JP2021500862A - 核酸ポリペプチド組成物およびその使用 - Google Patents

核酸ポリペプチド組成物およびその使用 Download PDF

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JP2021500862A
JP2021500862A JP2020517456A JP2020517456A JP2021500862A JP 2021500862 A JP2021500862 A JP 2021500862A JP 2020517456 A JP2020517456 A JP 2020517456A JP 2020517456 A JP2020517456 A JP 2020517456A JP 2021500862 A JP2021500862 A JP 2021500862A
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polynucleotide
molecule
polynucleic acid
oligonucleotide
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JP2020517456A
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JP2021500862A5 (enExample
Inventor
ジョン ギール，アンドリュー
ジョン ギール，アンドリュー
ラマナ ドパラプディ，ヴェンカタ
ラマナ ドパラプディ，ヴェンカタ
ダニエル アリアス，ジョエル
ダニエル アリアス，ジョエル
サイ−ホ チュウ，デイヴィッド
サイ−ホ チュウ，デイヴィッド
カラミアン コクラン，マイケル
カラミアン コクラン，マイケル
バーク，ロブ
コヴァッチ，フィリップ
マレコヴァ，バルボラ
Original Assignee
アビディティー バイオサイエンシーズ，インク．
アビディティー バイオサイエンシーズ，インク．
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Publication of JP2021500862A publication Critical patent/JP2021500862A/ja
Publication of JP2021500862A5 publication Critical patent/JP2021500862A5/ja
Priority to JP2023147876A priority Critical patent/JP7689552B2/ja
Priority to JP2025088275A priority patent/JP2025143247A/ja
Pending legal-status Critical Current

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    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
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    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
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    • A61K47/50—Medicinal 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
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    • A61K47/56—Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule
    • A61K47/59—Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes
    • A61K47/60—Medicinal 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 macromolecular compound, e.g. an oligomeric, polymeric or dendrimeric molecule obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyureas or polyurethanes the organic macromolecular compound being a polyoxyalkylene oligomer, polymer or dendrimer, e.g. PEG, PPG, PEO or polyglycerol
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    • A61K47/6835—Medicinal 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6849—Medicinal 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site the antibody targeting a receptor, a cell surface antigen or a cell surface determinant
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    • A61K47/50—Medicinal 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
    • A61K47/51—Medicinal 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
    • A61K47/68—Medicinal 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment
    • A61K47/6835—Medicinal 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 antibody, an immunoglobulin or a fragment thereof, e.g. an Fc-fragment the modifying agent being an antibody or an immunoglobulin bearing at least one antigen-binding site
    • A61K47/6883—Polymer-drug antibody conjugates, e.g. mitomycin-dextran-Ab; DNA-polylysine-antibody complex or conjugate used for therapy
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • C07—ORGANIC CHEMISTRY
    • C07D—HETEROCYCLIC COMPOUNDS
    • C07D239/00—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings
    • C07D239/02—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings
    • C07D239/24—Heterocyclic compounds containing 1,3-diazine or hydrogenated 1,3-diazine rings not condensed with other rings having three or more double bonds between ring members or between ring members and non-ring members
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    • C07D239/52—Two oxygen atoms
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    • C—CHEMISTRY; METALLURGY
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
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    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H19/00—Compounds containing a hetero ring sharing one ring hetero atom with a saccharide radical; Nucleosides; Mononucleotides; Anhydro-derivatives thereof
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    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K16/00—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies
    • C07K16/18—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans
    • C07K16/28—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants
    • C07K16/2863—Immunoglobulins [IG], e.g. monoclonal or polyclonal antibodies against material from animals or humans against receptors, cell surface antigens or cell surface determinants against receptors for growth factors, growth regulators
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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
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    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol

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  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
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  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
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  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Genetics & Genomics (AREA)
  • Epidemiology (AREA)
  • Biochemistry (AREA)
  • Immunology (AREA)
  • Biotechnology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Cell Biology (AREA)
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  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
JP2020517456A 2017-10-04 2018-10-04 核酸ポリペプチド組成物およびその使用 Pending JP2021500862A (ja)

Priority Applications (2)

Application Number Priority Date Filing Date Title
JP2023147876A JP7689552B2 (ja) 2017-10-04 2023-09-12 核酸ポリペプチド組成物およびその使用
JP2025088275A JP2025143247A (ja) 2017-10-04 2025-05-27 核酸ポリペプチド組成物およびその使用

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762568238P 2017-10-04 2017-10-04
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Families Citing this family (38)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102775461B1 (ko) 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
US11603532B2 (en) 2017-06-02 2023-03-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
CA3065523A1 (en) 2017-06-02 2018-12-06 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
SG11202003126VA (en) 2017-10-04 2020-05-28 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and uses thereof
CN118638787A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
EP3735252A4 (en) * 2018-01-04 2021-10-06 Avidity Biosciences, Inc. HETERODUPLEX NUCLEIC ACID MOLECULES AND USES THEREOF
JP2021521140A (ja) 2018-04-12 2021-08-26 ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． オリゴヌクレオチド組成物及びその使用方法
CA3098624A1 (en) 2018-05-11 2019-11-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods of use thereof
US11168141B2 (en) 2018-08-02 2021-11-09 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2020028832A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
EP3830259A4 (en) 2018-08-02 2022-05-04 Dyne Therapeutics, Inc. MUSCLE TARGETED COMPLEXES AND THEIR USES IN THE TREATMENT OF FACIOSCAPULOHUMERAL MUSCULAR DYSTROPHY
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
IL312067B2 (en) 2018-12-21 2025-08-01 Avidity Biosciences Inc Anti-transferrin receptor antibodies and uses thereof
WO2020247782A1 (en) * 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
JP7581252B2 (ja) * 2019-06-06 2024-11-12 アビディティー バイオサイエンシーズ，インク． Ｕｎａアミダイトおよびその使用
WO2021154477A1 (en) 2020-01-31 2021-08-05 Dyne Therapeutics, Inc. Anti-transferrin receptor (tfr) antibody and uses thereof
TW202144572A (zh) 2020-03-19 2021-12-01 美商亞維代堤生物科學公司 治療臉肩胛肱骨肌肉失養症之組合物及方法
KR102927945B1 (ko) 2020-03-27 2026-02-23 어비디티 바이오사이언시스 인크. 근이영양증의 치료용 조성물 및 방법
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11672872B2 (en) 2021-07-09 2023-06-13 Dyne Therapeutics, Inc. Anti-transferrin receptor antibody and uses thereof
US11771776B2 (en) 2021-07-09 2023-10-03 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating dystrophinopathies
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
CN111996193B (zh) * 2020-09-11 2024-02-20 北京键凯科技股份有限公司 一种有效抑制表皮生长因子受体表达的siRNA序列
WO2023283623A1 (en) 2021-07-09 2023-01-12 Dyne Therapeutics, Inc. Muscle targeting complexes and formulations for treating dystrophinopathies
EP4401792A4 (en) 2021-09-16 2025-12-03 Avidity Biosciences Inc COMPOSITIONS AND METHODS OF TREATMENT OF FACIO-SCAPULO-HUMERAL MUSCULAR DYSTROPHY
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
US12491257B2 (en) 2023-06-27 2025-12-09 Avidity Biosciences, Inc. Compositions and methods of using PRKAG2-targeting antibody-oligonucleotide conjugates
WO2025006955A2 (en) * 2023-06-30 2025-01-02 Ionis Pharmaceuticals, Inc. Cd29 targeted oligonucleotides and uses thereof
AU2024308906A1 (en) 2023-06-30 2026-02-05 Atrium Therapeutics, Inc. Compositions and methods of using pln-targeting antibody-oligonucleotide conjugates
CN117122578B (zh) * 2023-08-28 2025-12-26 中国人民解放军空军军医大学 一种负载jph203的改性聚合物纳米粒、其制备方法和应用
CN117820389A (zh) * 2023-12-22 2024-04-05 康羽生命科学技术(苏州)有限公司 5'-O-DMTr-2'-O-MOE-5-甲基尿苷的制备方法
WO2025191337A2 (en) * 2024-03-11 2025-09-18 Synerk Biotech Limited Oligonucleotide terminal phosphate derivatives and method of use
WO2026048956A1 (ja) * 2024-08-29 2026-03-05 住友化学株式会社 核酸オリゴマーの製造方法
TW202619721A (zh) * 2024-09-18 2026-05-16 美商美國禮來大藥廠 製備用於rna合成之亞磷醯胺化物之改良化學方法

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070004665A1 (en) * 2002-02-20 2007-01-04 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
JP2013532122A (ja) * 2010-04-28 2013-08-15 アイシス ファーマシューティカルズ， インコーポレーテッド 修飾ヌクレオシド、その類似体、およびこれらから調製されるオリゴマー化合物
JP2015536927A (ja) * 2012-10-26 2015-12-24 エヌライフ、セラピューティックス、ソシエダッド、リミターダＮｌｉｆｅ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｓ．Ｌ． 特定のニューロン種へのオリゴヌクレオチド分子の選択的送達によるパーキンソン病の治療のための組成物および方法
US20170035796A1 (en) * 2015-08-07 2017-02-09 Arrowhead Pharmaceuticals, Inc. RNAi Therapy for Hepatitis B Virus Infection
JP2017525705A (ja) * 2014-08-20 2017-09-07 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． 修飾二本鎖ｒｎａ剤

Family Cites Families (86)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4694778A (en) 1984-05-04 1987-09-22 Anicon, Inc. Chemical vapor deposition wafer boat
JP2528107B2 (ja) 1985-03-15 1996-08-28 サマ−トン，ジエ−ムス ポリヌクレオチド測定試薬と方法
US5185444A (en) 1985-03-15 1993-02-09 Anti-Gene Deveopment Group Uncharged morpolino-based polymers having phosphorous containing chiral intersubunit linkages
AU649074B2 (en) 1990-10-12 1994-05-12 Max-Planck-Gesellschaft Zur Forderung Der Wissenschaften E.V. Modified ribozymes
EP0531597A1 (en) 1991-09-12 1993-03-17 Merrell Dow Pharmaceuticals Inc. Novel unsaturated acyclic phosphonate derivatives of purine and pyrimidine
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
CA2192203A1 (en) 1994-06-17 1995-12-28 Werner Hofheinz N,n'-bis(quinolin-4-yl)-diamine derivatives, their preparation and their use as antimalarials
US5889136A (en) 1995-06-09 1999-03-30 The Regents Of The University Of Colorado Orthoester protecting groups in RNA synthesis
CA2249195A1 (en) 1996-03-18 1997-09-25 Board Of Regents, The University Of Texas System Immunoglobin-like domains with increased half lives
US5849902A (en) 1996-09-26 1998-12-15 Oligos Etc. Inc. Three component chimeric antisense oligonucleotides
US6111086A (en) 1998-02-27 2000-08-29 Scaringe; Stephen A. Orthoester protecting groups
CA2323757C (en) 1998-04-02 2011-08-02 Genentech, Inc. Antibody variants and fragments thereof
GB9816575D0 (en) 1998-07-31 1998-09-30 Zeneca Ltd Novel compounds
CA2328356A1 (en) 1999-12-22 2001-06-22 Itty Atcravi Recreational vehicles
US6884869B2 (en) 2001-04-30 2005-04-26 Seattle Genetics, Inc. Pentapeptide compounds and uses related thereto
JP2005504020A (ja) 2001-07-03 2005-02-10 アイシス・ファーマシューティカルス・インコーポレーテッド ヌクレアーゼ耐性キメラオリゴヌクレオチド
JP2005508634A (ja) 2001-10-29 2005-04-07 マクギル・ユニヴァーシティ 非環式リンカー含有オリゴヌクレオチド及びその用途
US20040012626A1 (en) * 2002-07-22 2004-01-22 Brookins Timothy J. Method for creating configurable and customizable web user interfaces
US20040023220A1 (en) 2002-07-23 2004-02-05 Lawrence Greenfield Integrated method for PCR cleanup and oligonucleotide removal
ES2605443T3 (es) 2003-11-06 2017-03-14 Seattle Genetics, Inc. Conjugados de auristatina con anticuerpos anti-HER2 o anti-CD22 y su uso en terapia
JP2008519863A (ja) 2004-11-12 2008-06-12 シアトル ジェネティクス インコーポレイティッド Ｎ末端にアミノ安息香酸単位を有するオーリスタチン
LT2735568T (lt) 2006-05-10 2017-11-27 Sarepta Therapeutics, Inc. Oligonukleotido analogai, turintys katijonines jungtis tarp subvienetų
BRPI0714381A2 (pt) 2006-07-10 2013-03-05 Biogen Idec Inc composiÇÕes e mÉtodos para inibir crescimento de cÂnceres deficientes em smad4
NZ580712A (en) 2007-05-22 2011-12-22 Marina Biotech Inc Hydroxymethyl substituted rna oligonucleotides and rna complexes containing acyclic monomers
EP2220102B1 (en) 2007-11-06 2014-04-16 Clinical Micro Sensors, Inc. Baseless nucleotide analogues and uses thereof
US20110065774A1 (en) 2008-01-31 2011-03-17 Alnylam Pharmaceuticals Chemically modified oligonucleotides and uses thereof
WO2009117531A1 (en) 2008-03-18 2009-09-24 Seattle Genetics, Inc. Auristatin drug linker conjugates
US8575123B2 (en) 2008-04-11 2013-11-05 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
TWI465247B (zh) 2008-04-11 2014-12-21 Catalyst Biosciences Inc 經修飾的因子ｖｉｉ多肽和其用途
SI2607484T1 (sl) 2008-10-27 2016-11-30 Biomarin Technologies B.V. Metode in sredstva za učinkovito preskakovanje eksona 45 v pre-mRNA Duchennove mišične distrofije
CN102459595A (zh) 2009-04-24 2012-05-16 普罗森那技术公司 用于治疗dmd的包含肌苷的寡核苷酸
WO2010129666A1 (en) 2009-05-05 2010-11-11 Altermune Technologies, Llc Chemically programmable immunity
US8927513B2 (en) 2009-07-07 2015-01-06 Alnylam Pharmaceuticals, Inc. 5′ phosphate mimics
MX2012004029A (es) 2009-10-07 2012-05-08 Macrogenics Inc Polipeptidos que contienen la region fc que exhiben funcion efectora mejorada debido a alteraciones del grado de fucosilacion, y metodos para su uso.
CN103068405A (zh) 2010-04-15 2013-04-24 西雅图基因公司 靶向吡咯并苯并二氮杂卓结合物
MX368648B (es) 2010-04-15 2019-10-09 Seattle Genetics Inc Pirrolobenzodiazepinas usadas para tratar enfermedades proliferativas.
US9725479B2 (en) * 2010-04-22 2017-08-08 Ionis Pharmaceuticals, Inc. 5′-end derivatives
WO2011139699A2 (en) 2010-04-28 2011-11-10 Isis Pharmaceuticals, Inc. 5' modified nucleosides and oligomeric compounds prepared therefrom
KR101981705B1 (ko) 2010-05-28 2019-05-24 사렙타 쎄러퓨틱스, 인코퍼레이티드 변형된 서브유니트간 결합 및/또는 말단 그룹을 갖는 올리고뉴클레오타이드 유사체
JP2013531981A (ja) 2010-06-11 2013-08-15 アンチセンス・ファーマ・ゲーエムベーハー 選択的オリゴヌクレオチド修飾の方法
WO2012031243A2 (en) 2010-09-03 2012-03-08 Avi Biopharma, Inc. dsRNA MOLECULES COMPRISING OLIGONUCLEOTIDE ANALOGS HAVING MODIFIED INTERSUBUNIT LINKAGES AND/OR TERMINAL GROUPS
US8501930B2 (en) 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
CA2839593A1 (en) 2011-07-15 2013-01-24 Sarepta Therapeutics, Inc. Methods and compositions for manipulating translation of protein isoforms from alternative initiation start sites
CN107365339A (zh) 2011-07-19 2017-11-21 波涛生命科学有限公司 合成官能化核酸的方法
CA2850373C (en) 2011-10-14 2019-07-16 Seattle Genetics, Inc. Pyrrolobenzodiazepines and targeted conjugates
US10010623B2 (en) 2012-02-16 2018-07-03 Ucl Business Plc Lysosome-cleavable linker
US9610362B2 (en) 2012-03-16 2017-04-04 Valerion Therapeutics, Llc Antisense conjugates for decreasing expression of DMPK
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
CN104640572B (zh) 2012-05-15 2018-04-27 索伦托医疗有限公司 药物偶联物，偶联方法，及其用途
US9504756B2 (en) 2012-05-15 2016-11-29 Seattle Genetics, Inc. Self-stabilizing linker conjugates
DK2872147T3 (da) 2012-07-13 2023-02-20 Wave Life Sciences Ltd Fremgangsmåde til fremstilling af kirale oligonukleotider
DK2839860T3 (da) 2012-10-12 2019-06-17 Medimmune Ltd Pyrrolobenzodiazepiner og konjugater deraf
EP2915815B1 (en) 2012-10-31 2018-01-17 Takeda Pharmaceutical Company Limited New modified nucleic acid
US10131682B2 (en) 2012-11-24 2018-11-20 Hangzhou Dac Biotech Co., Ltd. Hydrophilic linkers and their uses for conjugation of drugs to a cell binding molecules
US9243022B2 (en) 2012-12-21 2016-01-26 Alios Biopharma, Inc. Substituted nucleosides, nucleotides and analogs thereof
KR20210132742A (ko) 2012-12-21 2021-11-04 알트루바이오 인코퍼레이티드 친수성 자기-희생 링커 및 그것의 포합체
US9481905B2 (en) 2013-02-19 2016-11-01 Orizhan Bioscience Limited Method of using neutrilized DNA (N-DNA) as surface probe for high throughput detection platform
EP3964237B1 (en) 2013-03-15 2026-02-18 Regeneron Pharmaceuticals, Inc. Biologically active molecules, conjugates thereof, and therapeutic uses
EP2777714A1 (en) 2013-03-15 2014-09-17 NBE-Therapeutics LLC Method of producing an immunoligand/payload conjugate by means of a sequence-specific transpeptidase enzyme
US9295731B2 (en) 2013-04-01 2016-03-29 Mark Quang Nguyen Cleavable drug conjugates, compositions thereof and methods of use
GB2529356B (en) 2013-04-28 2020-12-23 Genequantum Healthcare Co Ltd Novel linkers, coupling intermediates, conjugates, preparation method and application thereof
US11229711B2 (en) 2013-06-06 2022-01-25 Magenta Therapeutics, Inc. Linkers for antibody-drug conjugates and related compounds, compositions, and methods of use
WO2015038426A1 (en) 2013-09-13 2015-03-19 Asana Biosciences, Llc Self-immolative linkers containing mandelic acid derivatives, drug-ligand conjugates for targeted therapies and uses thereof
AU2014337317A1 (en) 2013-10-15 2016-09-15 Sorrento Therapeutics Inc. Drug-conjugates with a targeting molecule and two different drugs
PT3057585T (pt) 2013-10-15 2020-10-21 Seagen Inc Fármaco-ligadores peguilados para farmacocinética melhorada de conjugados de fármaco-ligando
WO2015069587A2 (en) 2013-11-06 2015-05-14 Merck Sharp & Dohme Corp. Peptide containing conjugates for dual molecular delivery of oligonucleotides
EP3094728B1 (en) 2014-01-16 2022-03-09 Wave Life Sciences Ltd. Chiral design
EP3137606A4 (en) 2014-05-01 2018-04-25 Larry J. Smith METHODS AND MODIFICATIONS THAT PRODUCE ssRNAi COMPOUNDS WITH ENHANCED ACTIVITY, POTENCY AND DURATION OF EFFECT
RS60762B1 (sr) 2015-04-03 2020-10-30 Univ Massachusetts Oligonukleotidna jedinjenja za ciljane irnk hantingtina
KR102644053B1 (ko) 2015-05-19 2024-03-06 사렙타 쎄러퓨틱스 인코퍼레이티드 펩티드 올리고뉴클레오티드 콘주게이트
JOP20160211B1 (ar) 2015-10-01 2021-08-17 Arrowhead Pharmaceuticals Inc تراكيب وأساليب لتثبيط تعبير جيني للـ lpa
WO2017148880A1 (en) 2016-03-01 2017-09-08 F. Hoffmann-La Roche Ag Obinutuzumab variants having altered cell death induction
US10858656B2 (en) 2016-04-01 2020-12-08 Avidity Biosciences, Inc. KRAS nucleic acids and uses thereof
KR102775461B1 (ko) * 2016-04-01 2025-02-28 어비디티 바이오사이언시스 인크. 핵산-폴리펩타이드 조성물 및 이의 용도
TWI912633B (zh) 2016-06-06 2026-01-21 美商愛羅海德製藥公司 5'-環-磷酸修飾之核苷酸
DK3473270T5 (da) * 2016-06-20 2024-09-09 Genahead Bio Inc Konjugat af antistof-lægemiddel
WO2018045317A1 (en) 2016-09-02 2018-03-08 Dicerna Pharmaceuticals, Inc. 4'-phosphate analogs and oligonucleotides comprising the same
KR20240172236A (ko) 2017-01-06 2024-12-09 어비디티 바이오사이언시스 인크. 핵산-폴리펩티드 조성물 및 엑손 스키핑을 유도하는 방법
EP3643706B1 (en) 2017-06-16 2023-11-15 Eisai R&D Management Co., Ltd. Modified nucleic acid monomers and oligonucleic acid analogues, with high biological stability and target gene silencing activity, for use in therapy and diagnosis of cancer and viral diseases
SG11202003126VA (en) 2017-10-04 2020-05-28 Avidity Biosciences Inc Nucleic acid-polypeptide compositions and uses thereof
CN118638787A (zh) 2017-12-06 2024-09-13 艾维迪提生物科学公司 治疗肌萎缩和强直性肌营养不良的组合物和方法
EP3735252A4 (en) 2018-01-04 2021-10-06 Avidity Biosciences, Inc. HETERODUPLEX NUCLEIC ACID MOLECULES AND USES THEREOF
CN109020990A (zh) 2018-09-20 2018-12-18 广西壮族自治区药用植物园 苯基苯并呋喃化合物、及其制备方法、组合物和医药用途
JP7581252B2 (ja) 2019-06-06 2024-11-12 アビディティー バイオサイエンシーズ，インク． Ｕｎａアミダイトおよびその使用
WO2020247782A1 (en) 2019-06-06 2020-12-10 Avidity Biosciences, Inc. Nucleic acid-polypeptide compositions and uses thereof
WO2021081495A1 (en) 2019-10-25 2021-04-29 The Johns Hopkins University Polymeric nanoparticles for intracellular protein delivery

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20070004665A1 (en) * 2002-02-20 2007-01-04 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
JP2013532122A (ja) * 2010-04-28 2013-08-15 アイシス ファーマシューティカルズ， インコーポレーテッド 修飾ヌクレオシド、その類似体、およびこれらから調製されるオリゴマー化合物
JP2015536927A (ja) * 2012-10-26 2015-12-24 エヌライフ、セラピューティックス、ソシエダッド、リミターダＮｌｉｆｅ Ｔｈｅｒａｐｅｕｔｉｃｓ， Ｓ．Ｌ． 特定のニューロン種へのオリゴヌクレオチド分子の選択的送達によるパーキンソン病の治療のための組成物および方法
JP2017525705A (ja) * 2014-08-20 2017-09-07 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． 修飾二本鎖ｒｎａ剤
US20170035796A1 (en) * 2015-08-07 2017-02-09 Arrowhead Pharmaceuticals, Inc. RNAi Therapy for Hepatitis B Virus Infection

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AU2018346507A1 (en) 2020-04-23
US20220395580A1 (en) 2022-12-15
JP2024001021A (ja) 2024-01-09
MX2026005875A (es) 2026-06-01
KR20250096867A (ko) 2025-06-27
US11110180B2 (en) 2021-09-07
CA3077213A1 (en) 2019-04-11
US20250152726A1 (en) 2025-05-15
CN118267396A (zh) 2024-07-02
WO2019071028A1 (en) 2019-04-11
US20190192681A1 (en) 2019-06-27
US11364302B1 (en) 2022-06-21
KR20200060496A (ko) 2020-05-29
MX2020003596A (es) 2020-07-22
US12364769B2 (en) 2025-07-22
JP7689552B2 (ja) 2025-06-06
AU2025203731A1 (en) 2025-06-12
SG11202003126VA (en) 2020-05-28
MA50753A (fr) 2020-08-12
CN111655268B (zh) 2024-03-26
AU2018346507B2 (en) 2025-03-13
EP3691657A1 (en) 2020-08-12
KR102822647B1 (ko) 2025-06-19

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