AU2014287002A1 - Oligonucleotide-ligand conjugates and process for their preparation - Google Patents

Oligonucleotide-ligand conjugates and process for their preparation Download PDF

Info

Publication number
AU2014287002A1
AU2014287002A1 AU2014287002A AU2014287002A AU2014287002A1 AU 2014287002 A1 AU2014287002 A1 AU 2014287002A1 AU 2014287002 A AU2014287002 A AU 2014287002A AU 2014287002 A AU2014287002 A AU 2014287002A AU 2014287002 A1 AU2014287002 A1 AU 2014287002A1
Authority
AU
Australia
Prior art keywords
aco
oligonucleotide
oac
achn
nucleotide
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.)
Abandoned
Application number
AU2014287002A
Other languages
English (en)
Inventor
Muthusamy Jayaraman
Pachamuthu Kandasamy
Alexander V. Kelin
Muthiah Manoharan
Shigeo Matsuda
Jayaprakash K. Nair
Kallanthottathil G. Rajeev
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Alnylam Pharmaceuticals Inc
Original Assignee
Alnylam Pharmaceuticals Inc
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 Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of AU2014287002A1 publication Critical patent/AU2014287002A1/en
Priority to AU2020202093A priority Critical patent/AU2020202093B2/en
Priority to AU2022204575A priority patent/AU2022204575B2/en
Priority to AU2024219618A priority patent/AU2024219618A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • 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
    • 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
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • 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
    • C12N15/111General methods applicable to biologically active non-coding nucleic acids
    • 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
    • C12N2310/35Nature 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/32Special delivery means, e.g. tissue-specific
    • 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
    • C12N2330/00Production
    • C12N2330/30Production chemically synthesised

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Saccharide Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
AU2014287002A 2013-07-11 2014-07-11 Oligonucleotide-ligand conjugates and process for their preparation Abandoned AU2014287002A1 (en)

Priority Applications (3)

Application Number Priority Date Filing Date Title
AU2020202093A AU2020202093B2 (en) 2013-07-11 2020-03-24 Oligonucleotide-ligand conjugates and process for their preparation
AU2022204575A AU2022204575B2 (en) 2013-07-11 2022-06-28 Oligonucleotide-ligand conjugates and process for their preparation
AU2024219618A AU2024219618A1 (en) 2013-07-11 2024-09-11 Oligonucleotide-ligand conjugates and process for their preparation

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361845279P 2013-07-11 2013-07-11
US61/845,279 2013-07-11
PCT/US2014/046425 WO2015006740A2 (en) 2013-07-11 2014-07-11 Oligonucleotide-ligand conjugates and process for their preparation

Related Child Applications (1)

Application Number Title Priority Date Filing Date
AU2020202093A Division AU2020202093B2 (en) 2013-07-11 2020-03-24 Oligonucleotide-ligand conjugates and process for their preparation

Publications (1)

Publication Number Publication Date
AU2014287002A1 true AU2014287002A1 (en) 2016-02-11

Family

ID=51230239

Family Applications (4)

Application Number Title Priority Date Filing Date
AU2014287002A Abandoned AU2014287002A1 (en) 2013-07-11 2014-07-11 Oligonucleotide-ligand conjugates and process for their preparation
AU2020202093A Active AU2020202093B2 (en) 2013-07-11 2020-03-24 Oligonucleotide-ligand conjugates and process for their preparation
AU2022204575A Active AU2022204575B2 (en) 2013-07-11 2022-06-28 Oligonucleotide-ligand conjugates and process for their preparation
AU2024219618A Pending AU2024219618A1 (en) 2013-07-11 2024-09-11 Oligonucleotide-ligand conjugates and process for their preparation

Family Applications After (3)

Application Number Title Priority Date Filing Date
AU2020202093A Active AU2020202093B2 (en) 2013-07-11 2020-03-24 Oligonucleotide-ligand conjugates and process for their preparation
AU2022204575A Active AU2022204575B2 (en) 2013-07-11 2022-06-28 Oligonucleotide-ligand conjugates and process for their preparation
AU2024219618A Pending AU2024219618A1 (en) 2013-07-11 2024-09-11 Oligonucleotide-ligand conjugates and process for their preparation

Country Status (7)

Country Link
US (3) US10808246B2 (enExample)
EP (2) EP4039278A1 (enExample)
JP (4) JP6702862B2 (enExample)
AU (4) AU2014287002A1 (enExample)
CA (2) CA3177846A1 (enExample)
DK (1) DK3019200T3 (enExample)
WO (1) WO2015006740A2 (enExample)

Families Citing this family (120)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102482890B1 (ko) 2013-05-01 2022-12-30 아이오니스 파마수티컬즈, 인코포레이티드 아포지질단백질 (a) 발현을 조절하는 조성물 및 방법
PE20170010A1 (es) * 2014-05-01 2017-03-04 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion del factor b del complemento
WO2015179693A1 (en) 2014-05-22 2015-11-26 Isis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
EP3151839A4 (en) * 2014-06-06 2018-02-28 Ionis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds
EP3865576A1 (en) 2014-12-15 2021-08-18 Dicerna Pharmaceuticals, Inc. Ligand-modified double-stranded nucleic acids
EP3303365A4 (en) * 2015-05-29 2019-04-24 Ascendis Pharma Inc. PRODRUGS WITH A PYROGLUTAMATE LINKER
US20180193471A1 (en) * 2015-07-16 2018-07-12 Kyowa Hakko Kirin Co., Ltd. ß2GPI GENE EXPRESSION-SUPPRESSING NUCLEIC ACID CONJUGATE
CN105524124B (zh) * 2015-12-31 2018-10-30 天津英吉诺科技有限公司 一种n-乙酰氨基半乳糖的绿色合成方法
ES2858090T3 (es) 2016-01-29 2021-09-29 Kyowa Kirin Co Ltd Complejo de ácidos nucleicos
CN109069529B (zh) 2016-03-07 2021-08-20 箭头药业股份有限公司 用于治疗性化合物的靶向配体
EP3442983A1 (en) * 2016-04-14 2019-02-20 H. Hoffnabb-La Roche Ag TRITYL-MONO-Ga1NAc COMPOUNDS AND THEIR USE
SG11201809002RA (en) 2016-04-29 2018-11-29 Univ Nanyang Tech G-quadruplex-containing antisense oligonucleotides
WO2018004004A1 (ja) 2016-06-30 2018-01-04 協和発酵キリン株式会社 核酸複合体
WO2018013525A1 (en) 2016-07-11 2018-01-18 Translate Bio Ma, Inc. Nucleic acid conjugates and uses thereof
US10781175B2 (en) 2016-07-15 2020-09-22 Am Chemicals Llc Solid supports and phosphoramidite building blocks for oligonucleotide conjugates
CN109462981B (zh) 2016-09-02 2023-07-07 箭头药业股份有限公司 靶向配体
CN109661233A (zh) * 2016-10-06 2019-04-19 Ionis 制药公司 缀合低聚化合物的方法
CN110536964A (zh) 2017-03-10 2019-12-03 国立研究开发法人国立成育医疗研究中心 反义寡核苷酸和糖原贮积病Ia型预防或治疗用组合物
AU2018237139A1 (en) * 2017-03-22 2019-10-17 The Regents Of The University Of California Modified oligonucleotides and therapeutic uses thereof
LT3607069T (lt) 2017-04-05 2023-01-10 Silence Therapeutics Gmbh Produktai ir kompozicijos
CN110603330B (zh) 2017-04-28 2023-09-05 协和麒麟株式会社 寡核苷酸衍生物或其盐
WO2019027009A1 (ja) * 2017-08-02 2019-02-07 協和発酵キリン株式会社 核酸複合体
WO2019027015A1 (ja) * 2017-08-02 2019-02-07 協和発酵キリン株式会社 核酸複合体
CN111527207B (zh) * 2017-09-14 2024-04-26 詹森生物制药有限公司 GalNAc衍生物
WO2019105418A1 (zh) 2017-12-01 2019-06-06 苏州瑞博生物技术有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
US11414661B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method therefor and use thereof
JP7365052B2 (ja) * 2017-12-01 2023-10-19 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
KR102834361B1 (ko) * 2017-12-01 2025-07-17 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도
US11414665B2 (en) 2017-12-01 2022-08-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate comprising the same, and preparation method and use thereof
WO2019105403A1 (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
CN111050807B (zh) * 2017-12-01 2024-05-28 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的组合物与缀合物及制备方法和用途
CA3087106A1 (en) 2017-12-29 2019-07-04 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
WO2019140102A1 (en) 2018-01-10 2019-07-18 Translate Bio Ma, Inc. Compositions and methods for facilitating delivery of synthetic nucleic acids to cells
CN115976028B (zh) * 2018-03-09 2025-03-25 第一三共株式会社 糖原病Ia型治疗药
WO2019193189A1 (en) * 2018-04-05 2019-10-10 Silence Therapeutics Gmbh siRNAs WITH AT LEAST TWO LIGANDS AT DIFFERENT ENDS
SG11202010910QA (en) 2018-05-07 2020-12-30 Alnylam Pharmaceuticals Inc Extrahepatic delivery
JP2021533800A (ja) * 2018-08-21 2021-12-09 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Suzhou Ribo Life Science Co., Ltd. 核酸、当該核酸を含む薬物組成物及び複合体ならびにその使用
CN111655297A (zh) * 2018-09-30 2020-09-11 苏州瑞博生物技术有限公司 一种siRNA缀合物及其制备方法和用途
GB2579253A (en) * 2018-11-28 2020-06-17 Pedanius Therapeutics Ltd Antibacterial antisense agents
US20220127605A1 (en) 2018-11-30 2022-04-28 Kyowa Kirin Co., Ltd. Nucleic acid conjugate
JP7507495B2 (ja) * 2018-12-28 2024-06-28 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
GB201821269D0 (en) 2018-12-28 2019-02-13 Nippon Shinyaku Co Ltd Myostatin signal inhibitor
CN111377984B (zh) * 2018-12-29 2024-03-05 苏州瑞博生物技术股份有限公司 化合物和缀合物及其制备方法和用途
CN111377985B (zh) * 2018-12-29 2023-11-10 苏州瑞博生物技术股份有限公司 化合物和缀合物及其制备方法和用途
CN118562796A (zh) * 2019-01-18 2024-08-30 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
EP3974530A4 (en) * 2019-05-22 2023-07-12 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, METHOD FOR PREPARATION AND USE
EP3974529A4 (en) * 2019-05-22 2024-02-07 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, PHARMACEUTICAL COMPOSITION, CONJUGATE, PROCESS OF PREPARATION AND USE
CN118256498A (zh) 2019-05-22 2024-06-28 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN113614230B (zh) * 2019-05-22 2024-04-09 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
CN111973618B (zh) * 2019-05-23 2024-02-02 苏州瑞博生物技术股份有限公司 核酸、药物组合物与siRNA缀合物及制备方法和用途
CN111973619B (zh) * 2019-05-23 2024-01-30 苏州瑞博生物技术股份有限公司 核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途
CN113891939B (zh) 2019-05-24 2024-04-02 苏州瑞博生物技术股份有限公司 核酸、药物组合物与缀合物及制备方法和用途
US20220235359A1 (en) 2019-05-24 2022-07-28 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
US20220186221A1 (en) 2019-05-24 2022-06-16 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition and conjugate, preparation method and use
CN112007040B (zh) * 2019-05-31 2024-01-30 苏州瑞博生物技术股份有限公司 用于治疗乙型病毒性肝炎的联合用药物
TW202122093A (zh) 2019-08-29 2021-06-16 大陸商蘇州瑞博生物技術股份有限公司 化合物、藥物綴合物、試劑盒及其用途
CA3150715A1 (en) 2019-09-10 2021-03-18 Makoto Koizumi Conjugate of galnac-oligonucleotide for delivery to liver and manufacturing method thereof
EP4446336A3 (en) 2019-09-23 2025-06-25 Koranex Capital Neoglycoconjugates as vaccines and therapeutic tools
EP4055166A2 (en) * 2019-11-06 2022-09-14 Alnylam Pharmaceuticals, Inc. Extrahepatic delivery
WO2021108662A1 (en) * 2019-11-26 2021-06-03 Dtx Pharma, Inc. Compound comprising a nucleic acid and a half-life extension motif
CN112876534B (zh) * 2019-11-29 2024-02-09 苏州瑞博生物技术股份有限公司 肝靶向化合物及缀合物
WO2021153047A1 (ja) * 2020-01-29 2021-08-05 住友化学株式会社 核酸オリゴマーの製造方法
CN111269279B (zh) * 2020-02-24 2022-08-05 陕西师范大学 一种在温和条件下制备卤代糖的方法
JP2023520764A (ja) 2020-03-24 2023-05-19 ジェネレーション バイオ カンパニー 第ix因子治療薬を発現するための非ウイルス性dnaベクター及びその使用
MX2022011806A (es) 2020-03-24 2023-01-11 Generation Bio Co Vectores de adn no virales y usos de estos para expresar agentes terapéuticos contra gaucher.
CN113683651A (zh) * 2020-05-19 2021-11-23 上海京新生物医药有限公司 一种GalNAc中间体的制备方法
KR20230016201A (ko) 2020-05-22 2023-02-01 웨이브 라이프 사이언시스 리미티드 이중 가닥 올리고뉴클레오티드 조성물 및 이와 관련된 방법
WO2021249352A1 (zh) 2020-06-10 2021-12-16 正大天晴药业集团股份有限公司 双链siRNA类似物的缀合物
JP2024501288A (ja) 2020-12-23 2024-01-11 フラッグシップ パイオニアリング イノベーションズ シックス,エルエルシー 修飾tremの組成物及びその使用
CN116669746B (zh) 2020-12-29 2025-09-09 苏州瑞博生物技术股份有限公司 一种核酸、含有该核酸的药物组合物与siRNA缀合物及制备方法和用途
WO2022189861A1 (en) 2021-03-08 2022-09-15 Tollys Carbohydrate conjugates of tlr3 ligands and uses thereof
AU2022264509A1 (en) 2021-04-27 2023-12-14 Generation Bio Co. Non-viral dna vectors expressing therapeutic antibodies and uses thereof
EP4329884A1 (en) 2021-04-27 2024-03-06 Generation Bio Co. Non-viral dna vectors expressing anti-coronavirus antibodies and uses thereof
WO2022246195A1 (en) * 2021-05-20 2022-11-24 Olix Us, Inc. Functional moieties and their uses and synthetic preparation
CN115572241B (zh) * 2021-06-21 2024-08-30 润佳(苏州)医药科技有限公司 双价化合物、偶联物及其用途
JPWO2023282344A1 (enExample) 2021-07-08 2023-01-12
IL310001A (en) 2021-07-08 2024-03-01 Nippon Shinyaku Co Ltd Precipitation suppressing agent
EP4368186A4 (en) 2021-07-08 2025-06-25 Nippon Shinyaku Co., Ltd. NEPHROTOXICITY REDUCING AGENT
CA3225015A1 (en) 2021-07-16 2023-01-19 Zicai Liang Nucleic acid, composition and conjugate containing same, and preparation method and use
WO2023284763A1 (zh) 2021-07-16 2023-01-19 苏州瑞博生物技术股份有限公司 双链寡核苷酸、含双链寡核苷酸的组合物与缀合物及制备方法和用途
KR20240070519A (ko) * 2021-08-05 2024-05-21 사네진 바이오 유에스에이 인크. 1'-알킬 개질된 리보스 유도체 및 사용 방법
JP2024534502A (ja) 2021-09-22 2024-09-20 セーンジーン バイオ ユーエスエー インコーポレイティド オリゴヌクレオチドのインビボ送達に使用するための2’-アルキル又は3’-アルキル修飾リボース誘導体
EP4413015A1 (en) 2021-10-05 2024-08-14 Sanegene Bio USA Inc. Polyhydroxylated cyclopentane derivatives and methods of use
WO2023097251A1 (en) * 2021-11-23 2023-06-01 Yale University Oligonucleotide-containing transcription factor targeting chimeras
KR20240169616A (ko) 2022-02-22 2024-12-03 사네진 바이오 유에스에이 인크. 5'-변형된 카르보시클릭 리보뉴클레오티드 유도체 및 사용 방법
CN120129745A (zh) 2022-03-10 2025-06-10 日本新药株式会社 抗病毒反义低聚物
KR20240161965A (ko) 2022-03-14 2024-11-13 제너레이션 바이오 컴퍼니 이종 프라임 부스트 백신 조성물 및 사용 방법
CN118891366A (zh) 2022-03-16 2024-11-01 第一三共株式会社 抑制转铁蛋白受体2的表达的siRNA
CN118871585A (zh) 2022-03-16 2024-10-29 第一三共株式会社 具有RNAi活性的化学-修饰的寡核苷酸
WO2023207615A1 (zh) * 2022-04-26 2023-11-02 南京明德新药研发有限公司 一类含由天然核苷酸组成突出端的双链RNAi化合物
US20250346620A1 (en) * 2022-04-28 2025-11-13 Dicerna Pharmaceuticals, Inc. Methods for synthesis of peracetylgalactosamine-1-pentanoic acid
US20250304957A1 (en) 2022-05-13 2025-10-02 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
EP4544043A1 (en) 2022-06-22 2025-04-30 Flagship Pioneering Innovations VI, LLC Compositions of modified trems and uses thereof
CN119654412A (zh) * 2022-06-27 2025-03-18 中美瑞康核酸技术(南通)研究院有限公司 激活补体因子h表达的寡核苷酸调节剂
JP2025522880A (ja) 2022-06-30 2025-07-17 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 環状ジスルフィド修飾リン酸ベースのオリゴヌクレオチドプロドラッグ
EP4555088A1 (en) 2022-07-11 2025-05-21 Sanegene Bio USA Inc. Optimized 2'- modified ribose derivatives and methods of use
CA3263391A1 (en) 2022-08-05 2024-02-08 Sanegene Bio Usa Inc DOUBLE-STRANDED RNA TARGETING ANGIOTENSINOGEN (AGT) AND ITS METHODS OF USE
WO2024040222A1 (en) 2022-08-19 2024-02-22 Generation Bio Co. Cleavable closed-ended dna (cedna) and methods of use thereof
KR20250088573A (ko) 2022-10-14 2025-06-17 사네진 바이오 유에스에이 인크. C3을 표적화하는 소형 간섭 rna 및 그의 용도
AU2023404700A1 (en) 2022-12-02 2025-07-10 Shanghai Argo Biopharmaceutical Co., Ltd. Bicyclic abasic nucleic acid analogs and oligomeric compounds prepared therefrom
EP4638743A2 (en) 2022-12-19 2025-10-29 Sanegene Bio USA Inc. Small interfering rna targeting cfb and uses thereof
CN116178458A (zh) * 2022-12-26 2023-05-30 成都药明康德新药开发有限公司 一种糖苷化合物的合成
WO2024160204A1 (en) * 2023-01-30 2024-08-08 Primelink Biotherapeutics (Suzhou) Co., Ltd. Polymer-oligonucleotide conjugates
US20240309383A1 (en) 2023-02-24 2024-09-19 Suzhou Sanegene Bio Inc. Small interfering rna targeting hbv and uses thereof
US20240309380A1 (en) 2023-03-03 2024-09-19 Sanegene Bio Usa Inc. Small interfering rna targeting apoc3 and uses thereof
WO2024238385A2 (en) 2023-05-12 2024-11-21 Alnylam Pharmaceuticals, Inc. Single-stranded loop oligonucleotides
WO2024263915A2 (en) 2023-06-21 2024-12-26 Sanegene Bio Usa Inc. Double stranded rna targeting proprotein convertase subtilisin kexin 9 (pcsk9) and methods of use thereof
CN119219716A (zh) * 2023-06-30 2024-12-31 上海维申医药有限公司 新型GalNAc的靶向递送片段及其制备和应用
WO2025016441A1 (zh) * 2023-07-19 2025-01-23 上海维申医药有限公司 新型GalNAc的靶向递送片段及其制备和应用
US20250064939A1 (en) 2023-07-24 2025-02-27 Sanegene Bio Usa Inc. Lipid-based enhancement agent for rna delivery and therapy
WO2025042786A1 (en) 2023-08-18 2025-02-27 Flagship Pioneering Innovations Vi, Llc Compositions comprising circular polyribonucleotides and uses thereof
CN117384226A (zh) * 2023-09-14 2024-01-12 苏州盛诺维生物科技有限公司 一种GalNAc类化合物的合成工艺
WO2025096809A1 (en) 2023-10-31 2025-05-08 Korro Bio, Inc. Oligonucleotides comprising phosphoramidate internucleotide linkages
TW202523695A (zh) 2023-11-22 2025-06-16 美商旗艦先鋒創新有限責任(Vii)公司 用於治療非酒精性脂肪性肝病之方法及組成物
WO2025140299A1 (zh) * 2023-12-26 2025-07-03 广东东阳光药业股份有限公司 一种新型的化合物及其用途
WO2025155911A1 (en) 2024-01-18 2025-07-24 Proqr Therapeutics Ii B.V. Antisense oligonucleotides for the treatment of hurler syndrome
WO2025207934A1 (en) 2024-03-28 2025-10-02 Lycia Therapeutics, Inc. Lysosomal targeting bifunctional molecules for degradation of thyroid stimulating hormone receptor autoantibodies
WO2025228441A2 (en) 2024-04-30 2025-11-06 Sanegene Bio Usa Inc. Small interfering rna targeting inhbe and uses thereof
CN118146284B (zh) * 2024-05-08 2024-07-26 北京悦康科创医药科技股份有限公司 一种GalNAc化合物、其与寡核苷酸缀合物及制备方法
WO2025245188A2 (en) 2024-05-21 2025-11-27 Flagship Pioneering Innovations Vii, Llc Methods of treating liver steatosis and non-alcoholic fatty liver disease

Family Cites Families (42)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5453566A (en) 1986-03-28 1995-09-26 Calgene, Inc. Antisense regulation of gene expression in plant/cells
US4987071A (en) 1986-12-03 1991-01-22 University Patents, Inc. RNA ribozyme polymerases, dephosphorylases, restriction endoribonucleases and methods
CA1340323C (en) 1988-09-20 1999-01-19 Arnold E. Hampel Rna catalyst for cleaving specific rna sequences
GB8822492D0 (en) 1988-09-24 1988-10-26 Considine J Apparatus for removing tumours from hollow organs of body
WO1991003162A1 (en) 1989-08-31 1991-03-21 City Of Hope Chimeric dna-rna catalytic sequences
US6365730B1 (en) 1990-06-19 2002-04-02 Gene Shears Pty. Limited DNA-Armed ribozymes and minizymes
US5789573A (en) 1990-08-14 1998-08-04 Isis Pharmaceuticals, Inc. Antisense inhibition of ICAM-1, E-selectin, and CMV IE1/IE2
DE69123979T2 (de) 1990-10-12 1997-04-30 Max Planck Gesellschaft Abgeänderte ribozyme
DE4216134A1 (de) 1991-06-20 1992-12-24 Europ Lab Molekularbiolog Synthetische katalytische oligonukleotidstrukturen
US5652094A (en) 1992-01-31 1997-07-29 University Of Montreal Nucleozymes
AU687736B2 (en) 1992-05-11 1998-03-05 Ribozyme Pharmaceuticals, Inc. Method and reagent for inhibiting viral replication
EP0786522A2 (en) 1992-07-17 1997-07-30 Ribozyme Pharmaceuticals, Inc. Enzymatic RNA molecules for treatment of stenotic conditions
US5574142A (en) * 1992-12-15 1996-11-12 Microprobe Corporation Peptide linkers for improved oligonucleotide delivery
IL108367A0 (en) 1993-01-27 1994-04-12 Hektoen Inst For Medical Resea Antisense polynzcleotide inhibition of human growth factor-sensitive cancer cells
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5591317A (en) 1994-02-16 1997-01-07 Pitts, Jr.; M. Michael Electrostatic device for water treatment
US5631359A (en) 1994-10-11 1997-05-20 Ribozyme Pharmaceuticals, Inc. Hairpin ribozymes
AU3889595A (en) 1994-10-05 1996-05-02 Amgen, Inc. Method for inhibiting smooth muscle cell proliferation and oligonucleotides for use therein
US5783683A (en) 1995-01-10 1998-07-21 Genta Inc. Antisense oligonucleotides which reduce expression of the FGFRI gene
US5681824A (en) 1995-06-05 1997-10-28 Eisai Co., Ltd. Substituted liposaccharides useful in the treatment and prevention of endotoxemia
US5747470A (en) 1995-06-07 1998-05-05 Gen-Probe Incorporated Method for inhibiting cellular proliferation using antisense oligonucleotides to gp130 mRNA
US5739119A (en) 1996-11-15 1998-04-14 Galli; Rachel L. Antisense oligonucleotides specific for the muscarinic type 2 acetylcholine receptor MRNA
US6300319B1 (en) * 1998-06-16 2001-10-09 Isis Pharmaceuticals, Inc. Targeted oligonucleotide conjugates
EP3450559A1 (en) 2003-03-07 2019-03-06 Alnylam Pharmaceuticals, Inc. Therapeutic compositions
ES2702942T3 (es) 2003-04-17 2019-03-06 Alnylam Pharmaceuticals Inc Agentes de ARNi modificados
AU2006279906B2 (en) 2005-08-10 2012-05-10 Alnylam Pharmaceuticals, Inc. Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US20070213292A1 (en) 2005-08-10 2007-09-13 The Rockefeller University Chemically modified oligonucleotides for use in modulating micro RNA and uses thereof
US8362229B2 (en) * 2006-02-08 2013-01-29 Quark Pharmaceuticals, Inc. Tandem siRNAS
EP3705125B1 (en) * 2007-12-04 2023-07-05 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
AU2009234266B2 (en) * 2008-04-11 2015-08-06 Tekmira Pharmaceuticals Corporation Site-specific delivery of nucleic acids by combining targeting ligands with endosomolytic components
US9198972B2 (en) * 2010-01-28 2015-12-01 Alnylam Pharmaceuticals, Inc. Monomers and oligonucleotides comprising cycloaddition adduct(s)
US8501930B2 (en) * 2010-12-17 2013-08-06 Arrowhead Madison Inc. Peptide-based in vivo siRNA delivery system
EP2594575A1 (en) * 2011-11-15 2013-05-22 Université de Strasbourg Mannosylated compounds useful for the prevention and the treatment of infectious diseases
MY167390A (en) * 2011-11-18 2018-08-16 Alnylam Pharmaceuticals Inc Rnai agents, compositions and methods of use thereof for treating transthyretin (ttr) associated diseases
US8577565B2 (en) 2011-12-16 2013-11-05 GM Global Technology Operations LLC Limiting branch pressure to a solenoid valve in a fluid circuit
AR090906A1 (es) 2012-05-02 2014-12-17 Merck Sharp & Dohme Conjugados que contienen tetragalnac y procedimientos para la administracion de oligonucleotidos
SE536942C2 (sv) * 2012-07-24 2014-11-11 Reiz Ismet Flexibel dragnyckel
DK2879718T3 (da) * 2012-08-06 2023-06-19 Alnylam Pharmaceuticals Inc Proces til fremstilling af kulhydratkonjugerede RNA-stoffer
HUE035887T2 (en) * 2012-12-05 2018-05-28 Alnylam Pharmaceuticals Inc PCSK9 iRNA preparations and methods for their use
KR102482890B1 (ko) * 2013-05-01 2022-12-30 아이오니스 파마수티컬즈, 인코포레이티드 아포지질단백질 (a) 발현을 조절하는 조성물 및 방법
AU2014284152B2 (en) * 2013-06-21 2020-01-23 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
EP3151839A4 (en) * 2014-06-06 2018-02-28 Ionis Pharmaceuticals, Inc. Compositions and methods for enhanced intestinal absorption of conjugated oligomeric compounds

Also Published As

Publication number Publication date
WO2015006740A2 (en) 2015-01-15
US20220348914A1 (en) 2022-11-03
JP7296916B2 (ja) 2023-06-23
AU2020202093B2 (en) 2022-04-07
CA3177846A1 (en) 2015-01-15
WO2015006740A3 (en) 2015-05-07
CA2917161C (en) 2023-12-12
JP6702862B2 (ja) 2020-06-03
JP7676460B2 (ja) 2025-05-14
EP3019200B1 (en) 2022-03-23
EP4039278A1 (en) 2022-08-10
AU2020202093A1 (en) 2020-04-09
US20250075208A1 (en) 2025-03-06
AU2022204575B2 (en) 2024-06-13
AU2022204575A1 (en) 2022-07-21
JP2016529230A (ja) 2016-09-23
DK3019200T3 (da) 2022-06-20
US20160376585A1 (en) 2016-12-29
JP2023073507A (ja) 2023-05-25
US12173288B2 (en) 2024-12-24
CA2917161A1 (en) 2015-01-15
AU2024219618A1 (en) 2024-10-03
JP2025107295A (ja) 2025-07-17
EP3019200A2 (en) 2016-05-18
JP2020114886A (ja) 2020-07-30
US10808246B2 (en) 2020-10-20

Similar Documents

Publication Publication Date Title
AU2022204575B2 (en) Oligonucleotide-ligand conjugates and process for their preparation
CN108699558B (zh) 核酸复合物
CN103068834B (zh) 用于反向合成rna的亚磷酰胺
CN104781271B (zh) 具有生物可逆的基团的多核苷酸
AU2016202354A1 (en) Chemical modifications of monomers and oligonucleotides with cycloaddition
AU2011302152A1 (en) Modified iRNA agents
Taherpour et al. 2, 6-Bis (functionalized) purines as metal-ion-binding surrogate nucleobases that enhance hybridization with unmodified 2′-O-methyl oligoribonucleotides
WO2017010575A1 (ja) β2GPI遺伝子発現抑制核酸複合体
KR20210098477A (ko) 핵산 복합체
Terrazas et al. Synthesis and properties of small interfering RNA duplexes carrying 5-ethyluridine residues
Zhao Chemical synthesis of glycosylated oligoribonucleotides for siRNA development
Terrazas Martínez et al. Synthesis and properties of small interfering RNA duplexes carrying 5-ethyluridine residues
JP2011004682A (ja) オリゴヌクレオチド誘導体及びオリゴヌクレオチド誘導体を用いたオリゴヌクレオチド構築物

Legal Events

Date Code Title Description
MK5 Application lapsed section 142(2)(e) - patent request and compl. specification not accepted