PL3439703T3 - Kwas nukleinowy połączony z trójwartościowym glikokoniugatem - Google Patents

Kwas nukleinowy połączony z trójwartościowym glikokoniugatem

Info

Publication number
PL3439703T3
PL3439703T3 PL17715730T PL17715730T PL3439703T3 PL 3439703 T3 PL3439703 T3 PL 3439703T3 PL 17715730 T PL17715730 T PL 17715730T PL 17715730 T PL17715730 T PL 17715730T PL 3439703 T3 PL3439703 T3 PL 3439703T3
Authority
PL
Poland
Prior art keywords
glycoconjugate
trivalent
nucleic acid
acid linked
linked
Prior art date
Application number
PL17715730T
Other languages
English (en)
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 PL3439703T3 publication Critical patent/PL3439703T3/pl

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)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (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)
  • Heart & Thoracic Surgery (AREA)
  • Pain & Pain Management (AREA)
  • Cardiology (AREA)
  • Endocrinology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Child & Adolescent Psychology (AREA)
  • Rheumatology (AREA)
  • Virology (AREA)
  • Communicable Diseases (AREA)
PL17715730T 2016-04-05 2017-04-05 Kwas nukleinowy połączony z trójwartościowym glikokoniugatem PL3439703T3 (pl)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
EP16163939.8A EP3228326A1 (en) 2016-04-05 2016-04-05 Nucleic acid linked to a trivalent glycoconjugate
EP17715730.2A EP3439703B1 (en) 2016-04-05 2017-04-05 Nucleic acid linked to a trivalent glycoconjugate
PCT/EP2017/058112 WO2017174657A1 (en) 2016-04-05 2017-04-05 Nucleic acid linked to a trivalent glycoconjugate

Publications (1)

Publication Number Publication Date
PL3439703T3 true PL3439703T3 (pl) 2020-10-19

Family

ID=55697082

Family Applications (1)

Application Number Title Priority Date Filing Date
PL17715730T PL3439703T3 (pl) 2016-04-05 2017-04-05 Kwas nukleinowy połączony z trójwartościowym glikokoniugatem

Country Status (11)

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

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MA45478A (fr) * 2016-04-11 2019-02-20 Arbutus Biopharma Corp Compositions de conjugués d'acides nucléiques ciblés
EP3483269A1 (en) 2017-11-13 2019-05-15 Silence Therapeutics GmbH Products and compositions
SI3607068T1 (sl) * 2017-04-05 2023-02-28 Silence Therapeutics Gmbh Z RNA interferenco posredovano inhibiranje TMPRSS6
EP3385272A1 (en) 2017-04-05 2018-10-10 Silence Therapeutics GmbH Further novel oligonucleotide-ligand conjugates
EP3550022A1 (en) 2018-04-05 2019-10-09 Silence Therapeutics GmbH Products and compositions
RS63836B1 (sr) * 2017-04-05 2023-01-31 Silence Therapeutics Gmbh Proizvodi i sastavi
EP4029942A1 (en) 2017-04-05 2022-07-20 Silence Therapeutics GmbH Products and compositions
WO2018185253A1 (en) * 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Ligand modified double-stranded nucleic acids
WO2018185252A1 (en) 2017-04-05 2018-10-11 Silence Therapeutics Gmbh Nucleic acid conjugates
CN111527206A (zh) * 2017-11-13 2020-08-11 赛伦斯治疗有限责任公司 用于抑制靶标基因表达的包含二硫代磷酸酯键的核酸
EP3483270A1 (en) 2017-11-13 2019-05-15 Silence Therapeutics GmbH Products and compositions
IL274503B2 (en) * 2017-11-13 2024-10-01 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of lpa in a cell
EP3735461A1 (en) 2017-11-13 2020-11-11 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of aldh2 in a cell
EP3549610A1 (en) 2018-04-05 2019-10-09 Silence Therapeutics GmbH Nucleic acid conjugates
KR102857148B1 (ko) 2018-04-05 2025-09-11 사일런스 테라퓨틱스 게엠베하 안티센스 가닥의 5' 단부에 비닐포스포네이트를 갖는 siRNA
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
JP7245328B2 (ja) 2018-11-13 2023-03-23 サイレンス・セラピューティクス・ゲゼルシャフト・ミット・ベシュレンクテル・ハフツング 細胞におけるlpaの発現を抑制するための核酸
EP3730617A1 (en) 2019-04-26 2020-10-28 Silence Therapeutics GmbH Nucleic acids for inhibiting expression of c3 in a cell
CA3119234A1 (en) 2018-11-23 2020-05-28 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of c3 in a cell
SG11202105413QA (en) 2018-11-26 2021-06-29 Asociacion Centro De Investig Cooperativa En Biociencias Cic Biogune Methods for diagnosing and/or treating acute or chronic liver, kidney or lung disease
AU2020269323A1 (en) 2019-05-07 2021-10-28 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of PROS1 in a cell
MX2022002397A (es) 2019-08-27 2022-08-25 Silence Therapeutics Gmbh Acidos nucleicos para inhibir la expresion de c3 en una celula.
JP7672394B2 (ja) 2019-09-03 2025-05-07 アークトゥラス・セラピューティクス・インコーポレイテッド 治療的に活性な複合体のアシアロ糖タンパク質受容体媒介送達
CA3158917A1 (en) * 2019-10-24 2021-04-29 Genevant Sciences Gmbh Conjugates and methods for treating acromegaly
JP2023528016A (ja) 2020-05-27 2023-07-03 アソシアシオン・セントロ・デ・インベスティガシオン・コオペラティバ・エン・ビオシエンシアス-セイセ ビオグネ 細胞におけるcnnm4の発現を阻害するための核酸
EP4240851A1 (en) 2020-11-04 2023-09-13 Universität Bern Nucleic acids for inhibiting expression of pros1 in a cell
TW202237840A (zh) 2020-11-04 2022-10-01 瑞士伯恩大學 用於在細胞中抑制pros1之表現的核酸
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
US20240240187A1 (en) 2021-04-27 2024-07-18 Silence Therapeutics Gmbh Sirna targeting tmprss6 for the treatment of myeloproliferative disorders
AU2022336157A1 (en) 2021-09-02 2024-03-14 Silence Therapeutics Gmbh Nucleic acids for inhibiting expression of complement factor b (cfb) in a cell
CN117043338A (zh) * 2021-11-29 2023-11-10 上海舶望制药有限公司 用于抑制乙型肝炎病毒(hbv)蛋白表达的组合物和方法
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
EP4687983A1 (en) 2023-03-27 2026-02-11 Silence Therapeutics GmbH Compounds and compositions for use in stem cell transplantation
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
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
GB202311334D0 (en) 2023-07-24 2023-09-06 Astrazeneca Ab Multivalent cargo-carrying complexes and uses thereof
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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
WO2026013624A2 (en) 2024-07-12 2026-01-15 Novartis Ag Double stranded rnai agents, compositions and methods of use

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Also Published As

Publication number Publication date
CN109562188B (zh) 2022-12-06
CN116036117A (zh) 2023-05-02
EP3228326A1 (en) 2017-10-11
US20210079397A1 (en) 2021-03-18
AU2017245687B2 (en) 2023-03-09
CA3019981A1 (en) 2017-10-12
ES2805026T3 (es) 2021-02-10
AU2017245687A1 (en) 2018-10-25
JP2019510793A (ja) 2019-04-18
EP3439703B1 (en) 2020-04-29
EP3744350A1 (en) 2020-12-02
EP3439703A1 (en) 2019-02-13
WO2017174657A1 (en) 2017-10-12
DK3439703T3 (da) 2020-06-29
JP7120925B2 (ja) 2022-08-17
HUE049931T2 (hu) 2020-11-30
US11912993B2 (en) 2024-02-27
CN109562188A (zh) 2019-04-02
US10913945B2 (en) 2021-02-09
US20190119676A1 (en) 2019-04-25

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