HK1220724A1 - 反義綴合物化合物 - Google Patents

反義綴合物化合物

Info

Publication number
HK1220724A1
HK1220724A1 HK16108771.0A HK16108771A HK1220724A1 HK 1220724 A1 HK1220724 A1 HK 1220724A1 HK 16108771 A HK16108771 A HK 16108771A HK 1220724 A1 HK1220724 A1 HK 1220724A1
Authority
HK
Hong Kong
Prior art keywords
apob
conjugate compounds
antisense conjugate
antisense
compounds
Prior art date
Application number
HK16108771.0A
Other languages
English (en)
Inventor
南納.阿爾貝克
亨里克.弗呂登倫.漢森
蘇珊.卡姆勒
瑪麗.林霍爾姆
馬克.圖爾納Ø
亨里克.奧魯姆
Original Assignee
Roche Innovation Ct Copenhagen As
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
Priority claimed from PCT/EP2013/073859 external-priority patent/WO2014076196A1/en
Application filed by Roche Innovation Ct Copenhagen As filed Critical Roche Innovation Ct Copenhagen As
Publication of HK1220724A1 publication Critical patent/HK1220724A1/zh

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/712Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61P9/00Drugs for disorders of the cardiovascular system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00Drugs for disorders of the cardiovascular system
    • A61P9/10Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
    • 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
    • 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
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/341Gapmers, i.e. of the type ===---===
    • 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)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Heart & Thoracic Surgery (AREA)
  • Cardiology (AREA)
  • Vascular Medicine (AREA)
  • Urology & Nephrology (AREA)
  • Hematology (AREA)
  • Diabetes (AREA)
  • Obesity (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
HK16108771.0A 2013-11-14 2016-07-21 反義綴合物化合物 HK1220724A1 (zh)

Applications Claiming Priority (6)

Application Number Priority Date Filing Date Title
EP13192930 2013-11-14
PCT/EP2013/073859 WO2014076196A1 (en) 2012-11-15 2013-11-14 Anti apob antisense conjugate compounds
PCT/EP2013/073858 WO2014076195A1 (en) 2012-11-15 2013-11-14 Oligonucleotide conjugates
EP14153266 2014-01-30
EP14167879 2014-05-12
PCT/EP2014/074554 WO2015071388A1 (en) 2013-11-14 2014-11-14 Apob antisense conjugate compounds

Publications (1)

Publication Number Publication Date
HK1220724A1 true HK1220724A1 (zh) 2017-05-12

Family

ID=53056817

Family Applications (1)

Application Number Title Priority Date Filing Date
HK16108771.0A HK1220724A1 (zh) 2013-11-14 2016-07-21 反義綴合物化合物

Country Status (10)

Country Link
US (1) US20160289677A1 (zh)
EP (1) EP3068885A1 (zh)
JP (1) JP2017501684A (zh)
KR (1) KR20160083876A (zh)
CN (1) CN105722980A (zh)
CA (1) CA2928349A1 (zh)
HK (1) HK1220724A1 (zh)
MX (1) MX2016005855A (zh)
RU (1) RU2016122168A (zh)
WO (1) WO2015071388A1 (zh)

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EP2141233B1 (en) 2002-11-18 2016-10-19 Roche Innovation Center Copenhagen A/S Antisense design
BRPI0923225A2 (pt) 2008-12-02 2016-10-04 Chiralgen Ltd metodo para sintese de acidos nucleicos modificados no atomo de fosforo
CA2767253A1 (en) 2009-07-06 2011-01-13 Ontorii, Inc. Novel nucleic acid prodrugs and methods of use thereof
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
EP2734208B1 (en) 2011-07-19 2017-03-01 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
KR20220139425A (ko) 2012-07-13 2022-10-14 웨이브 라이프 사이언시스 리미티드 키랄 제어
WO2014010250A1 (en) 2012-07-13 2014-01-16 Chiralgen, Ltd. Asymmetric auxiliary group
BR112015010116A2 (pt) 2012-11-15 2017-08-22 Roche Innovation Ct Copenhagen As COMPOSTOS CONJUGADOS ANTI-SENTIDO ANTI-ApoB
CR20190269A (es) 2013-05-01 2019-09-13 Ionis Pharmaceuticals Inc COMPOSICIONES Y MÉTODOS PARA MODULAR LA EXPRESIÓN DE TTR Y VHB (Divisonal 2015-0612)
EP3095459A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having antitumor effect and antitumor agent
EP3095461A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
MY193116A (en) 2014-01-16 2022-09-26 Wave Life Sciences Ltd Chiral design
MX2016014013A (es) 2014-05-01 2016-11-15 Ionis Pharmaceuticals Inc Composiciones y metodos para modular la expresion del receptor de la hormona del crecimiento.
JP6940477B2 (ja) * 2015-08-06 2021-09-29 エフ.ホフマン−ラ ロシュ アーゲーF. Hoffmann−La Roche Aktiengesellschaft GalNAc酸誘導体の調製法
WO2017053995A1 (en) * 2015-09-25 2017-03-30 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
CR20180233A (es) * 2015-10-09 2018-05-25 Wave Life Sciences Ltd Composiciones oligonucleotídicas y sus métodos
WO2017084987A1 (en) 2015-11-16 2017-05-26 F. Hoffmann-La Roche Ag GalNAc CLUSTER PHOSPHORAMIDITE
WO2017178656A1 (en) 2016-04-14 2017-10-19 Roche Innovation Center Copenhagen A/S TRITYL-MONO-GalNAc COMPOUNDS AND THEIR USE
WO2018024849A1 (en) 2016-08-03 2018-02-08 Aalborg Universitet ANTISENSE OLIGONUCLEOTIDES (ASOs) DESIGNED TO INHIBIT IMMUNE CHECKPOINT PROTEINS
WO2018164275A1 (ja) 2017-03-10 2018-09-13 国立研究開発法人国立成育医療研究センター アンチセンスオリゴヌクレオチドおよび糖原病Ia型予防または治療用組成物
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
WO2018215049A1 (en) 2017-05-23 2018-11-29 F. Hoffmann-La Roche Ag Process for galnac oligonucleotide conjugates
KR20210130854A (ko) * 2018-03-09 2021-11-01 다이이찌 산쿄 가부시키가이샤 당원병 Ia형 치료약
US20230293562A1 (en) * 2020-06-24 2023-09-21 Sapreme Technologies B.V. Therapeutic combination of galnac-oligonucleotide conjugate and saponin, and uses thereof
WO2022055351A1 (en) * 2020-09-10 2022-03-17 Sapreme Technologies B.V. Conjugate of saponin, oligonucleotide and galnac
WO2022055352A1 (en) * 2020-09-10 2022-03-17 Sapreme Technologies B.V. Semicarbazone-based saponin conjugate
WO2022164316A1 (en) * 2021-01-26 2022-08-04 Sapreme Technologies B.V. Semicarbazone-based saponin conjugate
CA3213673A1 (en) 2021-03-26 2022-09-29 Neumirna Therapeutics Aps Microrna-27b inhibitors
JP2024510663A (ja) 2021-03-26 2024-03-08 ニューミルナ セラピューティクス エーピーエス マイクロrna-134阻害剤
JP2024522272A (ja) 2021-06-04 2024-06-13 ニューミルナ セラピューティクス エーピーエス アデノシンキナーゼを標的とするアンチセンスオリゴヌクレオチド
JP2024530839A (ja) * 2021-06-18 2024-08-26 サプリーム テクノロジーズ,ベー.フェー. サポニン、オリゴヌクレオチド、およびGalNAcの複合体
AU2022329462A1 (en) 2021-08-19 2024-03-28 Neumirna Therapeutics Aps Antisense oligonucleotides targeting adenosine kinase
WO2023038517A1 (en) * 2021-09-09 2023-03-16 Sapreme Technologies B.V. Semicarbazone-based saponin conjugate
CN115487308A (zh) * 2022-09-29 2022-12-20 北京大学 反义核酸糖基缀合物及其制备方法和在肝癌治疗中的应用

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EA200800823A1 (ru) * 2005-09-15 2008-10-30 Сантарис Фарма А/С Соединения-антагонисты рнк для ингибирования экспрессии аро-в100
EP3141265A1 (en) * 2007-12-04 2017-03-15 Alnylam Pharmaceuticals, Inc. Carbohydrate conjugates as delivery agents for oligonucleotides
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JP2012529279A (ja) * 2009-06-12 2012-11-22 サンタリス ファーマ アー/エス 新規の強力な抗apobアンチセンス化合物
EP3023495B1 (en) * 2010-02-24 2019-05-08 Arrowhead Pharmaceuticals, Inc. Compositions for targeted delivery of sirna
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BR112015018161A2 (pt) * 2013-01-30 2017-08-22 Hoffmann La Roche Conjugado de oligômeros antisense de lna, composição farmacêutica e uso de um conjugado de oligômeros antisense de lna
CR20190269A (es) * 2013-05-01 2019-09-13 Ionis Pharmaceuticals Inc COMPOSICIONES Y MÉTODOS PARA MODULAR LA EXPRESIÓN DE TTR Y VHB (Divisonal 2015-0612)

Also Published As

Publication number Publication date
RU2016122168A (ru) 2017-12-19
MX2016005855A (es) 2016-07-13
EP3068885A1 (en) 2016-09-21
KR20160083876A (ko) 2016-07-12
WO2015071388A1 (en) 2015-05-21
JP2017501684A (ja) 2017-01-19
US20160289677A1 (en) 2016-10-06
CA2928349A1 (en) 2015-05-21
CN105722980A (zh) 2016-06-29

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