HUE049931T2 - Nukleinsav, amely kapcsolva van trivalens glikokonjugátumhoz - Google Patents

Nukleinsav, amely kapcsolva van trivalens glikokonjugátumhoz

Info

Publication number
HUE049931T2
HUE049931T2 HUE17715730A HUE17715730A HUE049931T2 HU E049931 T2 HUE049931 T2 HU E049931T2 HU E17715730 A HUE17715730 A HU E17715730A HU E17715730 A HUE17715730 A HU E17715730A HU E049931 T2 HUE049931 T2 HU E049931T2
Authority
HU
Hungary
Prior art keywords
glycoconjugate
trivalent
nucleic acid
acid linked
linked
Prior art date
Application number
HUE17715730A
Other languages
English (en)
Hungarian (hu)
Inventor
Christian Frauendorf
Mark Cameron
Original Assignee
Silence Therapeutics Gmbh
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 Silence Therapeutics Gmbh filed Critical Silence Therapeutics Gmbh
Publication of HUE049931T2 publication Critical patent/HUE049931T2/hu

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7125Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • 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
    • 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
    • A61K47/56Medicinal 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
    • 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
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/04Anorexiants; Antiobesity agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00Drugs for disorders of the metabolism
    • A61P3/06Antihyperlipidemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P31/00Antiinfectives, i.e. antibiotics, antiseptics, chemotherapeutics
    • A61P31/12Antivirals
    • 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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • A61P5/06Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P5/00Drugs for disorders of the endocrine system
    • A61P5/06Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH
    • A61P5/08Drugs for disorders of the endocrine system of the anterior pituitary hormones, e.g. TSH, ACTH, FSH, LH, PRL, GH for decreasing, blocking or antagonising the activity of the anterior pituitary hormones
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/04Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P7/00Drugs for disorders of the blood or the extracellular fluid
    • A61P7/06Antianaemics
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • 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
    • C12N2320/32Special delivery means, e.g. tissue-specific

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • General Health & Medical Sciences (AREA)
  • Veterinary Medicine (AREA)
  • Medicinal Chemistry (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Diabetes (AREA)
  • Molecular Biology (AREA)
  • Epidemiology (AREA)
  • Hematology (AREA)
  • Genetics & Genomics (AREA)
  • Biochemistry (AREA)
  • Biomedical Technology (AREA)
  • Obesity (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Cardiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Endocrinology (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Child & Adolescent Psychology (AREA)
  • Virology (AREA)
  • Communicable Diseases (AREA)
HUE17715730A 2016-04-05 2017-04-05 Nukleinsav, amely kapcsolva van trivalens glikokonjugátumhoz HUE049931T2 (hu)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
EP16163939.8A EP3228326A1 (en) 2016-04-05 2016-04-05 Nucleic acid linked to a trivalent glycoconjugate

Publications (1)

Publication Number Publication Date
HUE049931T2 true HUE049931T2 (hu) 2020-11-30

Family

ID=55697082

Family Applications (1)

Application Number Title Priority Date Filing Date
HUE17715730A HUE049931T2 (hu) 2016-04-05 2017-04-05 Nukleinsav, amely kapcsolva van trivalens glikokonjugátumhoz

Country Status (11)

Country Link
US (2) US10913945B2 (enExample)
EP (3) EP3228326A1 (enExample)
JP (1) JP7120925B2 (enExample)
CN (2) CN116036117A (enExample)
AU (1) AU2017245687B2 (enExample)
CA (1) CA3019981A1 (enExample)
DK (1) DK3439703T3 (enExample)
ES (1) ES2805026T3 (enExample)
HU (1) HUE049931T2 (enExample)
PL (1) PL3439703T3 (enExample)
WO (1) WO2017174657A1 (enExample)

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MA45478A (fr) * 2016-04-11 2019-02-20 Arbutus Biopharma Corp Compositions de conjugués d'acides nucléiques ciblés
EP3385272A1 (en) * 2017-04-05 2018-10-10 Silence Therapeutics GmbH Further novel oligonucleotide-ligand conjugates
RS63887B1 (sr) 2017-04-05 2023-02-28 Silence Therapeutics Gmbh Inhibicija tmprss6 posredovana rnk interferencijom
EP3483269A1 (en) 2017-11-13 2019-05-15 Silence Therapeutics GmbH Products and compositions
WO2018185252A1 (en) 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Nucleic acid conjugates
CA3056179A1 (en) 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Products and compositions
WO2018185253A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Ligand modified double-stranded nucleic acids
CN110520531A (zh) 2017-04-05 2019-11-29 赛伦斯治疗有限责任公司 产品和组合物
EP3550022A1 (en) 2018-04-05 2019-10-09 Silence Therapeutics GmbH Products and compositions
WO2019092282A1 (en) 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of aldh2 in a cell
CA3081910A1 (en) * 2017-11-13 2019-05-16 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of a target gene comprising phosphorodithioate linkages
EP3483270A1 (en) 2017-11-13 2019-05-15 Silence Therapeutics GmbH Products and compositions
FI3710586T3 (fi) 2017-11-13 2023-03-02 Nukleiinihappoja LPA:n ekspression inhiboimiseksi solussa
EP3549610A1 (en) 2018-04-05 2019-10-09 Silence Therapeutics GmbH Nucleic acid conjugates
EP3775207A1 (en) 2018-04-05 2021-02-17 Silence Therapeutics GmbH Sirnas with vinylphosphonate at the 5' end of the antisense strand
EP3550021A1 (en) 2018-04-05 2019-10-09 Silence Therapeutics GmbH Products and compositions for inhibiting expression of a target gene
EP3598995A1 (en) 2018-07-26 2020-01-29 Silence Therapeutics GmbH Products and compositions
WO2020099476A1 (en) * 2018-11-13 2020-05-22 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of lpa in a cell
EP3730617A1 (en) 2019-04-26 2020-10-28 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of c3 in a cell
WO2020104669A1 (en) 2018-11-23 2020-05-28 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of c3 in a cell
MX2021006210A (es) 2018-11-26 2021-08-19 Asociacion Centro De Investig Cooperativa En Biociencias Cic Biogune Metodos para diagnosticar y/o tratar una hepatopatia, nefropatia o neumopatia aguda o cronica.
US20220243202A1 (en) 2019-05-07 2022-08-04 Universität Bern Nucleic acids for inhibiting expression of pros1 in a cell
CN114981430A (zh) * 2019-08-27 2022-08-30 赛伦斯治疗有限责任公司 用于抑制细胞中的c3表达的核酸
KR20220058578A (ko) 2019-09-03 2022-05-09 아크투루스 쎄라퓨틱스, 인크. 치료 활성 접합체의 아시알로당단백질 수용체 매개 전달
IL292391A (en) * 2019-10-24 2022-06-01 Genevant Sciences Gmbh Bracelets and methods for treating acromegaly
US20240229041A2 (en) 2020-05-27 2024-07-11 Asociación Centro De Investigación Cooperativa En Biociencias-Cic Biogune Nucleic acids for inhibiting expression of cnnm4 in a cell
WO2022096424A1 (en) 2020-11-04 2022-05-12 Universität Bern Nucleic acids for inhibiting expression of pros1 in a cell
JP2023547512A (ja) 2020-11-04 2023-11-10 ウニベルジテート ベルン 細胞におけるpros1の発現を阻害するための核酸
WO2022184852A1 (en) 2021-03-03 2022-09-09 Silence Therapeutics Gmbh Conjugated nucleic acids comprising a phosphorodithioate for inhibiting gene expression in a cell
WO2022223557A1 (en) 2021-04-20 2022-10-27 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of xdh in a cell
AU2022265493A1 (en) 2021-04-27 2023-09-21 Silence Therapeutics Gmbh Sirna targeting tmprss6 for the treatment of myeloproliferative disorders
CN118234861A (zh) 2021-09-02 2024-06-21 赛伦斯治疗有限责任公司 用于抑制细胞中的补体因子b(cfb)表达的核酸
US20240271141A1 (en) * 2021-11-29 2024-08-15 Shanghai Argo Biopharmaceutical Co., Ltd. Composition and method for inhibiting expression of hepatitis b virus (hbv)protein
WO2023208106A1 (zh) * 2022-04-29 2023-11-02 石药集团中奇制药技术(石家庄)有限公司 具有肝靶向递送效应的化合物及其寡聚核苷酸缀合物
WO2024013334A1 (en) * 2022-07-15 2024-01-18 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of agt in a cell
WO2024126687A1 (en) 2022-12-16 2024-06-20 Silence Therapeutics Gmbh Nucleic acids conjugated to an albumin binding moiety
CN120981247A (zh) 2023-03-27 2025-11-18 赛伦斯治疗有限责任公司 用于干细胞移植的化合物和组合物
CN116444589B (zh) * 2023-06-15 2023-09-19 北京悦康科创医药科技股份有限公司 新型GalNAc化合物及其硫代寡核苷酸缀合物和偶联方法
GB202311334D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
GB202311324D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
WO2025021831A1 (en) 2023-07-24 2025-01-30 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
WO2025132845A1 (en) 2023-12-21 2025-06-26 Silence Therapeutics Gmbh Galnac phosphoramidites and their uses
WO2025162939A1 (en) 2024-01-29 2025-08-07 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of gpr146 in a cell
WO2025163186A1 (en) 2024-02-01 2025-08-07 Silence Therapeutics Gmbh Conjugated nucleic acids and nucleic acids comprising locked nucleosides and inverted nucleotides for inhibiting gene expression in a cell
WO2025169133A1 (en) 2024-02-09 2025-08-14 Astrazeneca Ab Double-stranded rnai agents and compositions for reducing expression of angiopoietin-like 3 (angptl3) and methods of use thereof

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Also Published As

Publication number Publication date
PL3439703T3 (pl) 2020-10-19
AU2017245687B2 (en) 2023-03-09
EP3439703B1 (en) 2020-04-29
WO2017174657A1 (en) 2017-10-12
CN109562188B (zh) 2022-12-06
US20190119676A1 (en) 2019-04-25
JP2019510793A (ja) 2019-04-18
EP3744350A1 (en) 2020-12-02
EP3439703A1 (en) 2019-02-13
US20210079397A1 (en) 2021-03-18
ES2805026T3 (es) 2021-02-10
CN116036117A (zh) 2023-05-02
CA3019981A1 (en) 2017-10-12
US10913945B2 (en) 2021-02-09
AU2017245687A1 (en) 2018-10-25
EP3228326A1 (en) 2017-10-11
DK3439703T3 (da) 2020-06-29
JP7120925B2 (ja) 2022-08-17
CN109562188A (zh) 2019-04-02
US11912993B2 (en) 2024-02-27

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