JP7033452B2 - 定義されたマルチコンジュゲートオリゴヌクレオチド - Google Patents

定義されたマルチコンジュゲートオリゴヌクレオチド Download PDF

Info

Publication number
JP7033452B2
JP7033452B2 JP2017565893A JP2017565893A JP7033452B2 JP 7033452 B2 JP7033452 B2 JP 7033452B2 JP 2017565893 A JP2017565893 A JP 2017565893A JP 2017565893 A JP2017565893 A JP 2017565893A JP 7033452 B2 JP7033452 B2 JP 7033452B2
Authority
JP
Japan
Prior art keywords
compound
stranded oligonucleotide
thiol
stranded
covalent linker
Prior art date
Legal status (The legal status 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 status listed.)
Active
Application number
JP2017565893A
Other languages
English (en)
Japanese (ja)
Other versions
JP2018518186A (ja
JP2018518186A5 (enExample
Inventor
フィリップ ハドヴィガー
ハンス‐ピーター ヴォルンロシェル
ジョナサン マイルズ ブラウン
ジェームズ エベレット ダールマン
クリスティン ケイ.エイチ. ニューマン
Original Assignee
エムペグ エルエイ リミテッド ライアビリティ カンパニー
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 エムペグ エルエイ リミテッド ライアビリティ カンパニー filed Critical エムペグ エルエイ リミテッド ライアビリティ カンパニー
Publication of JP2018518186A publication Critical patent/JP2018518186A/ja
Publication of JP2018518186A5 publication Critical patent/JP2018518186A5/ja
Application granted granted Critical
Publication of JP7033452B2 publication Critical patent/JP7033452B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Images

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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • A61K47/69Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit
    • A61K47/6905Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion
    • A61K47/6911Medicinal 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 conjugate being characterised by physical or galenical forms, e.g. emulsion, particle, inclusion complex, stent or kit the form being a colloid or an emulsion the form being a liposome
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Synthetic bilayered vehicles, e.g. liposomes or liposomes with cholesterol as the only non-phosphatidyl surfactant
    • 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
    • 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
    • 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
    • C12N15/115Aptamers, i.e. nucleic acids binding a target molecule specifically and with high affinity without hybridising therewith ; Nucleic acids binding to non-nucleic acids, e.g. aptamers
    • 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 nucleic acids [NA]
    • 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
    • C12N2310/315Phosphorothioates
    • 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
    • C12N2310/317Chemical structure of the backbone with an inverted bond, e.g. a cap structure
    • 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
    • C12N2310/346Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • 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
    • C12N2310/351Conjugate
    • 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
    • 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
    • C12N2310/50Physical structure
    • C12N2310/51Physical structure in polymeric form, e.g. multimers, concatemers

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biochemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Dispersion Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Medicinal Preparation (AREA)
JP2017565893A 2015-06-15 2016-06-15 定義されたマルチコンジュゲートオリゴヌクレオチド Active JP7033452B2 (ja)

Applications Claiming Priority (15)

Application Number Priority Date Filing Date Title
US201562175718P 2015-06-15 2015-06-15
US201562175714P 2015-06-15 2015-06-15
US201562175709P 2015-06-15 2015-06-15
US62/175,718 2015-06-15
US62/175,714 2015-06-15
US62/175,709 2015-06-15
US201562203243P 2015-08-10 2015-08-10
US62/203,243 2015-08-10
US201562216318P 2015-09-09 2015-09-09
US201562216317P 2015-09-09 2015-09-09
US201562216314P 2015-09-09 2015-09-09
US62/216,317 2015-09-09
US62/216,314 2015-09-09
US62/216,318 2015-09-09
PCT/US2016/037685 WO2016205410A2 (en) 2015-06-15 2016-06-15 Defined multi-conjugate oligonucleotides

Publications (3)

Publication Number Publication Date
JP2018518186A JP2018518186A (ja) 2018-07-12
JP2018518186A5 JP2018518186A5 (enExample) 2019-08-08
JP7033452B2 true JP7033452B2 (ja) 2022-03-10

Family

ID=57546088

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2017565893A Active JP7033452B2 (ja) 2015-06-15 2016-06-15 定義されたマルチコンジュゲートオリゴヌクレオチド

Country Status (11)

Country Link
US (3) US11352629B2 (enExample)
EP (2) EP4541374A2 (enExample)
JP (1) JP7033452B2 (enExample)
KR (2) KR20250016526A (enExample)
CN (1) CN108026527B (enExample)
AU (3) AU2016280709B2 (enExample)
CA (1) CA2988603A1 (enExample)
HK (1) HK1252970A1 (enExample)
IL (2) IL316159A (enExample)
MX (2) MX2017016088A (enExample)
WO (1) WO2016205410A2 (enExample)

Families Citing this family (32)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR101141544B1 (ko) 2009-03-13 2012-05-03 한국과학기술원 에스아이알엔에이 다중 접합체 및 이의 제조방법
WO2016161388A1 (en) 2015-04-03 2016-10-06 University Of Massachusetts Fully stabilized asymmetric sirna
IL316159A (en) 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
WO2017030973A1 (en) 2015-08-14 2017-02-23 University Of Massachusetts Bioactive conjugates for oligonucleotide delivery
JP7749201B6 (ja) * 2016-01-31 2025-10-21 ユニバーシティー オブ マサチューセッツ 分岐オリゴヌクレオチド
WO2018013525A1 (en) 2016-07-11 2018-01-18 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
WO2018013640A1 (en) 2016-07-15 2018-01-18 University Of Massachusetts Process of delivering small rnas to sperm
CA3033368A1 (en) 2016-08-12 2018-02-15 University Of Massachusetts Conjugated oligonucleotides
SG11201906728TA (en) * 2017-02-06 2019-08-27 Mpeg La Llc Multimeric oligonucleotides having decreased kidney clearance
CN110799647A (zh) 2017-06-23 2020-02-14 马萨诸塞大学 双尾自递送sirna及相关的方法
AU2018330495A1 (en) 2017-09-08 2020-03-26 Mina Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
WO2019140102A1 (en) 2018-01-10 2019-07-18 Translate Bio Ma, Inc. Compositions and methods for facilitating delivery of synthetic nucleic acids to cells
CN111886231B (zh) 2018-04-05 2023-08-01 株式会社Lg化学 基于咔唑的化合物和包含其的有机发光器件
EP3833763A4 (en) 2018-08-10 2023-07-19 University of Massachusetts MODIFIED OLIGONUCLEOTIDES FOR TARGETING SNPS
US11279930B2 (en) 2018-08-23 2022-03-22 University Of Massachusetts O-methyl rich fully stabilized oligonucleotides
AU2019346148A1 (en) * 2018-09-28 2021-05-27 Sirnaomics, Inc. Multi-targeting nucleic acid constructs composed of multiple oligonucleotides that modulate gene expression through complimentary interactions with targets
JP2022523467A (ja) 2019-01-18 2022-04-25 ユニバーシティ・オブ・マサチューセッツ 動的な薬物動態を修飾するアンカー
JP2022523226A (ja) * 2019-03-04 2022-04-21 エムペグ エルエイ リミテッド ライアビリティ カンパニー 増強された生理活性を有する多量体オリゴヌクレオチド
WO2020210207A2 (en) * 2019-04-11 2020-10-15 Northeastern University Oligonucleotide-polymer miktoarm conjugates and methods of use
CA3144467A1 (en) * 2019-07-30 2021-02-04 Jonathan Miles Brown Subcutaneous delivery of multimeric oligonucleotides with enhanced bioactivity
CN114502730A (zh) 2019-08-09 2022-05-13 马萨诸塞大学 经化学修饰的靶向snp的寡核苷酸
US12365894B2 (en) 2019-09-16 2025-07-22 University Of Massachusetts Branched lipid conjugates of siRNA for specific tissue delivery
CN111041025B (zh) 2019-12-17 2021-06-18 深圳市瑞吉生物科技有限公司 基于结合N-乙酰半乳糖胺多肽的mRNA靶向分子及其制备方法
CN112111524B (zh) * 2020-01-10 2024-02-27 深圳瑞吉生物科技有限公司 mRNA-GalNAc靶向分子的制备方法及其体内递送系统和应用
WO2021222635A2 (en) * 2020-04-30 2021-11-04 Mpeg La, L.L.C. Multimeric oligonucleotides with divided strands
CA3178559A1 (en) * 2020-05-19 2021-11-25 Jonathan Miles Brown Orthogonally linked multimeric oligonucleotides
CN111744019B (zh) 2020-07-01 2023-08-04 深圳瑞吉生物科技有限公司 基于甘露糖的mRNA靶向递送系统及其应用
JP2024506042A (ja) * 2021-02-08 2024-02-08 ラクティゲン セラピューティクス 多価オリゴヌクレオチド薬剤およびその使用方法
EP4359539A4 (en) 2021-06-23 2025-05-14 University Of Massachusetts ANTI-FLT1 OLIGONUCLEOTIDE COMPOUNDS OPTIMIZED FOR THE TREATMENT OF PREECLAMPSIA AND OTHER ANGIOGENIC DISORDERS
CN115010794A (zh) * 2022-06-30 2022-09-06 武汉瑞佶生物科技有限公司 蛋白质介导的mRNA靶向分子及其制备方法和应用
WO2024061157A1 (en) * 2022-09-19 2024-03-28 Kylonova (Xiamen) Biopharma Co., Ltd. Carbohydrate-oligonucleotide conjugates, pharmaceutical compositions, and therapeutic applications
WO2024228030A2 (en) 2023-05-04 2024-11-07 Argonaute RNA Limited Dual silencing

Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008109105A2 (en) 2007-03-06 2008-09-12 Flagship Ventures Methods and compositions for improved therapeutic effects with sirna
JP2011518784A (ja) 2008-04-11 2011-06-30 アルニラム ファーマスーティカルズ インコーポレイテッド 標的リガンドをエンドソーム分解性成分と組み合わせることによる核酸の部位特異的送達
US20130064786A1 (en) 2011-09-14 2013-03-14 Korea Advanced Institute Of Science And Technology siRNA HYDROGEL AND METHOD FOR MANUFACTURING THE SAME
US20140309281A1 (en) 2009-03-13 2014-10-16 Kaist Ip Co. Ltd. Multi-conjugate of sirna and preparing method thereof
JP2014527819A (ja) 2011-09-14 2014-10-23 ラナ セラピューティクス インコーポレイテッド 多量体オリゴヌクレオチド化合物

Family Cites Families (48)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5034506A (en) 1985-03-15 1991-07-23 Anti-Gene Development Group Uncharged morpholino-based polymers having achiral intersubunit linkages
US5714331A (en) 1991-05-24 1998-02-03 Buchardt, Deceased; Ole Peptide nucleic acids having enhanced binding affinity, sequence specificity and solubility
US5719262A (en) 1993-11-22 1998-02-17 Buchardt, Deceased; Ole Peptide nucleic acids having amino acid side chains
US5539082A (en) 1993-04-26 1996-07-23 Nielsen; Peter E. Peptide nucleic acids
GB9207381D0 (en) 1992-04-03 1992-05-13 Ici Plc Synthesis of oligonucleotides
US8202846B2 (en) 2000-03-16 2012-06-19 Cold Spring Harbor Laboratory Methods and compositions for RNA interference
AU2001245793A1 (en) 2000-03-16 2001-09-24 Cold Spring Harbor Laboratory Methods and compositions for rna interference
EP2390328A1 (en) 2001-09-28 2011-11-30 Max-Planck-Gesellschaft zur Förderung der Wissenschaften e.V. MicroRNA molecules
WO2004030634A2 (en) 2002-10-02 2004-04-15 Alnylam Pharmaceuticals Inc. Therapeutic compositions
EP3450559A1 (en) 2003-03-07 2019-03-06 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
US8969543B2 (en) 2003-04-03 2015-03-03 Bioneer Corporation SiRNA-hydrophilic polymer conjugates for intracellular delivery of siRNA and method thereof
EP1608735A4 (en) 2003-04-03 2008-11-05 Alnylam Pharmaceuticals RNAI CONJUGATES
CA2521464C (en) 2003-04-09 2013-02-05 Alnylam Pharmaceuticals, Inc. Irna conjugates
KR20060015505A (ko) 2003-04-13 2006-02-17 엔존 파마슈티컬즈, 인코포레이티드 올리고뉴클레오타이드 전구약물
CA2522349A1 (en) 2003-04-17 2004-11-04 Alnylam Pharmaceuticals, Inc. Protected monomers
US7851615B2 (en) 2003-04-17 2010-12-14 Alnylam Pharmaceuticals, Inc. Lipophilic conjugated iRNA agents
ES2702942T3 (es) 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
US20100170000A9 (en) 2004-10-25 2010-07-01 Devgen Nv Rna constructs
EP2314688B1 (en) 2004-11-12 2014-07-16 Asuragen, Inc. Methods and compositions involving miRNA and miRNA inhibitor molecules
KR20120115412A (ko) 2006-04-07 2012-10-17 이데라 파마슈티칼즈, 인코포레이티드 Tlr7 및 tlr8에 대한 안정화된 면역 조절성 rna〔simra〕 화합물
AU2007345648A1 (en) 2007-01-26 2008-08-07 City Of Hope Methods and compositions for the treatment of cancer or other diseases
WO2008109373A1 (en) 2007-03-02 2008-09-12 Mdrna, Inc. Nucleic acid compounds for inhibiting erbb gene expression and uses thereof
CA2692161C (en) 2007-07-09 2015-09-29 Idera Pharmaceuticals, Inc. Stabilized immune modulatory rna (simra) compounds
WO2010021720A1 (en) 2008-08-19 2010-02-25 Nektar Therapeutics Conjugates of small-interfering nucleic acids
US8637194B2 (en) * 2008-09-02 2014-01-28 Bio-Nano Power, Llc Bio-nano power cells and their uses
RU2015151857A (ru) 2008-12-02 2019-01-15 Уэйв Лайф Сайенсес Джапан, Инк. Способ синтеза модифицированных по атому фосфора нуклеиновых кислот
US9745574B2 (en) * 2009-02-04 2017-08-29 Rxi Pharmaceuticals Corporation RNA duplexes with single stranded phosphorothioate nucleotide regions for additional functionality
HRP20150566T1 (hr) 2009-08-27 2015-07-17 Idera Pharmaceuticals, Inc. Kompozicija za inhibiciju ekspresije gena i njegova upotreba
KR101678876B1 (ko) * 2010-01-15 2016-11-23 한국과학기술원 복합 유전자를 표적하는 siRNA 다중 접합체 및 이의 제조방법
ES2562817T3 (es) 2010-02-24 2016-03-08 Arrowhead Research Corporation Composiciones para el suministro dirigido de ARNip
AU2011223820B2 (en) 2010-03-01 2016-01-14 The Children's Hospital Of Philadelphia Nucleic acids for targeting multiple regions of the HCV genome
KR20130132475A (ko) 2010-12-17 2013-12-04 애로우헤드 리서치 코오포레이션 siRNA의 갈락토오스 클러스터-약동학적 조절제 표적 물질
US8691750B2 (en) 2011-05-17 2014-04-08 Axolabs Gmbh Lipids and compositions for intracellular delivery of biologically active compounds
EP3248982A1 (en) 2011-07-19 2017-11-29 Wave Life Sciences Ltd. Thiosulfonate reagents for the synthesis of functionalized nucleic acids
DE18200782T1 (de) 2012-04-02 2021-10-21 Modernatx, Inc. Modifizierte polynukleotide zur herstellung von proteinen im zusammenhang mit erkrankungen beim menschen
FI3597749T3 (fi) 2012-05-25 2023-10-09 Univ California Menetelmiä ja koostumuksia rna-ohjattua kohde-dna-modifikaatiota varten ja rna-ohjattua transkription modulaatiota varten
WO2014043544A1 (en) 2012-09-14 2014-03-20 Rana Therapeutics, Inc. Multimeric oligonucleotide compounds
US8697359B1 (en) 2012-12-12 2014-04-15 The Broad Institute, Inc. CRISPR-Cas systems and methods for altering expression of gene products
KR101629681B1 (ko) * 2013-06-24 2016-06-14 건국대학교 산학협력단 다중 리간드가 도입된 에스아이알엔에이 접합체
EP2845607A1 (en) 2013-09-09 2015-03-11 University of Vienna Antisense oligonucleotides with improved pharmacokinetic properties
KR102344559B1 (ko) 2013-12-12 2021-12-31 알닐람 파마슈티칼스 인코포레이티드 보체 성분 iRNA 조성물 및 이의 이용 방법
CA2935426C (en) 2014-01-30 2023-07-25 F. Hoffmann-La Roche Ag Polyoligomer compound with biocleavable conjugates for reducing or inhibiting expression of a nucleic acid target
IL316159A (en) 2015-06-15 2024-12-01 Mpeg La Llc Defined Oligonucleotide Multimers and Methods for Manufacture Thereof
US20180312839A1 (en) 2015-10-26 2018-11-01 Translate Bio Ma, Inc. Methods and compositions for increasing smn expression
CN117503923A (zh) 2016-01-05 2024-02-06 莱斯特大学 用于抑制有需要的受试者的纤维化的方法
SG11201906728TA (en) 2017-02-06 2019-08-27 Mpeg La Llc Multimeric oligonucleotides having decreased kidney clearance
CN111741967A (zh) 2017-11-30 2020-10-02 深圳市真迈生物科技有限公司 核苷类似物、制备方法及应用
CA3144467A1 (en) 2019-07-30 2021-02-04 Jonathan Miles Brown Subcutaneous delivery of multimeric oligonucleotides with enhanced bioactivity

Patent Citations (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008109105A2 (en) 2007-03-06 2008-09-12 Flagship Ventures Methods and compositions for improved therapeutic effects with sirna
JP2011518784A (ja) 2008-04-11 2011-06-30 アルニラム ファーマスーティカルズ インコーポレイテッド 標的リガンドをエンドソーム分解性成分と組み合わせることによる核酸の部位特異的送達
US20140309281A1 (en) 2009-03-13 2014-10-16 Kaist Ip Co. Ltd. Multi-conjugate of sirna and preparing method thereof
US20130064786A1 (en) 2011-09-14 2013-03-14 Korea Advanced Institute Of Science And Technology siRNA HYDROGEL AND METHOD FOR MANUFACTURING THE SAME
JP2014527819A (ja) 2011-09-14 2014-10-23 ラナ セラピューティクス インコーポレイテッド 多量体オリゴヌクレオチド化合物

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
Journal of Controlled Release, 2006, Vol.116, pp.123-129
Nature Materials, 2010, Vol.9, pp.272-278

Also Published As

Publication number Publication date
EP3307889A4 (en) 2019-06-26
US20240191240A1 (en) 2024-06-13
IL316159A (en) 2024-12-01
AU2016280709B2 (en) 2022-09-15
CN108026527A (zh) 2018-05-11
MX2017016088A (es) 2018-04-11
HK1252970A1 (zh) 2019-06-06
WO2016205410A3 (en) 2017-02-09
JP2018518186A (ja) 2018-07-12
US20190085331A1 (en) 2019-03-21
IL256169B1 (en) 2025-04-01
KR20180039621A (ko) 2018-04-18
MX2024001378A (es) 2024-02-27
AU2024278244A1 (en) 2025-01-02
WO2016205410A2 (en) 2016-12-22
AU2016280709A1 (en) 2018-01-04
AU2022283737A1 (en) 2023-02-02
CA2988603A1 (en) 2016-12-22
IL256169A (en) 2018-03-29
EP3307889A2 (en) 2018-04-18
US11352629B2 (en) 2022-06-07
CN108026527B (zh) 2022-05-10
US20220290148A1 (en) 2022-09-15
IL256169B2 (en) 2025-08-01
KR20250016526A (ko) 2025-02-03
EP4541374A2 (en) 2025-04-23
AU2022283737B2 (en) 2024-09-19
KR102761411B1 (ko) 2025-02-03
US11767531B2 (en) 2023-09-26

Similar Documents

Publication Publication Date Title
AU2022283737B2 (en) Defined multi-conjugate oligonucleotides
JP2018518186A5 (enExample)
US12378551B2 (en) Multimeric oligonucleotides having decreased kidney clearance
CN114466929A (zh) 具有增强的生物活性的多聚体寡核苷酸的皮下递送
WO2021026490A1 (en) Cns targeting with multimeric oligonucleotides
US20230287406A1 (en) Multimeric oligonucleotides with enhanced bioactivity

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20180205

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20181203

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20181204

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190612

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20190612

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20190625

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20200803

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20201102

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20210203

RD04 Notification of resignation of power of attorney

Free format text: JAPANESE INTERMEDIATE CODE: A7424

Effective date: 20210222

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20210705

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20211004

A601 Written request for extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A601

Effective date: 20211203

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20220104

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20220131

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20220228

R150 Certificate of patent or registration of utility model

Ref document number: 7033452

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250