KR20200131263A - 변형 올리고뉴클레오티드 및 타우병증에서의 사용 방법 - Google Patents

변형 올리고뉴클레오티드 및 타우병증에서의 사용 방법 Download PDF

Info

Publication number
KR20200131263A
KR20200131263A KR1020207027936A KR20207027936A KR20200131263A KR 20200131263 A KR20200131263 A KR 20200131263A KR 1020207027936 A KR1020207027936 A KR 1020207027936A KR 20207027936 A KR20207027936 A KR 20207027936A KR 20200131263 A KR20200131263 A KR 20200131263A
Authority
KR
South Korea
Prior art keywords
oligonucleotide
formula
modified
nucleotides
nucleobase
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.)
Ceased
Application number
KR1020207027936A
Other languages
English (en)
Korean (ko)
Inventor
안드레아스 에브네스
콘스탄틴 판 아우트리베 디에발레
세르게이 그리야즈노브
사울 마르티네즈 몬테로
비벡 쿠마르 라지완시
레오니드 베이겔만
Original Assignee
얀센 파마슈티카 엔.브이.
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 얀센 파마슈티카 엔.브이. filed Critical 얀센 파마슈티카 엔.브이.
Publication of KR20200131263A publication Critical patent/KR20200131263A/ko
Ceased legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • 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
    • A61K31/7125—Nucleic acids or oligonucleotides having modified internucleoside linkage, i.e. other than 3'-5' phosphodiesters
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • 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
    • C12N2310/11—Antisense
    • 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
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/335—Modified T or U
    • 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
    • C12N2310/341—Gapmers, i.e. of the type ===---===
    • 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
    • C12N2310/345—Spatial arrangement of the modifications having at least two different backbone 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
    • C12N2310/34—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
    • C12N2310/352—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
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3525—MOE, methoxyethoxy
    • 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
    • C12N2320/00—Applications; Uses
    • C12N2320/50—Methods for regulating/modulating their activity

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Neurology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Physics & Mathematics (AREA)
  • Biophysics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Psychiatry (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hospice & Palliative Care (AREA)
  • Epidemiology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Saccharide Compounds (AREA)
KR1020207027936A 2018-03-13 2019-03-13 변형 올리고뉴클레오티드 및 타우병증에서의 사용 방법 Ceased KR20200131263A (ko)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862642499P 2018-03-13 2018-03-13
US62/642,499 2018-03-13
PCT/EP2019/056312 WO2019175260A2 (en) 2018-03-13 2019-03-13 Modified oligonucleotides and methods of use in tauopathies

Publications (1)

Publication Number Publication Date
KR20200131263A true KR20200131263A (ko) 2020-11-23

Family

ID=65861258

Family Applications (1)

Application Number Title Priority Date Filing Date
KR1020207027936A Ceased KR20200131263A (ko) 2018-03-13 2019-03-13 변형 올리고뉴클레오티드 및 타우병증에서의 사용 방법

Country Status (15)

Country Link
US (2) US11453881B2 (https=)
EP (2) EP3922637A1 (https=)
JP (1) JP2021515784A (https=)
KR (1) KR20200131263A (https=)
CN (1) CN111868068A (https=)
AU (1) AU2019233612A1 (https=)
BR (1) BR112020018365A2 (https=)
CA (1) CA3091979A1 (https=)
EA (1) EA202092156A1 (https=)
ES (1) ES3063421T3 (https=)
IL (1) IL277238A (https=)
MA (1) MA52149A (https=)
MX (1) MX2020009532A (https=)
SG (1) SG11202008895WA (https=)
WO (1) WO2019175260A2 (https=)

Families Citing this family (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2831232A4 (en) 2012-03-30 2015-11-04 Univ Washington METHOD FOR MODULATING TAU EXPRESSION TO REDUCE PROBLEMS AND MODIFY A NEURODEGENERATIVE SYNDROME
TW202246503A (zh) 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
WO2021198958A1 (en) * 2020-04-01 2021-10-07 Janssen Biopharma, Inc. Nucleic acid polymers
TW202310854A (zh) * 2021-07-23 2023-03-16 美商百健Ｍａ公司 用於減少ｔａｕ表現之方法
CN121729493A (zh) * 2023-06-22 2026-03-24 戴纳立制药公司 用于调节τ蛋白表达的组合物和方法
US12551569B2 (en) 2024-03-31 2026-02-17 Arrowhead Pharmaceuticals, Inc. RNAi agents for inhibiting expression of microtubule associated protein tau (MAPT), compositions thereof, and methods of use
WO2026025082A1 (en) * 2024-07-25 2026-01-29 Biogen Ma Inc. Methods for treating alzheimer's disease

Family Cites Families (20)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
JP3756313B2 (ja) 1997-03-07 2006-03-15 武 今西 新規ビシクロヌクレオシド及びオリゴヌクレオチド類縁体
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
ES2234563T5 (es) 1999-02-12 2018-01-17 Daiichi Sankyo Company, Limited Nuevos análogos de nucleósidos y oligonucleótidos
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US7053207B2 (en) 1999-05-04 2006-05-30 Exiqon A/S L-ribo-LNA analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
JP4151751B2 (ja) 1999-07-22 2008-09-17 第一三共株式会社 新規ビシクロヌクレオシド類縁体
CN1317291C (zh) 1999-09-10 2007-05-23 杰龙公司 寡核苷酸n3′→p5′硫代氨基磷酸酯,其合成及应用
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP1794313B1 (en) * 2004-06-21 2013-05-15 Proteome Sciences Plc Screening methods using c-abl, fyn and syk in combination with tau protein
GB0605337D0 (en) * 2006-03-17 2006-04-26 Genomica Sau Treatment of CNS conditions
WO2013180038A1 (ja) * 2012-05-26 2013-12-05 株式会社ボナック デリバリー機能を有する遺伝子発現制御用の一本鎖核酸分子
US9228189B2 (en) 2012-10-26 2016-01-05 Geron Corporation C-myc antisense oligonucleotides and methods for using the same to treat cell-proliferative disorders
CN105264091B (zh) * 2013-03-14 2020-02-28 Ionis制药公司 用于调节tau表达的组合物和方法
TW202246503A (zh) * 2013-07-19 2022-12-01 美商百健Ｍａ公司 用於調節τ蛋白表現之組合物
BR112017014090A2 (pt) * 2014-12-29 2018-03-06 Bonac Corporation composição, e, métodos para produção de uma composição e estabilização de uma molécula de ácido nucleico.
US11077132B2 (en) * 2015-02-04 2021-08-03 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
PH12018501207B1 (en) * 2015-12-21 2024-02-23 Novartis Ag Compositions and methods for decreasing tau expression

Also Published As

Publication number Publication date
SG11202008895WA (en) 2020-10-29
AU2019233612A1 (en) 2020-09-10
ES3063421T3 (en) 2026-04-16
EP3765476A2 (en) 2021-01-20
WO2019175260A3 (en) 2019-11-14
WO2019175260A2 (en) 2019-09-19
CA3091979A1 (en) 2019-09-19
MA52149A (fr) 2021-01-20
JP2021515784A (ja) 2021-06-24
US11453881B2 (en) 2022-09-27
EP3765476B1 (en) 2025-11-26
US20240011029A1 (en) 2024-01-11
MX2020009532A (es) 2020-10-05
EP3765476C0 (en) 2025-11-26
BR112020018365A2 (pt) 2020-12-29
IL277238A (en) 2020-10-29
EA202092156A1 (ru) 2021-04-01
US20210095284A1 (en) 2021-04-01
EP3922637A1 (en) 2021-12-15
CN111868068A (zh) 2020-10-30

Similar Documents

Publication Publication Date Title
KR20200131263A (ko) 변형 올리고뉴클레오티드 및 타우병증에서의 사용 방법
JP7478210B2 (ja) 修飾オリゴヌクレオチド及び使用方法
KR102556825B1 (ko) Smn2 조정을 위한 조성물 및 방법
TW202020154A (zh) 用於減少atxn2表現之化合物及方法
CN111373043A (zh) 用于降低snca表达的化合物和方法
JP2024041845A (ja) ホスホロジチオアートヌクレオシド間結合を含むギャップマーオリゴヌクレオチド
KR20210008498A (ko) Fxi 발현을 감소시키기 위한 화합물 및 방법
CN116322707A (zh) 用于调节scn2a的化合物和方法
JP2023139252A (ja) 新規のチオホスホラミダイト
JP2024545052A (ja) 修飾骨格化学を有するスプライススイッチャーアンチセンスオリゴヌクレオチド
CN115279379A (zh) 用于调节smn2的化合物和方法
WO2018193428A1 (en) Modified oligomeric compounds comprising tricyclo-dna nucleosides and uses thereof
JP2025039646A (ja) トリシクロｄｎａヌクレオシドを含む改変オリゴマー化合物およびその使用
AU2021284360A1 (en) Treatment of neurological diseases using modulators of gene transcripts
JP2023545041A (ja) Ｄｍｄ患者におけるエクソン５１スキッピングによるジストロフィンレスキューのためのオリゴマー化合物
HK40031698A (en) Modified oligonucleotides for use in treatment of tauopathies
RU2802836C2 (ru) Модифицированные олигонуклеотиды и способы их применения
HK40121931A (zh) 具有修饰的主链化学的gapmer反义寡核苷酸
AU2024281125A1 (en) Novel scn10a rnai agents and uses thereof
HK40011819A (en) Compounds and methods for reducing tau expression
HK40011819B (en) Compounds and methods for reducing tau expression

Legal Events

Date Code Title Description
PA0105 International application

Patent event date: 20200928

Patent event code: PA01051R01D

Comment text: International Patent Application

PG1501 Laying open of application
A201 Request for examination
PA0201 Request for examination

Patent event code: PA02012R01D

Patent event date: 20220314

Comment text: Request for Examination of Application

E902 Notification of reason for refusal
PE0902 Notice of grounds for rejection

Comment text: Notification of reason for refusal

Patent event date: 20240531

Patent event code: PE09021S01D

E601 Decision to refuse application
PE0601 Decision on rejection of patent

Patent event date: 20240809

Comment text: Decision to Refuse Application

Patent event code: PE06012S01D