JP5665317B2 - アンチセンス化合物 - Google Patents

アンチセンス化合物 Download PDF

Info

Publication number
JP5665317B2
JP5665317B2 JP2009533545A JP2009533545A JP5665317B2 JP 5665317 B2 JP5665317 B2 JP 5665317B2 JP 2009533545 A JP2009533545 A JP 2009533545A JP 2009533545 A JP2009533545 A JP 2009533545A JP 5665317 B2 JP5665317 B2 JP 5665317B2
Authority
JP
Japan
Prior art keywords
wing
antisense oligonucleotide
nucleoside
modified
antisense
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.)
Active
Application number
JP2009533545A
Other languages
English (en)
Japanese (ja)
Other versions
JP2010507579A (ja
JP2010507579A5 (enExample
Inventor
スウェイズ，エリック・イー
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Ionis Pharmaceuticals Inc
Original Assignee
Ionis Pharmaceuticals Inc
Isis Pharmaceuticals Inc
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
Family has litigation
First worldwide family litigation filed litigation Critical https://patents.darts-ip.com/?family=39052713&utm_source=google_patent&utm_medium=platform_link&utm_campaign=public_patent_search&patent=JP5665317(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Ionis Pharmaceuticals Inc, Isis Pharmaceuticals Inc filed Critical Ionis Pharmaceuticals Inc
Publication of JP2010507579A publication Critical patent/JP2010507579A/ja
Publication of JP2010507579A5 publication Critical patent/JP2010507579A5/ja
Application granted granted Critical
Publication of JP5665317B2 publication Critical patent/JP5665317B2/ja
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • 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
    • A61K31/712—Nucleic acids or oligonucleotides having modified sugars, i.e. other than ribose or 2'-deoxyribose
    • 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
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • 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
    • C12N15/111—General methods applicable to biologically active non-coding nucleic acids
    • 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
    • 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
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • 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
    • C12N2310/34—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
    • C12N2310/346—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
    • C12N2310/351—Conjugate
    • 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
    • C12N2320/53—Methods for regulating/modulating their activity reducing unwanted side-effects

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)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Saccharide Compounds (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
JP2009533545A 2006-10-18 2007-10-18 アンチセンス化合物 Active JP5665317B2 (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US85289406P 2006-10-18 2006-10-18
US60/852,894 2006-10-18
PCT/US2007/081850 WO2008049085A1 (en) 2006-10-18 2007-10-18 Antisense compounds

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2014150706A Division JP2014221817A (ja) 2006-10-18 2014-07-24 アンチセンス化合物

Publications (3)

Publication Number Publication Date
JP2010507579A JP2010507579A (ja) 2010-03-11
JP2010507579A5 JP2010507579A5 (enExample) 2010-12-02
JP5665317B2 true JP5665317B2 (ja) 2015-02-04

Family

ID=39052713

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2009533545A Active JP5665317B2 (ja) 2006-10-18 2007-10-18 アンチセンス化合物
JP2014150706A Pending JP2014221817A (ja) 2006-10-18 2014-07-24 アンチセンス化合物

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2014150706A Pending JP2014221817A (ja) 2006-10-18 2014-07-24 アンチセンス化合物

Country Status (9)

Country Link
US (4) US9550988B2 (enExample)
EP (5) EP2092065B2 (enExample)
JP (2) JP5665317B2 (enExample)
AT (1) ATE540118T1 (enExample)
AU (3) AU2007310989B2 (enExample)
CA (1) CA2667055C (enExample)
DK (3) DK2092065T4 (enExample)
ES (3) ES2526295T5 (enExample)
WO (1) WO2008049085A1 (enExample)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014221817A (ja) * 2006-10-18 2014-11-27 アイシス ファーマシューティカルズ， インコーポレーテッド アンチセンス化合物

Families Citing this family (255)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA3044969A1 (en) 2006-05-05 2007-12-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gene expression
US8580756B2 (en) * 2007-03-22 2013-11-12 Santaris Pharma A/S Short oligomer antagonist compounds for the modulation of target mRNA
US8318496B2 (en) * 2007-10-04 2012-11-27 Isis Pharmaceuticals, Inc. Compounds and methods for improving cellular uptake of oligomeric compounds
EP2274423A2 (en) 2008-04-04 2011-01-19 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
HRP20171969T1 (hr) 2008-10-15 2018-04-06 Ionis Pharmaceuticals, Inc. Modulacija ekspresije faktora 11
US9394333B2 (en) 2008-12-02 2016-07-19 Wave Life Sciences Japan Method for the synthesis of phosphorus atom modified nucleic acids
MX342945B (es) 2009-07-06 2016-10-18 Ontorii Inc * Profármacos de ácido nucleico novedosos y métodos de uso de los mismos.
WO2011097644A2 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP3561060A1 (en) 2010-02-08 2019-10-30 Ionis Pharmaceuticals, Inc. Selective reduction of allelic variants
US20130225659A1 (en) * 2010-07-19 2013-08-29 Isis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
US8648053B2 (en) 2010-10-20 2014-02-11 Rosalind Franklin University Of Medicine And Science Antisense oligonucleotides that target a cryptic splice site in Ush1c as a therapeutic for Usher syndrome
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
WO2012068405A2 (en) 2010-11-17 2012-05-24 Isis Pharmaceuticals, Inc. Modulation of alpha synuclein expression
EP3467109A1 (en) 2011-02-08 2019-04-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
WO2012161806A1 (en) * 2011-02-25 2012-11-29 Isis Pharmaceuticals, Inc. Modulation of stat3 expression
MX347253B (es) 2011-04-21 2017-04-20 Ionis Pharmaceuticals Inc Modulación de la expresión del virus de hepatitis b (vhb).
US9353371B2 (en) 2011-05-02 2016-05-31 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with usher syndrome
US9605019B2 (en) 2011-07-19 2017-03-28 Wave Life Sciences Ltd. Methods for the synthesis of functionalized nucleic acids
EP2742056B2 (en) 2011-08-11 2020-06-10 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
US9580708B2 (en) 2011-09-14 2017-02-28 Rana Therapeutics, Inc. Multimeric oligonucleotides compounds
US9243291B1 (en) * 2011-12-01 2016-01-26 Isis Pharmaceuticals, Inc. Methods of predicting toxicity
BR112014021612A2 (pt) * 2012-03-12 2017-07-18 Santaris Pharma As composições e métodos para modulação da expressão de atxn3
WO2013159108A2 (en) 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
CA2873769A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating hemoglobin gene family expression
US20150159161A1 (en) * 2012-05-16 2015-06-11 Rana Therapeutics, Inc. Compositions and methods for modulating pten expression
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
BR112014028634A2 (pt) 2012-05-16 2017-06-27 Rana Therapeutics Inc composições e métodos para modulação da expressão de utrn
BR112014028644A2 (pt) 2012-05-16 2017-08-15 Rana Therapeutics Inc Composições e métodos para modulação da expressão de atp2a2
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
WO2013177468A2 (en) 2012-05-24 2013-11-28 Isis Pharmaceuticals, Inc. Methods and compositions for modulating apolipoprotein(a) expression
US9574193B2 (en) 2012-05-17 2017-02-21 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating apolipoprotein (a) expression
WO2013173789A2 (en) 2012-05-17 2013-11-21 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
US9828602B2 (en) 2012-06-01 2017-11-28 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with fibronectin
US9487780B2 (en) 2012-06-01 2016-11-08 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with fibronectin
KR20240148947A (ko) 2012-07-13 2024-10-11 웨이브 라이프 사이언시스 리미티드 키랄 제어
SG11201500239VA (en) 2012-07-13 2015-03-30 Wave Life Sciences Japan Asymmetric auxiliary group
SG11201500243WA (en) 2012-07-13 2015-04-29 Shin Nippon Biomedical Lab Ltd Chiral nucleic acid adjuvant
EP2895200B1 (en) 2012-09-14 2019-11-06 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
EP2906699A4 (en) 2012-10-11 2016-06-08 Ionis Pharmaceuticals Inc OLIGOMER COMPOUNDS WITH BICYCLIC NUCLEOSIDES AND USES THEREOF
EP4144845B1 (en) * 2012-10-12 2024-04-24 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
PT2929031T (pt) * 2012-12-05 2018-01-19 Alnylam Pharmaceuticals Inc Composições de irna de pcsk9 e métodos de seu uso
CN105008533A (zh) 2013-03-01 2015-10-28 国立大学法人东京医科齿科大学 嵌合单链反义多核苷酸和双链反义试剂
CN114015692B (zh) 2013-03-14 2024-12-31 阿尔尼拉姆医药品有限公司 补体组分C5 iRNA组合物及其使用方法
PE20152002A1 (es) 2013-05-01 2016-01-21 Isis Pharmaceuticals Inc Composiciones y metodos para modular la expresion de ttr y vhb
TW201536329A (zh) 2013-08-09 2015-10-01 Isis Pharmaceuticals Inc 用於調節失養性肌強直蛋白質激酶(dmpk)表現之化合物及方法
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
US20170268000A1 (en) 2013-12-02 2017-09-21 Ionis Pharmaceuticals, Inc. Antisense compounds and uses thereof
EP3095460A4 (en) 2014-01-15 2017-08-23 Shin Nippon Biomedical Laboratories, Ltd. Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent
WO2015108047A1 (ja) 2014-01-15 2015-07-23 株式会社新日本科学 免疫誘導活性を有するキラル核酸アジュバンド及び免疫誘導活性剤
WO2015108048A1 (ja) 2014-01-15 2015-07-23 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
ES2917473T3 (es) 2014-01-16 2022-07-08 Wave Life Sciences Ltd Diseño quiral
EP3119888B1 (en) 2014-03-19 2021-07-28 Ionis Pharmaceuticals, Inc. Compositions for modulating ataxin 2 expression
US10006027B2 (en) 2014-03-19 2018-06-26 Ionis Pharmaceuticals, Inc. Methods for modulating Ataxin 2 expression
SI3757214T1 (sl) 2014-04-01 2022-08-31 Biogen Ma Inc. Sestave za moduliranje izražanja SOD-1
RS59182B1 (sr) 2014-05-01 2019-10-31 Ionis Pharmaceuticals Inc Kompozicije i postupci za modulaciju ekspresije faktora b komplementa
US10472634B2 (en) 2014-06-04 2019-11-12 Ionis Pharmaceuticals, Inc. Antisense compounds targeting apolipoprotein E receptor 2
GB201410693D0 (en) 2014-06-16 2014-07-30 Univ Southampton Splicing modulation
EP3760208B1 (en) 2014-06-25 2024-05-29 The General Hospital Corporation Targeting human satellite ii (hsatii)
US10436802B2 (en) 2014-09-12 2019-10-08 Biogen Ma Inc. Methods for treating spinal muscular atrophy
KR20170058979A (ko) 2014-09-18 2017-05-29 더 유니버시티 오브 브리티쉬 콜롬비아 헌팅턴병 일배체형에 대한 대립 유전자-특이적 치료
AU2015327836B2 (en) 2014-10-03 2021-07-01 Cold Spring Harbor Laboratory Targeted augmentation of nuclear gene output
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
JP2017536119A (ja) * 2014-11-19 2017-12-07 ロシュ イノベーション センター コペンハーゲン エーエス キラルホスホロチオエート連結を含むｌｎａギャップマーオリゴヌクレオチド
EP3234131B1 (en) * 2014-12-16 2020-04-22 Roche Innovation Center Copenhagen A/S Chiral toxicity screening method
US10793855B2 (en) 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
WO2016115490A1 (en) 2015-01-16 2016-07-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of dux4
US11761951B2 (en) 2015-02-04 2023-09-19 Bristol-Myers Squibb Company Methods of selecting therapeutic molecules
UY36550A (es) 2015-02-04 2016-08-31 Roche Innovation Ct Copenhagen As Oligómeros antisentido de tau y sus usos
EP3256591A4 (en) 2015-02-13 2018-08-08 Translate Bio Ma, Inc. Hybrid oligonucleotides and uses thereof
WO2016130963A1 (en) * 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Compositions and methods for modulating rna
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
EP3271460A4 (en) 2015-03-17 2019-03-13 The General Hospital Corporation RNA INTERACTOM OF POLYCOMB REPRESSIVE COMPLEX 1 (PRC1)
WO2016167780A1 (en) 2015-04-16 2016-10-20 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
CN108025089A (zh) 2015-07-22 2018-05-11 波涛生命科学有限公司 寡核苷酸组合物及其方法
US10533175B2 (en) 2015-09-25 2020-01-14 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating Ataxin 3 expression
CN108603230A (zh) 2015-10-09 2018-09-28 南安普敦大学 基因表达的调节与蛋白质表达失调的筛选
WO2017096395A1 (en) 2015-12-04 2017-06-08 Ionis Pharmaceuticals, Inc. Methods of treating breast cancer
EP3390636B1 (en) 2015-12-14 2021-05-19 Cold Spring Harbor Laboratory Antisense oligomers for treatment of dravet syndrome
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
WO2017117496A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
AU2017205462A1 (en) 2016-01-05 2018-06-07 Ionis Pharmaceuticals, Inc. Methods for reducing LRRK2 expression
WO2017142054A1 (ja) 2016-02-17 2017-08-24 国立大学法人東京工業大学 人工ヌクレオシド及び人工ヌクレオチド並びに人工オリゴヌクレオチド
WO2017161168A1 (en) 2016-03-16 2017-09-21 Ionis Pharmaceuticals, Inc. Modulation of dyrk1b expression
US10961271B2 (en) 2016-03-16 2021-03-30 Ionis Pharmaceuticals, Inc. Methods of modulating KEAP1
CN109477108A (zh) 2016-05-04 2019-03-15 波涛生命科学有限公司 寡核苷酸组合物和其方法
CA3023514A1 (en) 2016-06-17 2017-12-21 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
EP3472346B1 (en) 2016-06-17 2023-01-18 F. Hoffmann-La Roche AG Papd5 and papd7 inhibitors for treating a hepatitis b infection
CN109757108A (zh) * 2016-07-05 2019-05-14 比奥马林技术公司 具有治疗遗传疾病的改善特征的包含双环支架部分的前体mRNA剪接转换或调节寡核苷酸
US11118179B2 (en) 2016-09-23 2021-09-14 Synthena Ag Mixed tricyclo-DNA, 2′-modified RNA oligonucleotide compositions and uses thereof
JP7043078B2 (ja) 2016-09-23 2022-03-29 国立大学法人 東京医科歯科大学 血液脳関門通過型ヘテロ２本鎖核酸
WO2018055577A1 (en) * 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
JP7129702B2 (ja) 2016-09-29 2022-09-02 国立大学法人 東京医科歯科大学 オーバーハングを有する二本鎖核酸複合体
KR20190065341A (ko) 2016-10-06 2019-06-11 아이오니스 파마수티컬즈, 인코포레이티드 올리고머 화합물들의 접합 방법
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
WO2018102397A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
WO2018102745A1 (en) 2016-12-02 2018-06-07 Cold Spring Harbor Laboratory Modulation of lnc05 expression
TWI830693B (zh) 2017-02-06 2024-02-01 日商日產化學工業股份有限公司 單股寡核苷酸
US11261440B2 (en) 2017-02-21 2022-03-01 Osaka University Antisense oligonucleic acid
JOP20190215A1 (ar) 2017-03-24 2019-09-19 Ionis Pharmaceuticals Inc مُعدّلات التعبير الوراثي عن pcsk9
WO2019004420A1 (ja) 2017-06-30 2019-01-03 国立大学法人東京医科歯科大学 ヘテロ二本鎖型ａｎｔｉｍｉＲ
CA3072076A1 (en) 2017-08-08 2019-02-14 Wave Life Sciences Ltd. Oligonucleotide compositions and methods thereof
EP3668984A4 (en) 2017-08-18 2021-09-08 Ionis Pharmaceuticals, Inc. MODULATION OF THE NOTCH SIGNALING PATH FOR THE TREATMENT OF RESPIRATORY DISORDERS
EP3673080B1 (en) 2017-08-25 2023-10-18 Stoke Therapeutics, Inc. Antisense oligomers for treatment of conditions and diseases
WO2019051173A1 (en) 2017-09-08 2019-03-14 Ionis Pharmaceuticals, Inc. MODULATORS OF SMAD7 EXPRESSION
EP3694995A1 (en) 2017-10-13 2020-08-19 Roche Innovation Center Copenhagen A/S Methods for identifying improved stereodefined phosphorothioate oligonucleotide variants of antisense oligonucleotides utilising sub-libraries of partially stereodefined oligonucleotides
TW202424191A (zh) 2017-10-16 2024-06-16 瑞士商赫孚孟拉羅股份公司 用於降低PAPD5及PAPD7 mRNA以治療B型肝炎感染之核酸分子
TWI809004B (zh) 2017-11-09 2023-07-21 美商Ionis製藥公司 用於降低snca表現之化合物及方法
CN111344408A (zh) 2017-12-11 2020-06-26 哥本哈根罗氏创新中心 用于调控fndc3b表达的寡核苷酸
WO2019115417A2 (en) 2017-12-12 2019-06-20 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating rb1 expression
CN118980816A (zh) 2017-12-21 2024-11-19 豪夫迈·罗氏有限公司 Htra1 rna拮抗剂的伴随诊断
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
KR20200104302A (ko) 2017-12-22 2020-09-03 로슈 이노베이션 센터 코펜하겐 에이/에스 신규 티오포스포르아미다이트
KR20200104347A (ko) 2017-12-22 2020-09-03 로슈 이노베이션 센터 코펜하겐 에이/에스 포스포로디티오에이트 인터뉴클레오시드 연결을 포함하는 갭머 올리고뉴클레오티드
US20200339982A1 (en) 2017-12-22 2020-10-29 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorodithioate internucleoside linkage
WO2019137883A1 (en) 2018-01-10 2019-07-18 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating pias4 expression
JP2021510295A (ja) 2018-01-12 2021-04-22 ロシュ イノベーション センター コペンハーゲン エーエス Ｇｓｋ３ｂ発現を調節するためのオリゴヌクレオチド
MX2020007369A (es) 2018-01-15 2020-10-28 Ionis Pharmaceuticals Inc Moduladores de la expresion de dnm2.
US20210095276A1 (en) 2018-01-17 2021-04-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating erc1 expression
US20210095277A1 (en) 2018-01-18 2021-04-01 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting srebp1
WO2019145386A1 (en) 2018-01-26 2019-08-01 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating csnk1d expression
KR20200108315A (ko) 2018-02-09 2020-09-17 제넨테크, 인크. Tmem106b의 발현을 조절하기 위한 올리고뉴클레오티드
AU2019218987B2 (en) 2018-02-12 2025-04-24 Ionis Pharmaceuticals, Inc. Modified compounds and uses thereof
US11674141B2 (en) 2018-02-28 2023-06-13 National University Corporation Tokyo Medical And Dental University Ischemic-lesion-site-specific gene therapy
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
WO2019177061A1 (ja) 2018-03-14 2019-09-19 国立大学法人東京医科歯科大学 核酸複合体
JP7333079B2 (ja) 2018-03-19 2023-08-24 国立大学法人 東京医科歯科大学 毒性が軽減した核酸
US20210054377A1 (en) 2018-03-20 2021-02-25 Tokyo Institute Of Technology Antisense oligonucleotide reduced in toxicity
US11661601B2 (en) 2018-03-22 2023-05-30 Ionis Pharmaceuticals, Inc. Methods for modulating FMR1 expression
JP7262816B2 (ja) 2018-03-22 2023-04-24 国立大学法人 東京医科歯科大学 ヘテロ核酸のｂｂｂ通過脂質リガンド
WO2019199720A1 (en) * 2018-04-09 2019-10-17 The Trustees Of Princeton University Compositions comprising immune system activators and method of using same
PE20201349A1 (es) 2018-04-11 2020-11-30 Ionis Pharmaceuticals Inc Moduladores de la expresion de ezh2
JP2021523227A (ja) 2018-05-04 2021-09-02 ストーク セラピューティクス，インク． コレステリルエステル蓄積症の処置のための方法及び組成物
EP3790971A1 (en) 2018-05-08 2021-03-17 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating myh7 expression
SG11202010012PA (en) 2018-05-09 2020-11-27 Ionis Pharmaceuticals Inc Compounds and methods for reducing fxi expression
WO2019217708A1 (en) 2018-05-09 2019-11-14 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
JP7379387B2 (ja) 2018-06-05 2023-11-14 エフ． ホフマン－ラ ロシュ アーゲー Ａｔｘｎ２発現を制御するためのオリゴヌクレオチド
AU2019287635B2 (en) * 2018-06-14 2026-02-12 Ionis Pharmaceuticals, Inc. Compounds and methods for increasing STMN2 expression
TWI833770B (zh) 2018-06-27 2024-03-01 美商Ionis製藥公司 用於減少 lrrk2 表現之化合物及方法
WO2020007702A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting bcl2l11
WO2020007700A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting spi1
WO2020007772A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting gbp-1
PE20220013A1 (es) 2018-07-03 2022-01-11 Hoffmann La Roche Oligonucleotidos para modular la expresion de tau
WO2020007889A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting stat1
WO2020007826A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mbtps1
WO2020011743A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mafb
WO2020011653A1 (en) 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting kynu
WO2020011744A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers5
WO2020011745A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers6
WO2020011869A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting tlr2
BR112021000538A2 (pt) 2018-07-13 2021-04-06 F. Hoffmann-La Roche Ag Oligonucleotídeos para modular a expressão de rtel1
WO2020018558A1 (en) 2018-07-17 2020-01-23 Aronora, Inc. Methods for safely reducing thrombopoietin
CA3106986A1 (en) 2018-07-25 2020-01-30 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn2 expression
WO2020022499A1 (ja) 2018-07-27 2020-01-30 国立大学法人大阪大学 老化の抑制、加齢性の疾患もしくは症状の予防、改善、もしくは治療、または寿命の延長のための組成物
MX2020013270A (es) 2018-07-31 2021-02-18 Roche Innovation Ct Copenhagen As Oligonucleotidos que comprenden enlace internucleosidico de fosforotritioato.
AU2019313527A1 (en) 2018-07-31 2021-02-11 Roche Innovation Center Copenhagen A/S Oligonucleotides comprising a phosphorotrithioate internucleoside linkage
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US12018087B2 (en) 2018-08-02 2024-06-25 Dyne Therapeutics, Inc. Muscle-targeting complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and methods of delivering oligonucleotide to a subject
US12370264B1 (en) 2018-08-02 2025-07-29 Dyne Therapeutics, Inc. Complexes comprising an anti-transferrin receptor antibody linked to an oligonucleotide and method of delivering oligonucleotide to a subject
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
AU2019316103B2 (en) 2018-08-02 2026-02-19 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2020038976A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting usp8
WO2020038973A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting sptlc1
WO2020038971A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting vcan
EP3620519A1 (en) 2018-09-04 2020-03-11 F. Hoffmann-La Roche AG Use of isolated milk extracellular vesicles for delivering oligonucleotides orally
EP3873920A1 (en) 2018-11-01 2021-09-08 F. Hoffmann-La Roche AG Antisense oligonucleotides targeting tia1
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
AU2019380940A1 (en) 2018-11-15 2021-06-03 Ionis Pharmaceuticals, Inc. Modulators of IRF5 expression
EP3884053A4 (en) 2018-11-21 2023-02-01 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR REDUCING PRION EXPRESSION
EP3894559A4 (en) * 2018-12-03 2023-04-05 Triplet Therapeutics, Inc. METHODS OF TREATMENT OF TRINUCLEOTIDE REPEAT EXPANSION DISORDERS RELATED TO MLH3 ACTIVITY
US20220042011A1 (en) 2018-12-21 2022-02-10 Hoffmann-La Roche Inc. Antisense oligonucleotides targeting card9
WO2020152303A1 (en) 2019-01-25 2020-07-30 F. Hoffmann-La Roche Ag Lipid vesicle for oral drug delivery
TW202214854A (zh) 2019-01-31 2022-04-16 美商Ionis製藥公司 Yap1表現之調節劑
CN113490742A (zh) 2019-02-20 2021-10-08 罗氏创新中心哥本哈根有限公司 膦酰基乙酸酯缺口聚物寡核苷酸
BR112021016354A2 (pt) 2019-02-20 2021-10-19 Roche Innovation Center Copenhagen A/S Composto, processo para a fabricação de um composto de fórmula, uso de um composto e invenção
EP3931348B1 (en) 2019-02-26 2023-08-09 Roche Innovation Center Copenhagen A/S Oligonucleotide formulation method
CN113748209A (zh) 2019-02-27 2021-12-03 斯托克制药公司 用于治疗病况和疾病的反义寡聚体
MX2021010152A (es) 2019-02-27 2021-09-14 Ionis Pharmaceuticals Inc Moduladores de la expresion de malat1.
JP7530106B2 (ja) 2019-03-14 2024-08-07 レナセラピューティクス株式会社 Ｉｈｈ発現を調節するための核酸複合体
MD3947684T2 (ro) 2019-03-29 2025-10-31 Ionis Pharmaceuticals Inc Compuși și metode pentru modularea UBE3A-ATS
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
WO2020209285A1 (ja) 2019-04-08 2020-10-15 国立大学法人東京医科歯科大学 筋疾患治療用医薬組成物
CN113950529A (zh) 2019-06-06 2022-01-18 豪夫迈·罗氏有限公司 靶向atxn3的反义寡核苷酸
EP4660307A2 (en) 2019-07-26 2025-12-10 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gfap
EP4032551A4 (en) 2019-09-18 2023-12-20 National University Corporation Tokyo Medical and Dental University NUCLEIC ACID COMPLEX
JP7793144B2 (ja) 2019-10-11 2026-01-05 国立大学法人東京科学大学 修飾ヘテロ核酸
KR20220079832A (ko) 2019-10-11 2022-06-14 스미또모 가가꾸 가부시끼가이샤 핵산 올리고머의 제조 방법
US20220251557A1 (en) * 2019-11-27 2022-08-11 Tokyo Institute Of Technology Method for reducing toxicity of antisense nucleic acids
EP4706689A2 (en) 2019-12-19 2026-03-11 Entrada Therapeutics, Inc. Compositions for delivery of antisense compounds
EP4081217A1 (en) 2019-12-24 2022-11-02 F. Hoffmann-La Roche AG Pharmaceutical combination of antiviral agents targeting hbv and/or an immune modulator for treatment of hbv
EP4081639A1 (en) 2019-12-24 2022-11-02 F. Hoffmann-La Roche AG Pharmaceutical combination of a therapeutic oligonucleotide targeting hbv and a tlr7 agonist for treatment of hbv
EP4097234A1 (en) 2020-01-28 2022-12-07 Université de Strasbourg Antisense oligonucleotide targeting linc00518 for treating melanoma
JP7667788B2 (ja) 2020-01-29 2025-04-23 住友化学株式会社 核酸オリゴマーの製造方法
US20230312635A1 (en) 2020-01-29 2023-10-05 Sumitomo Chemical Company, Limited Method for producing nucleic acid oligomer
AU2021212949A1 (en) 2020-01-31 2022-09-01 Nissan Chemical Corporation Antisense oligonucleotide of ATN1
CN120505310A (zh) 2020-02-28 2025-08-19 Ionis制药公司 用于调节smn2的化合物和方法
EP4116419A1 (en) 2020-03-04 2023-01-11 Nissan Chemical Corporation Antisense oligonucleotide of calm2
CN115335522A (zh) * 2020-03-05 2022-11-11 施能康公司 用于降低apoe表达的化合物和方法
WO2021187392A1 (ja) 2020-03-16 2021-09-23 国立大学法人東京医科歯科大学 モルホリノ核酸を含むヘテロ核酸
WO2021193954A1 (ja) 2020-03-27 2021-09-30 住友化学株式会社 核酸オリゴマーの製造方法
US20230151369A1 (en) * 2020-04-17 2023-05-18 The University Of British Columbia Compositions and methods for inhibiting tdp-43 and fus aggregation
IL297435A (en) 2020-05-01 2022-12-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating atxn1
AU2021270720A1 (en) 2020-05-11 2022-12-08 Stoke Therapeutics, Inc. OPA1 antisense oligomers for treatment of conditions and diseases
JP7799640B2 (ja) 2020-06-29 2026-01-15 アイオーニス ファーマシューティカルズ， インコーポレーテッド Ｐｌｐ１を調節するための化合物及び方法
WO2022009959A1 (ja) 2020-07-09 2022-01-13 住友化学株式会社 核酸オリゴマーの製造方法
US12384814B2 (en) 2020-07-28 2025-08-12 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
PE20231567A1 (es) 2020-08-07 2023-10-04 Ionis Pharmaceuticals Inc Compuestos y metodos para modular el scn2a
CN116209668A (zh) 2020-09-24 2023-06-02 住友化学株式会社 核酸寡聚物的制造方法
MX2023005736A (es) 2020-11-18 2023-05-25 Ionis Pharmaceuticals Inc Compuestos y metodos para modular la expresion de angiotensinogeno.
AR124227A1 (es) 2020-12-03 2023-03-01 Hoffmann La Roche Oligonucleótidos antisentido que actúan sobre atxn3
WO2022117745A1 (en) 2020-12-03 2022-06-09 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
CA3205040A1 (en) 2020-12-18 2022-06-23 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating factor xii
KR20230128050A (ko) 2020-12-31 2023-09-01 다인 세라퓨틱스, 인크. 근육 표적화 복합체 및 근긴장성 이영양증을 치료하기위한 그의 용도
US20240083990A1 (en) 2021-01-26 2024-03-14 Universite Brest Bretagne Occidentale Novel stim1 splicing variants and uses thereof
JP2024506709A (ja) * 2021-02-17 2024-02-14 キュー－ステート バイオサイエンシーズ， インコーポレイテッド Ｕｂｅ３ａアンチセンス治療剤
US20220370487A1 (en) * 2021-05-10 2022-11-24 Q-State Biosciences, Inc. Compositions targeting sodium channel 1.6
AU2022271873A1 (en) 2021-05-10 2024-01-04 Entrada Therapeutics, Inc. Compositions and methods for intracellular therapeutics
EP4349986A4 (en) 2021-05-25 2025-11-19 Univ Nat Corp Tokyo Medical & Dental HETERONUCLEIC ACID WITH A CONTENT OF ?????? OR ????
KR20240016342A (ko) 2021-05-31 2024-02-06 레나 테라퓨틱스 인크. 리간드-결합 핵산 복합체
KR20240023602A (ko) 2021-06-18 2024-02-22 아이오니스 파마수티컬즈, 인코포레이티드 Ifnar1 발현을 감소시키기 위한 화합물 및 방법
WO2022271818A1 (en) 2021-06-23 2022-12-29 Entrada Therapeutics, Inc. Antisense compounds and methods for targeting cug repeats
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
US11633498B2 (en) 2021-07-09 2023-04-25 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
US11969475B2 (en) 2021-07-09 2024-04-30 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
WO2023022229A1 (ja) 2021-08-19 2023-02-23 国立大学法人東京医科歯科大学 モルホリノ核酸を含む修飾ヘテロ核酸
US20240390508A1 (en) 2021-08-21 2024-11-28 Takeda Pharmaceutical Company Limited Human transferrin receptor binding peptide-drug conjugate
EP4396352A4 (en) 2021-09-01 2025-10-01 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION
WO2023042876A1 (ja) 2021-09-15 2023-03-23 国立大学法人東京医科歯科大学 ２'－修飾ヌクレオシドを含むヘテロ核酸
JPWO2023054350A1 (enExample) 2021-09-28 2023-04-06
WO2023080159A1 (ja) 2021-11-02 2023-05-11 レナセラピューティクス株式会社 リガンド結合核酸複合体
CN118318042A (zh) 2021-11-03 2024-07-09 豪夫迈·罗氏有限公司 用于调节载脂蛋白e4表达的寡核苷酸
IL312635A (en) 2021-11-11 2024-07-01 Hoffmann La Roche Pharmaceutical combinations for treatment of hbv
EP4453206A1 (en) 2021-12-20 2024-10-30 F. Hoffmann-La Roche AG Threose nucleic acid antisense oligonucleotides and methods thereof
TW202340468A (zh) 2022-01-10 2023-10-16 中國大陸商杭州浩博醫藥有限公司 B型肝炎病毒(hbv)表現之調節
CN118613268A (zh) 2022-01-20 2024-09-06 基因泰克公司 用于调节tmem106b表达的反义寡核苷酸
US11879125B2 (en) 2022-03-16 2024-01-23 Empirico Inc. GalNAc compositions for improving siRNA bioavailability
JPWO2023182274A1 (enExample) 2022-03-23 2023-09-28
AU2023245603A1 (en) 2022-03-28 2024-11-07 Empirico Inc. Modified oligonucleotides
IL316143A (en) 2022-04-15 2024-12-01 Dyne Therapeutics Inc Muscle targeting complexes and formulations for the treatment of myotonic dystrophy
EP4559925A1 (en) 2022-07-22 2025-05-28 Sumitomo Chemical Company, Limited Production method of oligonucleotide
JPWO2024024873A1 (enExample) 2022-07-28 2024-02-01
AU2023314808A1 (en) 2022-07-29 2025-03-20 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
EP4581143A1 (en) 2022-08-29 2025-07-09 University of Rochester Antisense oligonucleotide-based anti-fibrotic therapeutics
EP4332221A1 (en) 2022-08-29 2024-03-06 Roche Innovation Center Copenhagen A/S Threose nucleic acid antisense oligonucleotides and methods thereof
US12152052B2 (en) 2022-09-23 2024-11-26 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing MECP2 expression
WO2024073386A1 (en) * 2022-09-29 2024-04-04 Celanese Eva Performance Polymers Llc Implantable medical device for the delivery of a nucleic acid
WO2024071099A1 (ja) 2022-09-29 2024-04-04 国立大学法人東京医科歯科大学 ５'－シクロプロピレン修飾を含む核酸分子
KR20250092173A (ko) 2022-10-27 2025-06-23 스미또모 가가꾸 가부시끼가이샤 올리고뉴클레오티드의 제조 방법
EP4612184A1 (en) 2022-11-04 2025-09-10 Regeneron Pharmaceuticals, Inc. Calcium voltage-gated channel auxiliary subunit gamma 1 (cacng1) binding proteins and cacng1-mediated delivery to skeletal muscle
WO2024107765A2 (en) 2022-11-14 2024-05-23 Regeneron Pharmaceuticals, Inc. Compositions and methods for fibroblast growth factor receptor 3-mediated delivery to astrocytes
KR20250128304A (ko) 2022-12-26 2025-08-27 스미또모 가가꾸 가부시끼가이샤 올리고뉴클레오티드의 제조 방법
WO2024153586A1 (en) 2023-01-16 2024-07-25 Institut National De Recherche Pour L'agriculture, L'alimentation Et L'environnement Antisense molecules and their uses for the treatment of coronavirus infection, in particular the treatment of covid-19 related to sars-cov-2 infection
WO2024192115A1 (en) * 2023-03-14 2024-09-19 Thomas Jefferson University Compositions and methods for the treatment of cancer

Family Cites Families (84)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5118800A (en) 1983-12-20 1992-06-02 California Institute Of Technology Oligonucleotides possessing a primary amino group in the terminal nucleotide
FR2567892B1 (fr) 1984-07-19 1989-02-17 Centre Nat Rech Scient Nouveaux oligonucleotides, leur procede de preparation et leurs applications comme mediateurs dans le developpement des effets des interferons
US5591722A (en) 1989-09-15 1997-01-07 Southern Research Institute 2'-deoxy-4'-thioribonucleosides and their antiviral activity
EP0497875B1 (en) 1989-10-24 2000-03-22 Isis Pharmaceuticals, Inc. 2' modified oligonucleotides
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
GB9009980D0 (en) * 1990-05-03 1990-06-27 Amersham Int Plc Phosphoramidite derivatives,their preparation and the use thereof in the incorporation of reporter groups on synthetic oligonucleotides
DE69032425T2 (de) 1990-05-11 1998-11-26 Microprobe Corp., Bothell, Wash. Teststreifen zum Eintauchen für Nukleinsäure-Hybridisierungsassays und Verfahren zur kovalenten Immobilisierung von Oligonucleotiden
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
ES2103918T3 (es) 1991-10-17 1997-10-01 Ciba Geigy Ag Nucleosidos biciclicos, oligonucleotidos, procedimiento para su obtencion y productos intermedios.
US5359044A (en) 1991-12-13 1994-10-25 Isis Pharmaceuticals Cyclobutyl oligonucleotide surrogates
US20010044145A1 (en) † 1991-12-24 2001-11-22 Monia Brett P. Methods of using mammalian RNase H and compositions thereof
FR2687679B1 (fr) 1992-02-05 1994-10-28 Centre Nat Rech Scient Oligothionucleotides.
FR2692265B1 (fr) 1992-05-25 1996-11-08 Centre Nat Rech Scient Composes biologiquement actifs de type phosphotriesters.
EP0577558A2 (de) 1992-07-01 1994-01-05 Ciba-Geigy Ag Carbocyclische Nukleoside mit bicyclischen Ringen, Oligonukleotide daraus, Verfahren zu deren Herstellung, deren Verwendung und Zwischenproduckte
EP0673559A1 (en) 1992-12-14 1995-09-27 Honeywell Inc. Motor system with individually controlled redundant windings
MX9306994A (es) 1992-12-15 1994-06-30 Ericsson Telefon Ab L M Sistema de control de flujo para interruptores de paquete.
US5446786A (en) * 1993-03-03 1995-08-29 Northern Telecom Limited Two-wire telecommunications line detection arrangements
AU6449394A (en) * 1993-03-30 1994-10-24 Sterling Winthrop Inc. Acyclic nucleoside analogs and oligonucleotide sequences containing them
DE4311944A1 (de) 1993-04-10 1994-10-13 Degussa Umhüllte Natriumpercarbonatpartikel, Verfahren zu deren Herstellung und sie enthaltende Wasch-, Reinigungs- und Bleichmittelzusammensetzungen
FR2705099B1 (fr) 1993-05-12 1995-08-04 Centre Nat Rech Scient Oligonucléotides phosphorothioates triesters et procédé de préparation.
US5801154A (en) 1993-10-18 1998-09-01 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of multidrug resistance-associated protein
US5446137B1 (en) * 1993-12-09 1998-10-06 Behringwerke Ag Oligonucleotides containing 4'-substituted nucleotides
US5519134A (en) 1994-01-11 1996-05-21 Isis Pharmaceuticals, Inc. Pyrrolidine-containing monomers and oligomers
US5627053A (en) 1994-03-29 1997-05-06 Ribozyme Pharmaceuticals, Inc. 2'deoxy-2'-alkylnucleotide containing nucleic acid
US5597909A (en) 1994-08-25 1997-01-28 Chiron Corporation Polynucleotide reagents containing modified deoxyribose moieties, and associated methods of synthesis and use
US5792747A (en) 1995-01-24 1998-08-11 The Administrators Of The Tulane Educational Fund Highly potent agonists of growth hormone releasing hormone
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
DE69829760T3 (de) 1997-09-12 2016-04-14 Exiqon A/S Bi- und tri-zyklische - nukleosid, nukleotid und oligonukleotid-analoga
US6794499B2 (en) 1997-09-12 2004-09-21 Exiqon A/S Oligonucleotide analogues
US20030228597A1 (en) 1998-04-13 2003-12-11 Cowsert Lex M. Identification of genetic targets for modulation by oligonucleotides and generation of oligonucleotides for gene modulation
US6043352A (en) 1998-08-07 2000-03-28 Isis Pharmaceuticals, Inc. 2'-O-Dimethylaminoethyloxyethyl-modified oligonucleotides
KR100573231B1 (ko) 1999-02-12 2006-04-24 상꾜 가부시키가이샤 신규 뉴클레오시드 및 올리고뉴클레오티드 유사체
US7084125B2 (en) 1999-03-18 2006-08-01 Exiqon A/S Xylo-LNA analogues
US7098192B2 (en) * 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
AU776362B2 (en) 1999-05-04 2004-09-09 Roche Innovation Center Copenhagen A/S L-ribo-LNA analogues
US6525191B1 (en) 1999-05-11 2003-02-25 Kanda S. Ramasamy Conformationally constrained L-nucleosides
US20040002153A1 (en) 1999-07-21 2004-01-01 Monia Brett P. Modulation of PTEN expression via oligomeric compounds
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
WO2002094250A2 (en) * 2001-05-18 2002-11-28 Cureon A/S Therapeutic uses of lna-modified oligonucleotides in infectious diseases
US7888324B2 (en) * 2001-08-01 2011-02-15 Genzyme Corporation Antisense modulation of apolipoprotein B expression
US7407943B2 (en) * 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US7569575B2 (en) 2002-05-08 2009-08-04 Santaris Pharma A/S Synthesis of locked nucleic acid derivatives
US20040219565A1 (en) 2002-10-21 2004-11-04 Sakari Kauppinen Oligonucleotides useful for detecting and analyzing nucleic acids of interest
AU2003295387A1 (en) 2002-11-05 2004-06-03 Isis Parmaceuticals, Inc. Modified oligonucleotides for use in rna interference
EP1562971B1 (en) 2002-11-05 2014-02-12 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
JP4986109B2 (ja) * 2002-11-13 2012-07-25 ジェンザイム・コーポレーション アポリポタンパク質ｂ発現のアンチセンス調節
EP2752488B1 (en) † 2002-11-18 2020-02-12 Roche Innovation Center Copenhagen A/S Antisense design
DE602004022020D1 (de) * 2003-02-10 2009-08-27 Santaris Pharma As Oligomere verbindungen zur modulation der expression von survivin
WO2004106356A1 (en) 2003-05-27 2004-12-09 Syddansk Universitet Functionalized nucleotide derivatives
US7457046B2 (en) * 2003-07-01 2008-11-25 Canon Kabushiki Kaisha Zoom lens system and image-taking apparatus
DK1661905T3 (da) 2003-08-28 2012-07-23 Takeshi Imanishi Hidtil ukendte syntetiske nukleinsyrer af N-O-krydsbindingstype
US20050053981A1 (en) * 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
US20050074801A1 (en) * 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
NZ545134A (en) * 2003-09-18 2009-06-26 Lilly Co Eli Modulation of eIF4E expression
NZ548254A (en) † 2003-12-23 2008-09-26 Santaris Pharma As Oligomeric compounds for the modulation of BCL-2
GB0407382D0 (en) * 2004-03-31 2004-05-05 Univ Cambridge Tech Therapeutic methods and means
CA2569419A1 (en) 2004-06-03 2005-12-22 Isis Pharmaceuticals, Inc. Double strand compositions comprising differentially modified strands for use in gene modulation
JP5523705B2 (ja) * 2005-08-29 2014-06-18 レグルス・セラピューティクス・インコーポレイテッド Ｍｉｒ−１２２ａをモジュレートする使用方法
US20090203893A1 (en) 2005-08-29 2009-08-13 Regulus Therapeutics, Llc Antisense compounds having enhanced anti-microrna activity
JP5425474B2 (ja) 2006-01-26 2014-02-26 アイシス ファーマシューティカルズ， インコーポレーテッド ハンチンチン対する、組成物及びその使用
CA2640171C (en) 2006-01-27 2014-10-28 Isis Pharmaceuticals, Inc. 6-modified bicyclic nucleic acid analogs
CA3044969A1 (en) * 2006-05-05 2007-12-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating gene expression
JP5441688B2 (ja) * 2006-05-11 2014-03-12 アイシス ファーマシューティカルズ， インコーポレーテッド ５’修飾二環式核酸類似体
DK2092065T4 (da) * 2006-10-18 2019-10-21 Ionis Pharmaceuticals Inc Antisense-forbindelser
WO2008111908A1 (en) 2007-03-15 2008-09-18 Jyoti Chattopadhyaya Five- and six-membered conformationally locked 2',4'- carbocyclic ribo-thymidines for the treatment of infections and cancer
EP2170917B1 (en) † 2007-05-30 2012-06-27 Isis Pharmaceuticals, Inc. N-substituted-aminomethylene bridged bicyclic nucleic acid analogs
ES2386492T3 (es) † 2007-06-08 2012-08-21 Isis Pharmaceuticals, Inc. Análogos de ácidos nucleicos bicíclicos carbocíclicos
JP5572090B2 (ja) † 2007-08-15 2014-08-13 アイシス ファーマシューティカルズ， インコーポレーテッド テトラヒドロピラン核酸類似体
US8389488B2 (en) 2007-11-05 2013-03-05 Isis Pharmaceuticals, Inc. Antidotes to antisense compounds
WO2009067647A1 (en) 2007-11-21 2009-05-28 Isis Pharmaceuticals, Inc. Carbocyclic alpha-l-bicyclic nucleic acid analogs
EP2265627A2 (en) 2008-02-07 2010-12-29 Isis Pharmaceuticals, Inc. Bicyclic cyclohexitol nucleic acid analogs
EP2274423A2 (en) 2008-04-04 2011-01-19 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleosides and having reduced toxicity
DK2356129T3 (da) 2008-09-24 2013-05-13 Isis Pharmaceuticals Inc Substituerede alpha-L-bicykliske nukleosider
WO2010108035A1 (en) 2009-03-18 2010-09-23 Isis Pharmaceuticals, Inc. Compounds and methods for modulating toxic and proinflammatory effects
WO2011017521A2 (en) 2009-08-06 2011-02-10 Isis Pharmaceuticals, Inc. Bicyclic cyclohexose nucleic acid analogs
WO2012027033A1 (en) 2010-07-19 2012-03-01 Isis Pharmaceuticals, Inc. Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals
EP3467109A1 (en) 2011-02-08 2019-04-10 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
MX347253B (es) 2011-04-21 2017-04-20 Ionis Pharmaceuticals Inc Modulación de la expresión del virus de hepatitis b (vhb).
EP2742056B2 (en) 2011-08-11 2020-06-10 Ionis Pharmaceuticals, Inc. Selective antisense compounds and uses thereof
WO2013159108A2 (en) * 2012-04-20 2013-10-24 Isis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
EP2906699A4 (en) * 2012-10-11 2016-06-08 Ionis Pharmaceuticals Inc OLIGOMER COMPOUNDS WITH BICYCLIC NUCLEOSIDES AND USES THEREOF
CN112359042A (zh) † 2013-09-13 2021-02-12 Ionis制药公司 补体因子b的调节剂

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2014221817A (ja) * 2006-10-18 2014-11-27 アイシス ファーマシューティカルズ， インコーポレーテッド アンチセンス化合物

Also Published As

Publication number Publication date
DK2410053T3 (en) 2014-12-08
DK2410054T4 (da) 2020-02-10
CA2667055C (en) 2017-05-09
EP2410054B1 (en) 2017-01-25
EP2410053B1 (en) 2014-10-01
US9550988B2 (en) 2017-01-24
AU2007310989A1 (en) 2008-04-24
JP2014221817A (ja) 2014-11-27
AU2007310989B2 (en) 2014-05-29
ATE540118T1 (de) 2012-01-15
ES2526295T3 (es) 2015-01-09
JP2010507579A (ja) 2010-03-11
EP2092065B2 (en) 2019-07-24
AU2014203243A1 (en) 2014-07-10
EP2092065A1 (en) 2009-08-26
EP2092065B1 (en) 2012-01-04
WO2008049085A1 (en) 2008-04-24
ES2620472T5 (es) 2020-07-09
AU2016231660A1 (en) 2016-10-20
US20100197762A1 (en) 2010-08-05
EP2410054B2 (en) 2019-12-11
ES2526295T5 (es) 2021-05-04
US20240058370A1 (en) 2024-02-22
EP2410053A1 (en) 2012-01-25
ES2377327T3 (es) 2012-03-26
HK1166643A1 (en) 2012-11-02
DK2410053T4 (da) 2020-08-31
DK2092065T3 (da) 2012-04-02
US20170182082A1 (en) 2017-06-29
EP2410054A1 (en) 2012-01-25
ES2620472T3 (es) 2017-06-28
EP2410053B2 (en) 2020-07-15
EP3916095A1 (en) 2021-12-01
US10493092B2 (en) 2019-12-03
CA2667055A1 (en) 2008-04-24
AU2014203243B2 (en) 2016-06-30
US20200276221A1 (en) 2020-09-03
EP3202905A1 (en) 2017-08-09
DK2410054T3 (en) 2017-04-10
ES2377327T5 (es) 2020-04-28
DK2092065T4 (da) 2019-10-21

Similar Documents

Publication Publication Date Title
JP5665317B2 (ja) アンチセンス化合物
US12480126B2 (en) Modulation of dystrophia myotonica-protein kinase (DMPK) expression
US11981897B2 (en) Compounds and methods for modulation of dystrophia myotonica-protein kinase (DMPK) expression
HK1166643B (en) Antisense compounds

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20101012

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20101012

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20130313

A602 Written permission of extension of time

Free format text: JAPANESE INTERMEDIATE CODE: A602

Effective date: 20130613

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20130913

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20140324

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20140724

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A821

Effective date: 20140911

A911 Transfer to examiner for re-examination before appeal (zenchi)

Free format text: JAPANESE INTERMEDIATE CODE: A911

Effective date: 20141006

TRDD Decision of grant or rejection written
A01 Written decision to grant a patent or to grant a registration (utility model)

Free format text: JAPANESE INTERMEDIATE CODE: A01

Effective date: 20141110

A61 First payment of annual fees (during grant procedure)

Free format text: JAPANESE INTERMEDIATE CODE: A61

Effective date: 20141209

R150 Certificate of patent or registration of utility model

Ref document number: 5665317

Country of ref document: JP

Free format text: JAPANESE INTERMEDIATE CODE: R150

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250

R250 Receipt of annual fees

Free format text: JAPANESE INTERMEDIATE CODE: R250