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
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
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
EP13192930 2013-11-14
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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ES2607471T3 (es) 2002-11-18 2017-03-31 Roche Innovation Center Copenhagen A/S Diseño antisentido
WO2010064146A2 (en) 2008-12-02 2010-06-10 Chiralgen, Ltd. Method for the synthesis of phosphorus atom modified nucleic acids
IN2012DN00720A (zh) 2009-07-06 2015-06-19 Ontorii Inc
WO2012039448A1 (ja) 2010-09-24 2012-03-29 株式会社キラルジェン 不斉補助基
RU2014105311A (ru) 2011-07-19 2015-08-27 Уэйв Лайф Сайенсес Пте. Лтд. Способы синтеза функционализованных нуклеиновых кислот
PT2872485T (pt) 2012-07-13 2021-03-05 Wave Life Sciences Ltd Grupo auxiliar assimétrico
CA2878945A1 (en) 2012-07-13 2014-01-16 Wave Life Sciences Pte. Ltd. Chiral control
MX363068B (es) 2012-11-15 2019-03-07 Roche Innovation Ct Copenhagen As Conjugados de oligonucleotido.
PL2992009T3 (pl) 2013-05-01 2020-11-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji apolipoproteiny(a)
US10144933B2 (en) 2014-01-15 2018-12-04 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator
JPWO2015108048A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤
CN106068325B (zh) 2014-01-16 2021-07-09 波涛生命科学有限公司 手性设计
ES2812099T3 (es) 2014-05-01 2021-03-16 Ionis Pharmaceuticals Inc Composiciones y métodos para modular la expresión del receptor de la hormona del crecimiento
PL3331892T3 (pl) 2015-08-06 2019-11-29 Hoffmann La Roche Sposób wytwarzania pochodnych kwasu GalNAc
US20210052631A1 (en) * 2015-09-25 2021-02-25 Ionis Pharmaceuticals, Inc. Conjugated antisense compounds and their use
KR20180056766A (ko) * 2015-10-09 2018-05-29 웨이브 라이프 사이언시스 리미티드 뉴클레오티드 조성물 및 이의 방법
WO2017084987A1 (en) 2015-11-16 2017-05-26 F. Hoffmann-La Roche Ag GalNAc CLUSTER PHOSPHORAMIDITE
KR102468177B1 (ko) 2016-04-14 2022-11-16 에프. 호프만-라 로슈 아게 트리틸-모노-GalNAc 화합물 및 이의 용도
US20220354888A1 (en) 2016-08-03 2022-11-10 Aalborg Universitet ANTISENSE OLIGONUCLEOTIDES (ASOs) DESIGNED TO INHIBIT IMMUNE CHECKPOINT PROTEINS
JP7048574B2 (ja) 2017-03-10 2022-04-05 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
JP6884268B2 (ja) * 2018-03-09 2021-06-09 第一三共株式会社 糖原病Ia型治療薬
JP2023533693A (ja) * 2020-06-24 2023-08-04 サプリーム テクノロジーズ,ベー.フェー. GalNAc-オリゴヌクレオチドコンジュゲート及びサポニンの治療用配合物、及びその使用
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
WO2022200632A1 (en) 2021-03-26 2022-09-29 Neumirna Therapeutics Aps Microrna-134 inhibitors
CN117642505A (zh) 2021-06-04 2024-03-01 神经微核糖核酸治疗有限公司 靶向腺苷激酶的反义寡核苷酸
EP4355369A1 (en) * 2021-06-18 2024-04-24 Sapreme Technologies B.V. Conjugate of saponin, oligonucleotide and galnac
CA3228833A1 (en) 2021-08-19 2023-02-23 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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EP1931778A2 (en) * 2005-09-15 2008-06-18 Santaris Pharma A/S RNA ANTAGONIST COMPOUNDS FOR THE INHIBITION OF APO-Bl00 EXPRESSION
EP3156077B1 (en) * 2007-12-04 2022-03-09 Arbutus Biopharma Corporation Targeting lipids
EP2384197B1 (en) * 2008-12-31 2016-04-06 Roche Innovation Center Copenhagen A/S Use of lna apob antisense oligomers for the treatment of acute coronary syndromes
MX2011013078A (es) * 2009-06-12 2012-02-01 Santaris Pharma As Nuevos compuestos potentes antisentido anti apolipoproteina b (apob).
CA2788600C (en) * 2010-02-24 2019-12-03 Arrowhead Research Corporation Compositions for targeted delivery of sirna
CA2816155C (en) * 2010-12-17 2020-10-27 Arrowhead Research Corporation Galactose cluster-pharmacokinetic modulator targeting moiety for sirna
CA2893801A1 (en) * 2013-01-30 2014-08-07 F. Hoffmann-La Roche Ag Lna oligonucleotide carbohydrate conjugates
PL2992009T3 (pl) * 2013-05-01 2020-11-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji apolipoproteiny(a)

Also Published As

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

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