KR20250145702A - 표적화 조성물 - Google Patents

표적화 조성물

Info

Publication number
KR20250145702A
KR20250145702A KR1020257032179A KR20257032179A KR20250145702A KR 20250145702 A KR20250145702 A KR 20250145702A KR 1020257032179 A KR1020257032179 A KR 1020257032179A KR 20257032179 A KR20257032179 A KR 20257032179A KR 20250145702 A KR20250145702 A KR 20250145702A
Authority
KR
South Korea
Prior art keywords
compound
mmol
preparation
solution
seq
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.)
Pending
Application number
KR1020257032179A
Other languages
English (en)
Korean (ko)
Inventor
제임스 헤이즈
리처드 제이. 홀랜드
아담 저지
에이미 씨. 에이치. 리
앨런 디. 마틴
니콜라스 마이클 스니드
에밀리 피. 티
마크 우드
신 예
Original Assignee
아뷰터스 바이오파마 코포레이션
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 아뷰터스 바이오파마 코포레이션 filed Critical 아뷰터스 바이오파마 코포레이션
Publication of KR20250145702A publication Critical patent/KR20250145702A/ko
Pending legal-status Critical Current

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • 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
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—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
    • 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
    • A61K47/54—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 compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—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
    • 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
    • A61K47/54—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 compound
    • A61K47/554—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 compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00—Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12—Antivirals
    • A61P31/20—Antivirals for DNA viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H15/00—Compounds containing hydrocarbon or substituted hydrocarbon radicals directly attached to hetero atoms of saccharide radicals
    • C07H15/02—Acyclic radicals, not substituted by cyclic structures
    • C07H15/04—Acyclic radicals, not substituted by cyclic structures attached to an oxygen atom of the saccharide radical
    • C07H15/08—Polyoxyalkylene derivatives
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • 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
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07J—STEROIDS
    • C07J63/00—Steroids in which the cyclopenta(a)hydrophenanthrene skeleton has been modified by expansion of only one ring by one or two atoms
    • C07J63/008—Expansion of ring D by one atom, e.g. D homo steroids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/1131—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 viruses
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N15/117—Nucleic acids having immunomodulatory properties, e.g. containing CpG-motifs
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/322—2'-R Modification
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/344—Position-specific modifications, e.g. on every purine, at the 3'-end
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oncology (AREA)
  • Communicable Diseases (AREA)
  • Botany (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Immunology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicinal Preparation (AREA)
  • Peptides Or Proteins (AREA)
  • Saccharide Compounds (AREA)
KR1020257032179A 2017-04-11 2018-04-10 표적화 조성물 Pending KR20250145702A (ko)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
US201762484247P 2017-04-11 2017-04-11
US62/484,247 2017-04-11
US201762525071P 2017-06-26 2017-06-26
US62/525,071 2017-06-26
KR1020247011466A KR102866327B1 (ko) 2017-04-11 2018-04-10 표적화 조성물
PCT/US2018/026918 WO2018191278A2 (en) 2017-04-11 2018-04-10 Targeted compositions

Related Parent Applications (1)

Application Number Title Priority Date Filing Date
KR1020247011466A Division KR102866327B1 (ko) 2017-04-11 2018-04-10 표적화 조성물

Publications (1)

Publication Number Publication Date
KR20250145702A true KR20250145702A (ko) 2025-10-13

Family

ID=62063656

Family Applications (3)

Application Number Title Priority Date Filing Date
KR1020257032179A Pending KR20250145702A (ko) 2017-04-11 2018-04-10 표적화 조성물
KR1020247011466A Active KR102866327B1 (ko) 2017-04-11 2018-04-10 표적화 조성물
KR1020197033270A Active KR102656438B1 (ko) 2017-04-11 2018-04-10 표적화 조성물

Family Applications After (2)

Application Number Title Priority Date Filing Date
KR1020247011466A Active KR102866327B1 (ko) 2017-04-11 2018-04-10 표적화 조성물
KR1020197033270A Active KR102656438B1 (ko) 2017-04-11 2018-04-10 표적화 조성물

Country Status (15)

Country Link
US (3) US11427823B2 (enExample)
EP (2) EP3610017A2 (enExample)
JP (3) JP2020516296A (enExample)
KR (3) KR20250145702A (enExample)
CN (2) CN110741087B (enExample)
AU (2) AU2018250553B2 (enExample)
BR (1) BR112019021359A2 (enExample)
CA (1) CA3059426A1 (enExample)
MA (1) MA52546A (enExample)
MX (3) MX2019012280A (enExample)
NZ (1) NZ757927A (enExample)
PH (1) PH12019502333A1 (enExample)
SG (1) SG11201909433XA (enExample)
TW (2) TWI805577B (enExample)
WO (1) WO2018191278A2 (enExample)

Families Citing this family (29)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
MA45478A (fr) * 2016-04-11 2019-02-20 Arbutus Biopharma Corp Compositions de conjugués d'acides nucléiques ciblés
JP2020516296A (ja) 2017-04-11 2020-06-11 アルブータス・バイオファーマー・コーポレイション 標的化組成物
KR102834361B1 (ko) 2017-12-01 2025-07-17 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 이를 포함하는 조성물과 컨쥬게이트, 및 그의 제조 방법과 용도
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
EP3718572B1 (en) 2017-12-01 2024-07-31 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, composition and conjugate containing nucleic acid, preparation method and use
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
AU2018374219C1 (en) 2017-12-01 2023-05-11 Suzhou Ribo Life Science Co., Ltd. Double-stranded oligonucleotide, composition and conjugate comprising double-stranded oligonucleotide, preparation method therefor and use thereof
EP3732185B1 (en) 2017-12-29 2025-02-26 Suzhou Ribo Life Science Co., Ltd. Conjugates and preparation and use thereof
EP3842534A4 (en) 2018-08-21 2022-07-06 Suzhou Ribo Life Science Co., Ltd. NUCLEIC ACID, COMPOSITION AND CONJUGATE CONTAINING NUCLEIC ACID AND METHOD OF USE THEREOF
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
CN121081657A (zh) * 2018-11-02 2025-12-09 阿布特斯生物制药公司 二价靶向缀合物
MX2021005130A (es) * 2018-11-02 2021-11-04 Genevant Sciences Gmbh Metodos terapeuticos.
KR20210110839A (ko) 2018-12-28 2021-09-09 쑤저우 리보 라이프 사이언스 컴퍼니, 리미티드 핵산, 핵산을 함유하는 조성물 및 접합체, 이의 제조 방법 및 용도
MA55375A (fr) * 2019-03-20 2022-01-26 Arbutus Biopharma Corp Méthodes thérapeutiques de traitement de l'hépatite b
CA3139195A1 (en) 2019-05-22 2020-11-26 Suzhou Ribo Life Science Co., Ltd. Nucleic acid, pharmaceutical composition, conjugate, preparation method, and use
CN117701564A (zh) * 2019-05-22 2024-03-15 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
US20220305107A1 (en) 2019-06-18 2022-09-29 Janssen Sciences Ireland Unlimited Company COMBINATION OF HEPATITIS B VIRUS (HBV) VACCINES AND HBV-TARGETING RNAi
CN115884984A (zh) * 2019-12-06 2023-03-31 盖纳万科学有限公司 用于治疗肝纤维化的结合物和方法
JP2023510109A (ja) * 2019-12-20 2023-03-13 アルブータス・バイオファーマー・コーポレイション 合成プロセス及び中間体
CN116472063A (zh) * 2020-11-06 2023-07-21 阿布特斯生物制药公司 包含经修饰的siRNA的靶向性偶联物
CN114853829A (zh) * 2021-02-05 2022-08-05 上海拓界生物医药科技有限公司 一种核酸配体、其缀合物、制备方法及用途
WO2023109935A1 (zh) * 2021-12-16 2023-06-22 上海拓界生物医药科技有限公司 一种dsRNA、其制备方法及应用
CA3249102A1 (en) * 2022-01-20 2025-06-17 Chengdu Xinzhenghe Pharmaceutical Technology Co. Ltd Targeting Ligand and its Use
EP4471140A1 (en) * 2022-01-30 2024-12-04 Rona Bioscience, Limited Targeting ligand containing n-acetylgalactosamine
IL315546A (en) * 2022-03-16 2024-11-01 Empirico Inc GALNAC compositions to improve siRNA bioavailability
WO2024255761A1 (zh) * 2023-06-12 2024-12-19 大睿生物 包含糖的寡核苷酸递送配体
WO2025007274A1 (zh) * 2023-07-04 2025-01-09 成都先导药物开发股份有限公司 一种缀合物及其中间体化合物和用途
WO2025007907A1 (zh) * 2023-07-04 2025-01-09 大睿生物 包含肽的寡核苷酸递送配体
WO2025242321A1 (en) 2024-05-24 2025-11-27 Glaxosmithkline Intellectual Property (No.3) Limited Novel use

Family Cites Families (41)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US4683202A (en) 1985-03-28 1987-07-28 Cetus Corporation Process for amplifying nucleic acid sequences
US4683195A (en) 1986-01-30 1987-07-28 Cetus Corporation Process for amplifying, detecting, and/or-cloning nucleic acid sequences
US5708163A (en) 1994-03-15 1998-01-13 Sloan-Kettering Institute Of Cancer Research Synthesis of the breast tumor-associated antigen defined by monoclonalantibody MBRL and uses thereof
US6303120B1 (en) 1994-03-15 2001-10-16 Memorial Sloan-Kettering Institute For Cancer Research Synthesis of glycoconjugates of the lewis y epitope and uses thereof
US5885613A (en) 1994-09-30 1999-03-23 The University Of British Columbia Bilayer stabilizing components and their use in forming programmable fusogenic liposomes
EP1096921B1 (en) 1998-07-20 2003-04-16 Protiva Biotherapeutics Inc. Liposomal encapsulated nucleic acid-complexes
US7833992B2 (en) 2001-05-18 2010-11-16 Merck Sharpe & Dohme Conjugates and compositions for cellular delivery
WO2003070918A2 (en) 2002-02-20 2003-08-28 Ribozyme Pharmaceuticals, Incorporated Rna interference by modified short interfering nucleic acid
US9657294B2 (en) 2002-02-20 2017-05-23 Sirna Therapeutics, Inc. RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (siNA)
US8258288B2 (en) 2002-02-20 2012-09-04 Sirna Therapeutics, Inc. RNA interference mediated inhibition of respiratory syncytial virus (RSV) expression using short interfering nucleic acid (siNA)
ATE485031T1 (de) 2002-06-28 2010-11-15 Protiva Biotherapeutics Inc Verfahren und vorrichtung zur herstellung von liposomen
US20080207539A1 (en) * 2003-09-01 2008-08-28 Patrick Arbuthnot Self-Processing Rna Expression Cassette
WO2005026165A1 (en) 2003-09-12 2005-03-24 Warner-Lambert Company Llc Quinolone antibacterial agents
EP1937213B1 (en) 2005-07-27 2017-10-25 Protiva Biotherapeutics Inc. Systems and methods for manufacturing liposomes
US8956825B2 (en) 2007-05-24 2015-02-17 The United States Of America As Represented By The Department Of Veterans Affairs Intranuclear protein transduction through a nucleoside salvage pathway
EP4074344A1 (en) 2007-12-04 2022-10-19 Arbutus Biopharma Corporation Targeting lipids
CA2708171C (en) 2007-12-04 2018-02-27 Alnylam Pharmaceuticals, Inc. Folate conjugates
WO2009143390A2 (en) 2008-05-22 2009-11-26 Isis Pharmaceuticals, Inc. Methods for modulating expression of rbp4
CN101603042B (zh) * 2008-06-13 2013-05-01 厦门大学 可用于乙型肝炎病毒感染治疗的rna干扰靶点
KR101647334B1 (ko) 2008-07-10 2016-08-10 세리나 쎄라퓨틱스, 인코포레이티드 불활성 말단기를 가진 폴리옥사졸린, 보호된 개시기로부터 제조된 폴리옥사졸린 및 그와 관련된 화합물
EP2496238A4 (en) 2009-11-03 2013-10-02 Alnylam Pharmaceuticals Inc FORMULATED LIPID COMPOSITIONS AND METHODS FOR INHIBITING TRANSTHYRETIN EXPRESSION (TTR)
NZ601737A (en) 2010-02-24 2013-06-28 Arrowhead Res Corp Compositions for targeted delivery of sirna
EP3192800A1 (en) 2010-12-17 2017-07-19 Arrowhead Pharmaceuticals, Inc. Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
WO2013033230A1 (en) 2011-08-29 2013-03-07 Isis Pharmaceuticals, Inc. Oligomer-conjugate complexes and their use
DK3366775T4 (da) 2011-11-18 2025-10-27 Alnylam Pharmaceuticals Inc Modificerede rnai-midler
PE20142362A1 (es) * 2011-11-18 2015-01-30 Alnylam Pharmaceuticals Inc Agentes de iarn, composiciones y metodos de uso de los mismos para tratar enfermedades asociadas con transtiretina (ttr)
RU2015119411A (ru) 2012-11-15 2017-01-10 Рош Инновейшен Сентер Копенгаген А/С Конъюгаты антисмысловых соединений, направленные на аполипопротеин в
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
AU2014284152B2 (en) 2013-06-21 2020-01-23 Ionis Pharmaceuticals, Inc. Compositions and methods for modulation of target nucleic acids
SG10201908122XA (en) 2013-06-27 2019-10-30 Roche Innovation Ct Copenhagen As Antisense oligomers and conjugates targeting pcsk9
TW201610151A (zh) 2013-09-23 2016-03-16 阿尼拉製藥公司 治療或預防與甲狀腺素運載蛋白相關疾病之方法
EP4534092A3 (en) * 2014-05-01 2025-07-02 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating pkk expression
JOP20200115A1 (ar) 2014-10-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات وطرق لتثبيط التعبير الجيني عن hao1 (حمض أوكسيداز هيدروكسيلي 1 (أوكسيداز جليكولات))
SG11201702877TA (en) 2014-10-10 2017-05-30 Hoffmann La Roche Galnac phosphoramidites, nucleic acid conjugates thereof and their use
JOP20200092A1 (ar) * 2014-11-10 2017-06-16 Alnylam Pharmaceuticals Inc تركيبات iRNA لفيروس الكبد B (HBV) وطرق لاستخدامها
KR20250107273A (ko) 2015-05-06 2025-07-11 알닐람 파마슈티칼스 인코포레이티드 인자 ＸＩＩ(하게만 인자)(Ｆ12), 칼리크레인 Ｂ, 혈장(플레처 인자) 1(ＫＬＫＢ1) 및 키니노겐 1(ＫＮＧ1) ｉＲＮＡ 조성물 및 그의 이용 방법
MA45478A (fr) 2016-04-11 2019-02-20 Arbutus Biopharma Corp Compositions de conjugués d'acides nucléiques ciblés
JP2020516296A (ja) 2017-04-11 2020-06-11 アルブータス・バイオファーマー・コーポレイション 標的化組成物
CA3059446A1 (en) * 2017-04-18 2018-10-25 Alnylam Pharmaceuticals, Inc. Methods for the treatment of subjects having a hepatitis b virus (hbv) infection
MA55375A (fr) 2019-03-20 2022-01-26 Arbutus Biopharma Corp Méthodes thérapeutiques de traitement de l'hépatite b
US20220305107A1 (en) * 2019-06-18 2022-09-29 Janssen Sciences Ireland Unlimited Company COMBINATION OF HEPATITIS B VIRUS (HBV) VACCINES AND HBV-TARGETING RNAi

Also Published As

Publication number Publication date
MA52546A (fr) 2020-02-19
AU2025201146A1 (en) 2025-03-06
SG11201909433XA (en) 2019-11-28
WO2018191278A3 (en) 2018-11-22
KR102866327B1 (ko) 2025-10-02
MX2024004433A (es) 2024-08-28
CN110741087B (zh) 2024-05-14
JP2024174053A (ja) 2024-12-13
US11427823B2 (en) 2022-08-30
US12043833B2 (en) 2024-07-23
BR112019021359A2 (pt) 2020-05-05
JP2020516296A (ja) 2020-06-11
EP3950004A1 (en) 2022-02-09
TW201842183A (zh) 2018-12-01
US20250179496A1 (en) 2025-06-05
KR102656438B1 (ko) 2024-04-12
AU2018250553A1 (en) 2019-11-07
MX2024004432A (es) 2024-08-28
US20200407724A1 (en) 2020-12-31
CN110741087A (zh) 2020-01-31
TW202413641A (zh) 2024-04-01
CA3059426A1 (en) 2018-10-18
TWI805577B (zh) 2023-06-21
US20230110295A1 (en) 2023-04-13
JP7564173B2 (ja) 2024-10-08
AU2018250553B2 (en) 2024-11-21
MX2019012280A (es) 2020-01-23
CN118460536A (zh) 2024-08-09
EP3610017A2 (en) 2020-02-19
JP2023017897A (ja) 2023-02-07
WO2018191278A2 (en) 2018-10-18
KR20240049852A (ko) 2024-04-17
NZ757927A (en) 2023-07-28
PH12019502333A1 (en) 2020-09-28
KR20200005555A (ko) 2020-01-15

Similar Documents

Publication Publication Date Title
KR102866327B1 (ko) 표적화 조성물
JP7604427B2 (ja) 標的核酸コンジュゲート組成物
IL282794B1 (en) A membrane-destabilizing polymer conjugate and nucleic acid for use in delivering nucleic acid into the cell
HK40057109A (en) Targeted compositions
KR102905848B1 (ko) 표적화 핵산 접합체 조성물
BR112018070956B1 (pt) Composto, composição farmacêutica e uso de um composto

Legal Events

Date Code Title Description
A16 Divisional, continuation or continuation in part application filed

Free format text: ST27 STATUS EVENT CODE: A-0-1-A10-A16-DIV-PA0104 (AS PROVIDED BY THE NATIONAL OFFICE)

PA0104 Divisional application for international application

St.27 status event code: A-0-1-A10-A16-div-PA0104

PG1501 Laying open of application

St.27 status event code: A-1-1-Q10-Q12-nap-PG1501

Q12 Application published

Free format text: ST27 STATUS EVENT CODE: A-1-1-Q10-Q12-NAP-PG1501 (AS PROVIDED BY THE NATIONAL OFFICE)