AU2007310989B2 - Antisense compounds - Google Patents

Antisense compounds Download PDF

Info

Publication number
AU2007310989B2
AU2007310989B2 AU2007310989A AU2007310989A AU2007310989B2 AU 2007310989 B2 AU2007310989 B2 AU 2007310989B2 AU 2007310989 A AU2007310989 A AU 2007310989A AU 2007310989 A AU2007310989 A AU 2007310989A AU 2007310989 B2 AU2007310989 B2 AU 2007310989B2
Authority
AU
Australia
Prior art keywords
wing
nucleoside
antisense oligonucleotide
bicyclic
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
AU2007310989A
Other languages
English (en)
Other versions
AU2007310989A1 (en
Inventor
Eric E. Swayze
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
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=AU2007310989(B2) "Global patent litigation dataset” by Darts-ip is licensed under a Creative Commons Attribution 4.0 International License.
Application filed by Isis Pharmaceuticals Inc filed Critical Isis Pharmaceuticals Inc
Publication of AU2007310989A1 publication Critical patent/AU2007310989A1/en
Application granted granted Critical
Publication of AU2007310989B2 publication Critical patent/AU2007310989B2/en
Priority to AU2014203243A priority Critical patent/AU2014203243B2/en
Assigned to ISIS PHARMACEUTICALS, INC. reassignment ISIS PHARMACEUTICALS, INC. Amend patent request/document other than specification (104) Assignors: ISIS PHARMACEUTICALS, INC.
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)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Epidemiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (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)
AU2007310989A 2006-10-18 2007-10-18 Antisense compounds Active AU2007310989B2 (en)

Priority Applications (1)

Application Number Priority Date Filing Date Title
AU2014203243A AU2014203243B2 (en) 2006-10-18 2014-06-16 Antisense compounds

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
AU2014203243A Division AU2014203243B2 (en) 2006-10-18 2014-06-16 Antisense compounds

Publications (2)

Publication Number Publication Date
AU2007310989A1 AU2007310989A1 (en) 2008-04-24
AU2007310989B2 true AU2007310989B2 (en) 2014-05-29

Family

ID=39052713

Family Applications (3)

Application Number Title Priority Date Filing Date
AU2007310989A Active AU2007310989B2 (en) 2006-10-18 2007-10-18 Antisense compounds
AU2014203243A Active AU2014203243B2 (en) 2006-10-18 2014-06-16 Antisense compounds
AU2016231660A Abandoned AU2016231660A1 (en) 2006-10-18 2016-09-27 Antisense compounds

Family Applications After (2)

Application Number Title Priority Date Filing Date
AU2014203243A Active AU2014203243B2 (en) 2006-10-18 2014-06-16 Antisense compounds
AU2016231660A Abandoned AU2016231660A1 (en) 2006-10-18 2016-09-27 Antisense compounds

Country Status (9)

Country Link
US (4) US9550988B2 (enExample)
EP (5) EP2410054B2 (enExample)
JP (2) JP5665317B2 (enExample)
AT (1) ATE540118T1 (enExample)
AU (3) AU2007310989B2 (enExample)
CA (1) CA2667055C (enExample)
DK (3) DK2410053T4 (enExample)
ES (3) ES2526295T5 (enExample)
WO (1) WO2008049085A1 (enExample)

Families Citing this family (250)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
DK2363481T3 (en) 2006-05-05 2017-06-26 Ionis Pharmaceuticals Inc Compounds and methods for modulating gene expression
AU2007310989B2 (en) * 2006-10-18 2014-05-29 Isis Pharmaceuticals, Inc. Antisense compounds
US8580756B2 (en) * 2007-03-22 2013-11-12 Santaris Pharma A/S Short oligomer antagonist compounds for the modulation of target mRNA
WO2009046426A2 (en) * 2007-10-04 2009-04-09 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
DK2379084T3 (en) 2008-10-15 2018-01-22 Ionis Pharmaceuticals Inc MODULATION OF FACTOR 11 EXPRESSION
SG171914A1 (en) 2008-12-02 2011-07-28 Chiralgen Ltd Method for the synthesis of phosphorus atom modified nucleic acids
US9744183B2 (en) 2009-07-06 2017-08-29 Wave Life Sciences Ltd. Nucleic acid prodrugs and methods of use thereof
WO2011097643A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
CA2789038A1 (en) 2010-02-08 2011-08-11 Isis Pharmaceuticals, Inc. Selective reduction of allelic variants
EP3031920B1 (en) * 2010-07-19 2019-08-21 Ionis Pharmaceuticals, Inc. Modulation of dystrophia myotonica-protein kinase (dmpk) expression
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
EP3702460A1 (en) 2010-11-12 2020-09-02 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
AU2011329777B2 (en) 2010-11-17 2016-06-09 Ionis 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
SI3505528T1 (sl) 2011-04-21 2021-04-30 Glaxo Group Limited Modulacija izražanja virusa hepatitisa B (HBV)
WO2012151324A1 (en) 2011-05-02 2012-11-08 Isis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with usher syndrome
RU2014105311A (ru) 2011-07-19 2015-08-27 Уэйв Лайф Сайенсес Пте. Лтд. Способы синтеза функционализованных нуклеиновых кислот
DK2742135T4 (da) 2011-08-11 2020-07-13 Ionis Pharmaceuticals Inc Bindingsmodificerede gapped oligomeriske forbindelser og anvendelser deraf
EP2756080B1 (en) 2011-09-14 2019-02-20 Translate Bio MA, Inc. Multimeric oligonucleotide compounds
US9243291B1 (en) * 2011-12-01 2016-01-26 Isis Pharmaceuticals, Inc. Methods of predicting toxicity
CN104254610A (zh) * 2012-03-12 2014-12-31 桑塔里斯制药公司 调节atxn3表达的组合物和方法
EP3336189A1 (en) * 2012-04-20 2018-06-20 Ionis Pharmaceuticals, Inc. Oligomeric compounds comprising bicyclic nucleotides and uses thereof
WO2013173599A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating hemoglobin gene family expression
AU2013262649A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating smn gene family expression
EA201492116A1 (ru) 2012-05-16 2015-05-29 Рана Терапьютикс, Инк. Композиции и способы для модулирования экспрессии mecp2
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
EP2850182A4 (en) 2012-05-16 2016-01-20 Rana Therapeutics Inc COMPOSITIONS AND METHODS FOR MODULATING ATP2A2 GENE EXPRESSION
AU2013262656A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating UTRN expression
WO2013173605A1 (en) * 2012-05-16 2013-11-21 Rana Therapeutics, Inc. Compositions and methods for modulating pten expression
US20160002624A1 (en) 2012-05-17 2016-01-07 Isis Pharmaceuticals, Inc. Antisense oligonucleotide compositions
US9574193B2 (en) 2012-05-17 2017-02-21 Ionis Pharmaceuticals, Inc. Methods and compositions for modulating apolipoprotein (a) expression
KR20170142997A (ko) 2012-05-24 2017-12-28 아이오니스 파마수티컬즈, 인코포레이티드 아포지질단백질 (a) 발현을 조절하는 방법들 및 조성물들
US9487780B2 (en) 2012-06-01 2016-11-08 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with fibronectin
US9828602B2 (en) 2012-06-01 2017-11-28 Ionis Pharmaceuticals, Inc. Antisense compounds targeting genes associated with fibronectin
PT2872485T (pt) 2012-07-13 2021-03-05 Wave Life Sciences Ltd Grupo auxiliar assimétrico
BR112015000723A2 (pt) 2012-07-13 2017-06-27 Shin Nippon Biomedical Laboratories Ltd adjuvante de ácido nucléico quiral
KR102712879B1 (ko) 2012-07-13 2024-10-04 웨이브 라이프 사이언시스 리미티드 키랄 제어
CA2884608C (en) 2012-09-14 2025-05-20 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
WO2014059341A2 (en) 2012-10-12 2014-04-17 Isis Pharmaceuticals, Inc. Antisense compounds and uses thereof
MX367076B (es) * 2012-12-05 2019-08-05 Alnylam Pharmaceuticals Inc Composiciones de arni de proteina convertasa subtilisina kexina 9 (pcsk9) y metodos de uso de las mismas.
WO2014132671A1 (en) * 2013-03-01 2014-09-04 National University Corporation Tokyo Medical And Dental University Chimeric single-stranded antisense polynucleotides and double-stranded antisense agent
SG10201700239UA (en) * 2013-03-14 2017-02-27 Alnylam Pharmaceuticals Inc COMPLEMENT COMPONENT C5 iRNA COMPOSITIONS AND METHODS OF USE THEREOF
PL2992098T3 (pl) 2013-05-01 2019-09-30 Ionis Pharmaceuticals, Inc. Kompozycje i sposoby modulowania ekspresji hbv i ttr
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
WO2015084884A2 (en) 2013-12-02 2015-06-11 Isis Pharmaceuticals, Inc. Antisense compounds and uses thereof
JPWO2015108047A1 (ja) 2014-01-15 2017-03-23 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
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
JPWO2015108046A1 (ja) 2014-01-15 2017-03-23 株式会社新日本科学 抗アレルギー作用を有するキラル核酸アジュバンド及び抗アレルギー剤
PT3094728T (pt) 2014-01-16 2022-05-19 Wave Life Sciences Ltd Desenho 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
WO2015153800A2 (en) 2014-04-01 2015-10-08 Isis Pharmaceuticals, Inc. Compositions for modulating sod-1 expression
KR102369736B1 (ko) 2014-05-01 2022-03-02 아이오니스 파마수티컬즈, 인코포레이티드 보체 인자 b 발현을 조절하기 위한 조성물 및 방법
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)
WO2016040748A1 (en) 2014-09-12 2016-03-17 Ionis Pharmaceuticals, Inc. Compositions and methods for detection of smn protein in a subject and treatment of a subject
WO2016041058A1 (en) 2014-09-18 2016-03-24 The University Of British Columbia Allele-specific therapy for huntington disease haplotypes
JP6867945B2 (ja) 2014-10-03 2021-05-12 コールド スプリング ハーバー ラボラトリー 核内遺伝子出力の標的とされた増強
CA2966044A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
JP2017536366A (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 Ｌｎａキラルホスホロチオエート
EP3234131B1 (en) 2014-12-16 2020-04-22 Roche Innovation Center Copenhagen A/S Chiral toxicity screening method
WO2016112132A1 (en) 2015-01-06 2016-07-14 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of c9orf72 antisense transcript
US10538763B2 (en) 2015-01-16 2020-01-21 Ionis Pharmaceuticals, Inc. Compounds and methods for modulation of DUX4
ES2893114T3 (es) 2015-02-04 2022-02-08 Bristol Myers Squibb Co Métodos para seleccionar moléculas terapéuticas
US11077132B2 (en) * 2015-02-04 2021-08-03 F. Hoffmann-La Roche Ag Tau antisense oligomers and uses thereof
US20180055869A1 (en) * 2015-02-13 2018-03-01 Translate Bio Ma, Inc. Compositions and methods for modulating rna
WO2016130943A1 (en) 2015-02-13 2016-08-18 Rana Therapeutics, Inc. Hybrid oligonucleotides and uses thereof
US11129844B2 (en) 2015-03-03 2021-09-28 Ionis Pharmaceuticals, Inc. Compositions and methods for modulating MECP2 expression
US10900036B2 (en) 2015-03-17 2021-01-26 The General Hospital Corporation RNA interactome 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
MA43072A (fr) 2015-07-22 2018-05-30 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
PT3353303T (pt) 2015-09-25 2023-10-10 Academisch Ziekenhuis Leiden Composições e métodos para modulação da expressão de ataxina 3
EP3359685A1 (en) 2015-10-09 2018-08-15 University Of Southampton Modulation of gene expression and screening for deregulated protein expression
EP3389670A4 (en) 2015-12-04 2020-01-08 Ionis Pharmaceuticals, Inc. METHODS OF TREATING BREAST CANCER
US11096956B2 (en) 2015-12-14 2021-08-24 Stoke Therapeutics, Inc. Antisense oligomers and uses thereof
US11083745B2 (en) 2015-12-14 2021-08-10 Cold Spring Harbor Laboratory Antisense oligomers for treatment of autosomal dominant mental retardation-5 and Dravet Syndrome
US20190002887A1 (en) 2015-12-31 2019-01-03 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
EP3400300A4 (en) 2016-01-05 2019-08-07 Ionis Pharmaceuticals, Inc. PROCESS 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
EP3429690A4 (en) 2016-03-16 2019-10-23 Ionis Pharmaceuticals, Inc. METHOD FOR MODULATING KEAP1
MA45270A (fr) 2016-05-04 2017-11-09 Wave Life Sciences Ltd Compositions d'oligonucléotides et procédés associés
MA45496A (fr) 2016-06-17 2019-04-24 Hoffmann La Roche Molécules d'acide nucléique pour la réduction de l'arnm de padd5 ou pad7 pour le traitement d'une infection par l'hépatite b
WO2017219017A1 (en) 2016-06-17 2017-12-21 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
BR112019000061A2 (pt) * 2016-07-05 2019-11-26 Biomarin Technologies B.V. comutação e modulação de splicing de pré-mrna compreendendo porções de arcabouço bicíclico, com características melhoradas para o tratamento de distúrbios genéticos
CA3037663A1 (en) 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified-rna oligonucleotide compositions and uses thereof
WO2018055577A1 (en) * 2016-09-23 2018-03-29 Synthena Ag Mixed tricyclo-dna, 2'-modified rna oligonucleotide compositions and uses thereof
US11260134B2 (en) 2016-09-29 2022-03-01 National University Corporation Tokyo Medical And Dental University Double-stranded nucleic acid complex having overhang
US11400161B2 (en) 2016-10-06 2022-08-02 Ionis Pharmaceuticals, Inc. Method of conjugating oligomeric compounds
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
CA3043768A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
US11033570B2 (en) 2016-12-02 2021-06-15 Cold Spring Harbor Laboratory Modulation of Lnc05 expression
US11572558B2 (en) 2017-02-06 2023-02-07 Nissan Chemical Corporation Single-stranded oligonucleotide
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
EP3647423A4 (en) 2017-06-30 2021-03-24 National University Corporation Tokyo Medical and Dental University STRAIGHT DOUBLE-STRANDED ANTI-MIRROR
US20200362337A1 (en) 2017-08-08 2020-11-19 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
KR20240035631A (ko) 2017-08-25 2024-03-15 스톡 테라퓨틱스, 인크. 병태 및 질환 치료용 안티센스 올리고머
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
EP3724333A2 (en) 2017-12-11 2020-10-21 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating fndc3b expression
WO2019115417A2 (en) 2017-12-12 2019-06-20 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating rb1 expression
WO2019121838A1 (en) 2017-12-21 2019-06-27 F. Hoffmann-La Roche Ag Companion diagnostic for htra1 rna antagonists
WO2019126641A2 (en) 2017-12-21 2019-06-27 Ionis Pharmaceuticals, Inc. Modulation of frataxin expression
EP4092117A1 (en) 2017-12-22 2022-11-23 Roche Innovation Center Copenhagen A/S Gapmer oligonucleotides comprising a phosphorodithioate internucleoside linkage
BR112020012427A8 (pt) 2017-12-22 2024-04-02 Roche Innovation Ct Copenhagen As Oligonucleotídeos, sal farmaceuticamente aceitável, conjugado, composição farmacêutica e processo para a fabricação de um oligonucleotídeo
JP2021508327A (ja) 2017-12-22 2021-03-04 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 新規のチオホスホラミダイト
JP2021511022A (ja) 2018-01-10 2021-05-06 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 Ｐｉａｓ４発現を調節するためのオリゴヌクレオチド
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
CN111902537A (zh) 2018-01-15 2020-11-06 Ionis制药公司 Dnm2表达的调节剂
JP2021510525A (ja) 2018-01-17 2021-04-30 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 Ｅｒｃ１発現を調節するためのオリゴヌクレオチド
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
CN111868245B (zh) 2018-02-09 2024-11-19 豪夫迈·罗氏有限公司 用于调节tmem106b表达的寡核苷酸
EP3752612A4 (en) 2018-02-12 2021-11-10 Ionis Pharmaceuticals, Inc. MODIFIED COMPOUNDS AND USES THEREOF
JP7360170B2 (ja) 2018-02-28 2023-10-12 国立大学法人 東京医科歯科大学 虚血病変部位特異的な遺伝子治療法
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
WO2019169243A1 (en) 2018-03-02 2019-09-06 Ionis Pharmaceuticals, Inc. Compounds and methods for the modulation of 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
EP3769769A4 (en) 2018-03-19 2022-05-04 National University Corporation Tokyo Medical and Dental University NUCLEIC ACID WITH REDUCED TOXICITY
AU2019237599A1 (en) 2018-03-20 2020-11-12 Nissan Chemical Corporation Antisense oligonucleotide having reduced toxicity
WO2019182109A1 (ja) 2018-03-22 2019-09-26 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
WO2019183440A1 (en) 2018-03-22 2019-09-26 Ionis Pharmaceuticals, Inc. Methods for modulating fmr1 expression
KR20200141481A (ko) 2018-04-11 2020-12-18 아이오니스 파마수티컬즈, 인코포레이티드 Ezh2 발현의 조절제
US12060558B2 (en) 2018-05-04 2024-08-13 Stoke Therapeutics, Inc. Methods and compositions for treatment of cholesteryl ester storage disease
WO2019215175A1 (en) 2018-05-08 2019-11-14 Roche Innovation Center Copenhagen A/S Oligonucleotides for modulating myh7 expression
IL277970B2 (en) 2018-05-09 2025-03-01 Ionis Pharmaceuticals Inc Oligomeric compounds comprising modified oligonucleotides and conjugated groups for use in the treatment of end-stage renal disease, chronic kidney disease and coronary artery disease
MX2020011911A (es) 2018-05-09 2021-01-29 Ionis Pharmaceuticals Inc Compuestos y metodos para reducir de la expresion de atxn3.
EP3802824A1 (en) 2018-06-05 2021-04-14 F. Hoffmann-La Roche AG Oligonucleotides for modulating atxn2 expression
US11833168B2 (en) 2018-06-14 2023-12-05 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
WO2020007772A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting gbp-1
WO2020007700A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting spi1
WO2020007702A1 (en) 2018-07-02 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting bcl2l11
RU2768285C1 (ru) 2018-07-03 2022-03-23 Ф. Хоффманн-Ля Рош Аг Олигонуклеотиды для модуляции экспрессии тау-белка
WO2020007826A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting mbtps1
WO2020007889A1 (en) 2018-07-05 2020-01-09 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting stat1
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
WO2020011745A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers6
WO2020011744A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting cers5
WO2020011869A2 (en) 2018-07-11 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting tlr2
KR20210033004A (ko) 2018-07-13 2021-03-25 에프. 호프만-라 로슈 아게 Rtel1의 발현을 조절하기 위한 올리고뉴클레오티드
US12496311B2 (en) 2018-07-17 2025-12-16 Aronora, Inc. Methods for safely reducing thrombopoietin
AU2019310097A1 (en) 2018-07-25 2021-02-04 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ATXN2 expression
US20210308170A1 (en) 2018-07-27 2021-10-07 Osaka University Composition for suppression of aging, prevention, amelioration, or treatment of an age-related disease or symptom, or extension of lifespan
ES2924362T3 (es) 2018-07-31 2022-10-06 Roche Innovation Ct Copenhagen As Oligonucleótidos que comprenden un enlace internucleosídico fosforotritioato
MX2020013270A (es) 2018-07-31 2021-02-18 Roche Innovation Ct Copenhagen As Oligonucleotidos que comprenden enlace internucleosidico de fosforotritioato.
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
US12097263B2 (en) 2018-08-02 2024-09-24 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
WO2020028864A1 (en) 2018-08-02 2020-02-06 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating facioscapulohumeral muscular dystrophy
US11911484B2 (en) 2018-08-02 2024-02-27 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating myotonic dystrophy
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
WO2020038976A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting usp8
WO2020038971A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting vcan
WO2020038973A1 (en) 2018-08-23 2020-02-27 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting sptlc1
EP3620519A1 (en) 2018-09-04 2020-03-11 F. Hoffmann-La Roche AG Use of isolated milk extracellular vesicles for delivering oligonucleotides orally
WO2020089260A1 (en) 2018-11-01 2020-05-07 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
US11208650B2 (en) 2018-11-15 2021-12-28 Ionis Pharmaceuticals, Inc. Modulators of IRF5 expression
WO2020106996A1 (en) 2018-11-21 2020-05-28 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
JP2022514648A (ja) 2018-12-21 2022-02-14 ベーリンガー インゲルハイム インターナショナル ゲゼルシャフト ミット ベシュレンクテル ハフツング Ｃａｒｄ９を標的とするアンチセンスオリゴヌクレオチド
CN113365607A (zh) 2019-01-25 2021-09-07 豪夫迈·罗氏有限公司 用于口服药物递送的脂质囊泡
KR20210122809A (ko) 2019-01-31 2021-10-12 아이오니스 파마수티컬즈, 인코포레이티드 Yap1 발현의 조절인자
JP2022521510A (ja) 2019-02-20 2022-04-08 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 ホスホノアセテートギャップマー型オリゴヌクレオチド
KR20210132681A (ko) 2019-02-20 2021-11-04 로슈 이노베이션 센터 코펜하겐 에이/에스 신규 포스포라미디트
EP3931348B1 (en) 2019-02-26 2023-08-09 Roche Innovation Center Copenhagen A/S Oligonucleotide formulation method
BR112021015323A2 (pt) 2019-02-27 2021-10-05 Ionis Pharmaceuticals, Inc. Moduladores de expressão de malat1
EP3981431A4 (en) 2019-03-14 2023-06-28 Rena Therapeutics Inc. Nucleic acid complex for modulating ihh expression
TW202536179A (zh) 2019-03-29 2025-09-16 美商Ionis製藥公司 用於調節ube3a-ats之化合物及方法
US11286485B2 (en) 2019-04-04 2022-03-29 Hoffmann-La Roche Inc. Oligonucleotides for modulating ATXN2 expression
EP3954395A4 (en) 2019-04-08 2023-06-28 National University Corporation Tokyo Medical and Dental University Pharmaceutical composition for muscle disease treatment
WO2020245233A1 (en) 2019-06-06 2020-12-10 F. Hoffmann-La Roche Ag Antisense oligonucleotides targeting atxn3
EP3956450B1 (en) 2019-07-26 2025-08-13 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
US20240117346A1 (en) 2019-10-11 2024-04-11 National University Corporation Tokyo Medical And Dental University Modified hetero nucleic acids
CN114502566B (zh) 2019-10-11 2025-02-11 住友化学株式会社 核酸寡聚物的制造方法
EP4067489A4 (en) * 2019-11-27 2024-06-12 Tokyo Institute of Technology METHODS FOR REDUCING THE TOXICITY OF ANTISENSE NUCLEIC ACIDS
WO2021130270A1 (en) 2019-12-24 2021-07-01 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
EP4098655A4 (en) 2020-01-29 2024-02-28 Sumitomo Chemical Company, Limited METHOD FOR PRODUCING NUCLEIC ACID OLIGOMERS
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
JP7753106B2 (ja) 2020-01-31 2025-10-14 株式会社三和化学研究所 Ａｔｎ１のアンチセンスオリゴヌクレオチド
BR112022016238A2 (pt) 2020-02-28 2022-10-11 Ionis Pharmaceuticals Inc Compostos e métodos para modular smn2
US20230159925A1 (en) 2020-03-04 2023-05-25 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
US20230174981A1 (en) 2020-03-16 2023-06-08 National University Corporation Tokyo Medical And Dental University Heteronucleic acid containing morpholino nucleic acid
EP4130018A4 (en) 2020-03-27 2024-04-17 Sumitomo Chemical Company, Limited METHOD FOR PRODUCING A NUCLEIC ACID OLIGOMER
CN115768891A (zh) * 2020-04-17 2023-03-07 不列颠哥伦比亚大学 用于抑制tdp-43和fus聚集的组合物和方法
EP4143321A4 (en) 2020-05-01 2025-02-19 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating atxn1
CN115867657A (zh) 2020-05-11 2023-03-28 斯托克制药公司 用于治疗疾患和疾病的opa1反义寡聚物
IL298530A (en) 2020-06-29 2023-01-01 Ionis Pharmaceuticals Inc Compounds and methods for modulating plp1
CN115867559A (zh) 2020-07-09 2023-03-28 住友化学株式会社 核酸寡聚物的制造方法
AU2021315992A1 (en) 2020-07-28 2023-02-09 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
KR20230048052A (ko) 2020-08-07 2023-04-10 아이오니스 파마수티컬즈, 인코포레이티드 Scn2a를 조절하는 화합물 및 방법
JP7719788B2 (ja) 2020-09-24 2025-08-06 住友化学株式会社 核酸オリゴマーの製造方法
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
TW202237842A (zh) 2020-12-03 2022-10-01 瑞士商赫孚孟拉羅股份公司 靶向ａｔｘｎ３之反義寡核苷酸
US12018261B2 (en) 2020-12-18 2024-06-25 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating factor XII
JP2024503609A (ja) 2020-12-31 2024-01-26 ダイン セラピューティクス，インコーポレーテッド 筋緊張性ジストロフィーを処置するための筋標的化複合体およびその使用
EP4284513A1 (en) 2021-01-26 2023-12-06 Universite Brest Bretagne Occidentale Novel stim1 splicing variants and uses thereof
US20240358845A1 (en) 2021-05-10 2024-10-31 Entrada Therapeutics, Inc. Compositions and methods for intracellular therapeutics
US20220370487A1 (en) * 2021-05-10 2022-11-24 Q-State Biosciences, Inc. Compositions targeting sodium channel 1.6
CA3221584A1 (en) 2021-05-25 2022-12-01 National University Corporation Tokyo Medical And Dental University Heteronucleic acid containing scpbna or amna
AU2022285344A1 (en) 2021-05-31 2023-12-14 Rena Therapeutics Inc. Ligand-bound nucleic acid complex
EP4355759A1 (en) 2021-06-18 2024-04-24 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing ifnar1 expression
IL309001A (en) 2021-06-23 2024-02-01 Entrada Therapeutics Inc Antisense compounds and methods for targeting CUG repeats
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
US11638761B2 (en) 2021-07-09 2023-05-02 Dyne Therapeutics, Inc. Muscle targeting complexes and uses thereof for treating Facioscapulohumeral muscular dystrophy
EP4389891A4 (en) 2021-08-19 2025-10-29 Univ Nat Corp Tokyo Medical & Dental MODIFIED HETERONUCLEIC ACID CONTAINING MORPHOLINO NUCLEIC ACID
EP4389893A4 (en) 2021-08-21 2025-09-10 Takeda Pharmaceuticals Co PEPTIDE-DRUG CONJUGATE BINDING HUMAN TRANSFERRIN RECEPTOR
US11833221B2 (en) 2021-09-01 2023-12-05 Ionis Pharmaceuticals, Inc. Oligomeric compounds for reducing DMPK expression
EP4403191A1 (en) 2021-09-15 2024-07-24 National University Corporation Tokyo Medical and Dental University Heteronucleic acid including 2'-modified nucleoside
KR20240082343A (ko) 2021-09-28 2024-06-10 스미또모 가가꾸 가부시끼가이샤 정제 디클로로아세트산의 제조 방법
JPWO2023080159A1 (enExample) 2021-11-02 2023-05-11
EP4426833A1 (en) 2021-11-03 2024-09-11 F. Hoffmann-La Roche AG Oligonucleotides for modulating apolipoprotein e4 expression
WO2023083906A2 (en) 2021-11-11 2023-05-19 F. Hoffmann-La Roche Ag Pharmaceutical combinations for treatment of hbv
CN118434860A (zh) 2021-12-20 2024-08-02 豪夫迈·罗氏有限公司 苏糖核酸反义寡核苷酸及其方法
WO2023141507A1 (en) 2022-01-20 2023-07-27 Genentech, Inc. Antisense oligonucleotides for modulating tmem106b expression
AU2023234536A1 (en) 2022-03-16 2024-09-26 Empirico Inc. Galnac compositions for improving sirna bioavailability
KR20240162536A (ko) 2022-03-23 2024-11-15 스미또모 가가꾸 가부시끼가이샤 핵산 올리고머의 제조 방법
CN119278265A (zh) 2022-03-28 2025-01-07 艾姆皮瑞克公司 经修饰的寡核苷酸
JP2025512464A (ja) 2022-04-15 2025-04-17 ダイン セラピューティクス，インコーポレーテッド 筋緊張性ジストロフィーを処置するための筋標的化複合体および製剤
CN119403816A (zh) 2022-07-22 2025-02-07 住友化学株式会社 寡核苷酸的制造方法
WO2024024873A1 (ja) 2022-07-28 2024-02-01 住友化学株式会社 チオ化溶液
WO2024026474A1 (en) 2022-07-29 2024-02-01 Regeneron Pharmaceuticals, Inc. Compositions and methods for transferrin receptor (tfr)-mediated delivery to the brain and muscle
WO2024050261A1 (en) 2022-08-29 2024-03-07 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
CN120239607A (zh) 2022-09-23 2025-07-01 Ionis制药公司 用于降低mecp2表达的化合物和方法
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
CA3266197A1 (en) * 2022-09-29 2024-04-04 Celanese Eva Performance Polymers Llc IMPLANTABLE MEDICAL DEVICE FOR THE ADMINISTRATION OF NUCLEIC ACID
WO2024089953A1 (ja) 2022-10-27 2024-05-02 住友化学株式会社 オリゴヌクレオチドの製造方法
IL320016A (en) 2022-11-04 2025-06-01 Regeneron Pharma 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
EP4644404A1 (en) 2022-12-26 2025-11-05 Sumitomo Chemical Company, Limited Oligonucleotide production method
EP4652273A1 (en) 2023-01-16 2025-11-26 Institut National de la 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

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007027894A2 (en) * 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Antisense compounds having enhanced anti-microrna activity
WO2007027775A2 (en) * 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Methods for use in modulating mir-122a

Family Cites Families (82)

* 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
US5670633A (en) 1990-01-11 1997-09-23 Isis Pharmaceuticals, Inc. Sugar modified oligonucleotides that detect and modulate gene expression
US5646265A (en) 1990-01-11 1997-07-08 Isis Pharmceuticals, Inc. Process for the preparation of 2'-O-alkyl purine phosphoramidites
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
ES2116977T3 (es) 1990-05-11 1998-08-01 Microprobe Corp Soportes solidos para ensayos de hibridacion de acidos nucleicos y metodos para inmovilizar oligonucleotidos de modo covalente.
US6582908B2 (en) 1990-12-06 2003-06-24 Affymetrix, Inc. Oligonucleotides
DE59208572D1 (de) 1991-10-17 1997-07-10 Ciba Geigy Ag Bicyclische Nukleoside, Oligonukleotide, Verfahren zu deren Herstellung und Zwischenprodukte
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
EP0691968B1 (en) 1993-03-30 1997-07-16 Sanofi 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
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
US6770748B2 (en) 1997-03-07 2004-08-03 Takeshi Imanishi Bicyclonucleoside and oligonucleotide analogue
CA2303299C (en) 1997-09-12 2016-02-23 Exiqon A/S Oligonucleotide analogues
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
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
US7098192B2 (en) * 1999-04-08 2006-08-29 Isis Pharmaceuticals, Inc. Antisense oligonucleotide modulation of STAT3 expression
DE60033927T2 (de) 1999-05-04 2007-11-29 Santaris Pharma A/S L-ribo-lna analoge
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
US20030125241A1 (en) * 2001-05-18 2003-07-03 Margit Wissenbach Therapeutic uses of LNA-modified oligonucleotides in infectious diseases
US7407943B2 (en) * 2001-08-01 2008-08-05 Isis Pharmaceuticals, Inc. Antisense modulation of apolipoprotein B expression
US7888324B2 (en) 2001-08-01 2011-02-15 Genzyme Corporation 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
WO2004041889A2 (en) 2002-11-05 2004-05-21 Isis Pharmaceuticals, Inc. Polycyclic sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
AU2003290596B2 (en) 2002-11-05 2011-05-12 Isis Pharmaceuticals, Inc. Sugar surrogate-containing oligomeric compounds and compositions for use in gene modulation
EP2336318B1 (en) * 2002-11-13 2013-04-24 Genzyme Corporation Antisense modulation of apolipoprotein b expression
EP2141233B1 (en) 2002-11-18 2016-10-19 Roche Innovation Center Copenhagen A/S Antisense design
AU2004209598B2 (en) 2003-02-10 2009-08-06 Enzon Pharmaceuticals, Inc. Oligomeric compounds for the modulation of survivin expression
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
US20050074801A1 (en) * 2003-09-09 2005-04-07 Monia Brett P. Chimeric oligomeric compounds comprising alternating regions of northern and southern conformational geometry
US20050053981A1 (en) * 2003-09-09 2005-03-10 Swayze Eric E. Gapped oligomeric compounds having linked bicyclic sugar moieties at the termini
EA009670B1 (ru) * 2003-09-18 2008-02-28 Ай Эс Ай Эс ФАРМАСЬЮТИКАЛЗ, ИНК. МОДУЛИРОВАНИЕ ЭКСПРЕССИИ eIF4E
JP4642775B2 (ja) 2003-12-23 2011-03-02 サンタリス ファーマ アー／エス Ｂｃｌ−２のモデュレーションのためのオリゴマー化合物
GB0407382D0 (en) * 2004-03-31 2004-05-05 Univ Cambridge Tech Therapeutic methods and means
EP1765416A4 (en) 2004-06-03 2010-03-24 Isis Pharmaceuticals Inc DOUBLE-STRONG COMPOSITIONS WITH DIFFERENTLY MODIFIED STRANDS FOR USE IN GENE MODULATION
EP3210633B1 (en) 2006-01-26 2019-06-19 Ionis Pharmaceuticals, Inc. Compositions and their uses directed to huntingtin
PL2314594T3 (pl) 2006-01-27 2014-12-31 Isis Pharmaceuticals Inc Zmodyfikowane w pozycji 6 analogi bicykliczne kwasów nukleinowych
DK2363481T3 (en) * 2006-05-05 2017-06-26 Ionis Pharmaceuticals Inc Compounds and methods for modulating gene expression
CA2651453C (en) 2006-05-11 2014-10-14 Isis Pharmaceuticals, Inc. 5'-modified bicyclic nucleic acid analogs
AU2007310989B2 (en) * 2006-10-18 2014-05-29 Isis Pharmaceuticals, Inc. Antisense compounds
EP2134736A4 (en) 2007-03-15 2013-05-29 Jyoti Chattopadhyaya FIVE- AND SIX-PIECE CONFORMATIVELY FIXED 2 ', 4'-CARBOCYCLIC RIBOTHYMIDINE 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
US8278426B2 (en) † 2007-06-08 2012-10-02 Isis Pharmaceuticals, Inc. Carbocyclic bicyclic nucleic acid analogs
KR101654007B1 (ko) 2007-08-15 2016-09-05 아이오니스 파마수티컬즈, 인코포레이티드 테트라하이드로피란 핵산 유사체
WO2009061841A2 (en) 2007-11-05 2009-05-14 Isis Pharmaceuticals, Inc. Modified polynucleotides as 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
EP2356129B1 (en) 2008-09-24 2013-04-03 Isis Pharmaceuticals, Inc. Substituted alpha-l-bicyclic nucleosides
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
SI3505528T1 (sl) 2011-04-21 2021-04-30 Glaxo Group Limited Modulacija izražanja virusa hepatitisa B (HBV)
DK2742135T4 (da) 2011-08-11 2020-07-13 Ionis Pharmaceuticals Inc Bindingsmodificerede gapped oligomeriske forbindelser og anvendelser deraf
EP3336189A1 (en) * 2012-04-20 2018-06-20 Ionis 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
HRP20191600T1 (hr) † 2013-09-13 2019-11-29 Ionis Pharmaceuticals Inc Modulatori faktora b komplementa

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2007027894A2 (en) * 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Antisense compounds having enhanced anti-microrna activity
WO2007027775A2 (en) * 2005-08-29 2007-03-08 Isis Pharmaceuticals, Inc. Methods for use in modulating mir-122a

Also Published As

Publication number Publication date
AU2007310989A1 (en) 2008-04-24
DK2092065T4 (da) 2019-10-21
AU2014203243A1 (en) 2014-07-10
JP2010507579A (ja) 2010-03-11
AU2016231660A1 (en) 2016-10-20
DK2092065T3 (da) 2012-04-02
DK2410054T3 (en) 2017-04-10
ES2377327T5 (es) 2020-04-28
WO2008049085A1 (en) 2008-04-24
EP2410053B1 (en) 2014-10-01
JP2014221817A (ja) 2014-11-27
EP3202905A1 (en) 2017-08-09
ES2526295T3 (es) 2015-01-09
AU2014203243B2 (en) 2016-06-30
ES2620472T5 (es) 2020-07-09
EP2092065B2 (en) 2019-07-24
DK2410053T4 (da) 2020-08-31
ES2377327T3 (es) 2012-03-26
CA2667055A1 (en) 2008-04-24
EP2410054B1 (en) 2017-01-25
US9550988B2 (en) 2017-01-24
ATE540118T1 (de) 2012-01-15
US20100197762A1 (en) 2010-08-05
US20170182082A1 (en) 2017-06-29
ES2526295T5 (es) 2021-05-04
CA2667055C (en) 2017-05-09
EP2092065B1 (en) 2012-01-04
EP2410053B2 (en) 2020-07-15
EP2092065A1 (en) 2009-08-26
DK2410054T4 (da) 2020-02-10
US20200276221A1 (en) 2020-09-03
DK2410053T3 (en) 2014-12-08
HK1166643A1 (en) 2012-11-02
JP5665317B2 (ja) 2015-02-04
EP2410053A1 (en) 2012-01-25
ES2620472T3 (es) 2017-06-28
EP2410054B2 (en) 2019-12-11
US20240058370A1 (en) 2024-02-22
US10493092B2 (en) 2019-12-03
EP2410054A1 (en) 2012-01-25
EP3916095A1 (en) 2021-12-01

Similar Documents

Publication Publication Date Title
AU2007310989B2 (en) Antisense compounds
US11981897B2 (en) Compounds and methods for modulation of dystrophia myotonica-protein kinase (DMPK) expression
EP2019692B1 (en) Compounds and methods for modulating expression of gccr
WO2012012443A2 (en) Modulation of dystrophia myotonica-protein kinase (dmpk) expression
EP2505650A1 (en) Compounds and methods for modulating expression of PCSK9
HK1166643B (en) Antisense compounds
WO2011133923A1 (en) Modulation of lactosylceramide synthase (lcs) expression

Legal Events

Date Code Title Description
FGA Letters patent sealed or granted (standard patent)