical Inc Zusammensetzungen und verfahren zur verabreichung von oligonukleotiden über die speiseröhre
ATE237312T1 (de) 1998-07-20 2003-05-15 Protiva Biotherapeutics Inc In liposomen verkapselte nukleinsäurekomplexe
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
US7491805B2 (en) 2001-05-18 2009-02-17 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US6693187B1 (en) 2000-10-17 2004-02-17 Lievre Cornu Llc Phosphinoamidite carboxlates and analogs thereof in the synthesis of oligonucleotides having reduced internucleotide charge
US7109165B2 (en) 2001-05-18 2006-09-19 Sirna Therapeutics, Inc. Conjugates and compositions for cellular delivery
US20030130186A1 (en) 2001-07-20 2003-07-10 Chandra Vargeese Conjugates and compositions for cellular delivery
US9181551B2 (en) 2002-02-20 2015-11-10 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
EP1549352A4 (en) 2002-05-06 2005-07-27 Nucleonics Inc METHOD OF DISTRIBUTING NUCLEIC ACIDS
EP2314691A3 (en) * 2002-11-14 2012-01-18 Dharmacon, Inc. Fuctional and hyperfunctional siRNA
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
DK1620544T3 (en) 2003-04-17 2019-01-14 Alnylam Pharmaceuticals Inc MODIFIED iRNA AGENTS
US8017762B2 (en) 2003-04-17 2011-09-13 Alnylam Pharmaceuticals, Inc. Modified iRNA agents
US7723509B2 (en) 2003-04-17 2010-05-25 Alnylam Pharmaceuticals IRNA agents with biocleavable tethers
EP2465534B1 (en) * 2004-06-10 2017-03-29 Omeros Corporation Methods for treating conditions associated with MASP-2 dependent complement activation
CN1300312C (zh) 2004-08-23 2007-02-14 中国人民解放军军事医学科学院放射与辐射医学研究所 靶向去唾液酸糖蛋白受体的抗乙型肝炎病毒反义寡核苷酸的结构和用途
CN101065151B (zh) 2004-09-23 2014-12-10 健泰科生物技术公司 半胱氨酸改造的抗体和偶联物
US8008453B2 (en) 2005-08-12 2011-08-30 Amgen Inc. Modified Fc molecules
JP2007285749A (ja) * 2006-04-13 2007-11-01 Kitasato Gakuen インフルエンザ感染検査薬及び検査方法
KR101133799B1 (ko) * 2006-08-18 2012-04-24 에프. 호프만-라 로슈 아게 폴리뉴클레오타이드의 생체내 전달을 위한 다가접합체
JP4889437B2 (ja) * 2006-10-16 2012-03-07 オリンパス株式会社 微弱光撮像装置
JP2008241447A (ja) * 2007-03-27 2008-10-09 Fujifilm Corp 画像読取装置及び画像読取方法
US8877917B2 (en) 2007-04-23 2014-11-04 Alnylam Pharmaceuticals, Inc. Glycoconjugates of RNA interference agents
JP5265140B2 (ja) * 2007-06-14 2013-08-14 学校法人日本医科大学 卵巣癌の検出方法及び検出用キット
CA2910760C (en) 2007-12-04 2019-07-09 Muthiah Manoharan Targeting lipids
JP2010271078A (ja) * 2009-05-19 2010-12-02 Mcbi:Kk 認知機能障害疾患を含む精神疾患のバイオマーカーおよび該バイオマーカーを用いた認知機能障害疾患を含む精神疾患の検出方法
US20130024961A1 (en) 2009-12-01 2013-01-24 Christopher Burlak Methods of Modulating Thrombocytopenia and Modified Transgenic Pigs
KR101870915B1 (ko) * 2011-04-08 2018-06-25 유니버시티 오브 레스터 Ｍａｓｐ-2 의존적 보체 활성화와 관련된 질병을 치료하는 방법
WO2012177639A2 (en) * 2011-06-22 2012-12-27 Alnylam Pharmaceuticals, Inc. Bioprocessing and bioproduction using avian cell lines
AR090905A1 (es) 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y peptidos y procedimientos para la administracion de oligonucleotidos, composicion farmaceutica
JP6694382B2 (ja) 2013-06-21 2020-05-13 アイオーニス ファーマシューティカルズ， インコーポレーテッドＩｏｎｉｓ Ｐｈａｒｍａｃｅｕｔｉｃａｌｓ，Ｉｎｃ． 標的核酸を調節するための組成物および方法
WO2016170348A2 (en) * 2015-04-22 2016-10-27 Mina Therapeutics Limited Sarna compositions and methods of use
AU2016332900C1 (en) * 2015-09-29 2024-07-04 Amgen Inc. ASGR inhibitors

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US11732266B2 (en) 2023-08-22
JP2019524151A (ja) 2019-09-05
AU2017315952A1 (en) 2019-03-14
MX2024009873A (es) 2024-08-20
US20210108212A1 (en) 2021-04-15
CA3034463A1 (en) 2018-03-01
AR110629A1 (es) 2019-04-17
MX2019002271A (es) 2019-06-20
JP7082610B2 (ja) 2022-06-08
CA3034463C (en) 2025-09-16
UY37376A (es) 2018-03-23
JP2024019733A (ja) 2024-02-09
JP7472180B2 (ja) 2024-04-22
AU2017315952B2 (en) 2023-11-30
JP2022060357A (ja) 2022-04-14
EP3504333A1 (en) 2019-07-03
AU2024201349A1 (en) 2024-04-11
US20190309306A1 (en) 2019-10-10
US10870856B2 (en) 2020-12-22
WO2018039647A1 (en) 2018-03-01

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