JP2016522674A - 遺伝子発現を調節するための組成物及び方法 - Google Patents

遺伝子発現を調節するための組成物及び方法 Download PDF

Info

Publication number
JP2016522674A
JP2016522674A JP2015512857A JP2015512857A JP2016522674A JP 2016522674 A JP2016522674 A JP 2016522674A JP 2015512857 A JP2015512857 A JP 2015512857A JP 2015512857 A JP2015512857 A JP 2015512857A JP 2016522674 A JP2016522674 A JP 2016522674A
Authority
JP
Japan
Prior art keywords
oligonucleotide
nucleotide
stranded oligonucleotide
nucleotides
sequence
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.)
Pending
Application number
JP2015512857A
Other languages
English (en)
Japanese (ja)
Other versions
JP2016522674A5 (enExample
Inventor
アーサー エム. クリーグ,
アーサー エム. クリーグ,
ロメシュ スブラマニアン,
ロメシュ スブラマニアン,
ジェイムズ マクスウィゲン,
ジェイムズ マクスウィゲン,
ジェニー ティー. リー,
ジェニー ティー. リー,
Original Assignee
ラナ セラピューティクス インコーポレイテッド
ラナ セラピューティクス インコーポレイテッド
ザ ジェネラル ホスピタル コーポレイション
ザ ジェネラル ホスピタル コーポレイション
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by ラナ セラピューティクス インコーポレイテッド, ラナ セラピューティクス インコーポレイテッド, ザ ジェネラル ホスピタル コーポレイション, ザ ジェネラル ホスピタル コーポレイション filed Critical ラナ セラピューティクス インコーポレイテッド
Publication of JP2016522674A publication Critical patent/JP2016522674A/ja
Publication of JP2016522674A5 publication Critical patent/JP2016522674A5/ja
Pending legal-status Critical Current

Links

Classifications

    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/11Antisense
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/315Phosphorothioates
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/3212'-O-R Modification
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/32Chemical structure of the sugar
    • C12N2310/323Chemical structure of the sugar modified ring structure
    • C12N2310/3231Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/34Spatial arrangement of the modifications
    • C12N2310/343Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/30Chemical structure
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3517Marker; Tag

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Epidemiology (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • Organic Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
JP2015512857A 2012-05-16 2013-05-16 遺伝子発現を調節するための組成物及び方法 Pending JP2016522674A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261648077P 2012-05-16 2012-05-16
US61/648,077 2012-05-16
PCT/US2013/041437 WO2013173637A1 (en) 2012-05-16 2013-05-16 Compositions and methods for modulating gene expression

Publications (2)

Publication Number Publication Date
JP2016522674A true JP2016522674A (ja) 2016-08-04
JP2016522674A5 JP2016522674A5 (enExample) 2016-09-15

Family

ID=49584304

Family Applications (1)

Application Number Title Priority Date Filing Date
JP2015512857A Pending JP2016522674A (ja) 2012-05-16 2013-05-16 遺伝子発現を調節するための組成物及び方法

Country Status (4)

Country Link
US (1) US20150141320A1 (enExample)
EP (1) EP2850184A4 (enExample)
JP (1) JP2016522674A (enExample)
WO (1) WO2013173637A1 (enExample)

Cited By (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021514182A (ja) * 2018-02-09 2021-06-10 ジェネンテック, インコーポレイテッド Tmem106b発現を調節するためのオリゴヌクレオチド
JP2023503202A (ja) * 2020-02-12 2023-01-26 アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー,リミテッド アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用
JP2023523622A (ja) * 2020-04-29 2023-06-06 サリオジェン セラピューティクス インコーポレイテッド 遺伝性黄斑変性症の治療のための組成物及び方法

Families Citing this family (88)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20130225659A1 (en) 2010-07-19 2013-08-29 Isis Pharmaceuticals, Inc. Modulation of nuclear-retained rna
US9920317B2 (en) 2010-11-12 2018-03-20 The General Hospital Corporation Polycomb-associated non-coding RNAs
AU2011325956B2 (en) 2010-11-12 2016-07-14 The General Hospital Corporation Polycomb-associated non-coding RNAs
CA2873769A1 (en) 2012-05-16 2013-11-21 Rana Therapeutics Inc. Compositions and methods for modulating hemoglobin gene family expression
SG11201407483YA (en) 2012-05-16 2014-12-30 Rana Therapeutics Inc Compositions and methods for modulating smn gene family expression
BR112014028644A2 (pt) 2012-05-16 2017-08-15 Rana Therapeutics Inc Composições e métodos para modulação da expressão de atp2a2
US10837014B2 (en) 2012-05-16 2020-11-17 Translate Bio Ma, Inc. Compositions and methods for modulating SMN gene family expression
AU2013262709A1 (en) 2012-05-16 2015-01-22 Rana Therapeutics, Inc. Compositions and methods for modulating MECP2 expression
BR112014028634A2 (pt) 2012-05-16 2017-06-27 Rana Therapeutics Inc composições e métodos para modulação da expressão de utrn
CA3112080A1 (en) * 2013-06-26 2014-12-31 Mcgill University Markers to predict macrocyclic lactone resistance in dirofilaria immitis, the causative agent of heartworm disease
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
IL282239B2 (en) 2013-09-05 2023-10-01 Sarepta Therapeutics Inc Antisense-induced exon2 inclusion in acid alpha-glucosidase
WO2015051283A1 (en) 2013-10-04 2015-04-09 Rana Therapeutics, Inc. Compositions and methods for treating amyotrophic lateral sclerosis
US11162096B2 (en) 2013-10-14 2021-11-02 Ionis Pharmaceuticals, Inc Methods for modulating expression of C9ORF72 antisense transcript
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
EP3207138B1 (en) * 2014-10-17 2020-07-15 Alnylam Pharmaceuticals, Inc. Polynucleotide agents targeting aminolevulinic acid synthase-1 (alas1) and uses thereof
WO2016070060A1 (en) 2014-10-30 2016-05-06 The General Hospital Corporation Methods for modulating atrx-dependent gene repression
US10793855B2 (en) * 2015-01-06 2020-10-06 Ionis Pharmaceuticals, Inc. Compositions for modulating expression of C9ORF72 antisense transcript
ES2848377T3 (es) 2015-02-26 2021-08-09 Ionis Pharmaceuticals Inc Moduladores específicos de alelo de RODOPSINA P23H
GB201504124D0 (en) 2015-03-11 2015-04-22 Proqr Therapeutics B V Oligonucleotides
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
US10801024B2 (en) * 2015-05-20 2020-10-13 Indiana University Research And Technology Corporation Inhibition of lncRNA HOTAIR and related materials and methods
EP3124619B1 (en) * 2015-07-31 2019-03-06 Menicon Co., Ltd Reagents, method and kit for across and within dog breed glaucoma diagnosis
WO2017106211A1 (en) * 2015-12-14 2017-06-22 Cold Spring Harbor Laboratory Antisense oligomers for treatment of polycystic kidney disease
WO2017117496A1 (en) 2015-12-31 2017-07-06 Ionis Pharmaceuticals, Inc. Methods for reducing ataxin-2 expression
WO2017131236A1 (ja) 2016-01-29 2017-08-03 協和発酵キリン株式会社 核酸複合体
WO2017134252A1 (en) * 2016-02-05 2017-08-10 INSERM (Institut National de la Santé et de la Recherche Médicale) Antisense oligonucleotides effective to reduce the expression of menin in cancer cells of a subject
WO2017184529A1 (en) 2016-04-18 2017-10-26 Sarepta Therapeutics, Inc. Antisense oligomers and methods of using the same for treating diseases associated with the acid alpha-glucosidase gene
US10689709B2 (en) 2016-04-20 2020-06-23 JBS Science Inc. Kit and method for detecting mutations in CTNNB1 and hTERT, and use thereof in HCC detection and disease management
CA3023514A1 (en) 2016-06-17 2017-12-21 Ionis Pharmaceuticals, Inc. Modulation of gys1 expression
WO2018004004A1 (ja) 2016-06-30 2018-01-04 協和発酵キリン株式会社 核酸複合体
CR20180606A (es) 2016-07-01 2019-02-14 Hoffmann La Roche Oligonucleótidos antisentido para modular la expresión de htra1
WO2018009547A1 (en) * 2016-07-05 2018-01-11 The Usa, As Represented By The Secretary, Dept. Of Health And Human Services Diagnosing col6-related disorders and methods for treating same
JOP20190104A1 (ar) 2016-11-10 2019-05-07 Ionis Pharmaceuticals Inc مركبات وطرق لتقليل التعبير عن atxn3
US11118182B2 (en) 2016-11-17 2021-09-14 Ramot At Tel-Aviv University Ltd. Modulators of human KAI1 metastasis suppressor gene, methods and uses thereof
WO2018102397A1 (en) 2016-11-29 2018-06-07 PureTech Health LLC Exosomes for delivery of therapeutic agents
WO2018132755A1 (en) * 2017-01-12 2018-07-19 Duke University Compositions and methods for disrupting the molecular mechanisms associated with mitochondrial dysfunction and neurodegenerative disease
WO2018180005A1 (ja) * 2017-03-31 2018-10-04 国立研究開発法人日本医療研究開発機構 コンドロイチン硫酸生合成を阻害するアンチセンス核酸
GB201706009D0 (en) * 2017-04-13 2017-05-31 Proqr Therapeutics Ii Bv Antisense oligonucleotides for the treatment of stargardt disease
US20190055564A1 (en) * 2017-06-01 2019-02-21 F. Hoffmann-La Roche Ag Antisense oligonucleotides for modulating htra1 expression
EP4183882A1 (en) 2017-09-08 2023-05-24 MiNA Therapeutics Limited Stabilized hnf4a sarna compositions and methods of use
EP4219715A3 (en) 2017-09-08 2023-09-06 MiNA Therapeutics Limited Stabilized cebpa sarna compositions and methods of use
US20230201357A1 (en) * 2017-11-08 2023-06-29 Aptamer Diagnostic, Inc. D-dimer-specific aptamers and methods of use in diagnostics, therapeutic and theranostic purposes
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
HRP20250804T1 (hr) 2017-12-01 2025-09-12 The Texas A&M University System Antisense liječenje angelmanovog sindroma
BR112020016347A2 (pt) * 2018-02-21 2021-01-12 Bristol-Myers Squibb Company Oligonucleotídeos antisense de camk2d e seus usos
EP3759127A4 (en) 2018-03-02 2022-03-30 Ionis Pharmaceuticals, Inc. COMPOUNDS AND METHODS FOR MODULATING AMYLOID BETA PRECURSOR PROTEIN
TWI840345B (zh) 2018-03-02 2024-05-01 美商Ionis製藥公司 Irf4表現之調節劑
PE20201349A1 (es) 2018-04-11 2020-11-30 Ionis Pharmaceuticals Inc Moduladores de la expresion de ezh2
WO2019217708A1 (en) 2018-05-09 2019-11-14 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn3 expression
TWI851574B (zh) 2018-05-14 2024-08-11 美商阿尼拉製藥公司 血管收縮素原(AGT)iRNA組成物及其使用方法
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
WO2020011653A1 (en) * 2018-07-09 2020-01-16 Roche Innovation Center Copenhagen A/S Antisense oligonucleotides targeting kynu
EP3824086A1 (en) * 2018-07-19 2021-05-26 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of abca4
CA3106986A1 (en) 2018-07-25 2020-01-30 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing atxn2 expression
WO2020084162A1 (en) * 2018-10-26 2020-04-30 Vrije Universiteit Brussel Liver-specific nucleic acid regulatory elements and methods and use thereof
TW202028222A (zh) 2018-11-14 2020-08-01 美商Ionis製藥公司 Foxp3表現之調節劑
TW202214854A (zh) 2019-01-31 2022-04-16 美商Ionis製藥公司 Yap1表現之調節劑
AU2020239987A1 (en) * 2019-03-15 2021-11-04 University Of Massachusetts Oligonucleotides for tissue specific ApoE modulation
MD3947684T2 (ro) * 2019-03-29 2025-10-31 Ionis Pharmaceuticals Inc Compuși și metode pentru modularea UBE3A-ATS
US11739324B2 (en) 2019-06-25 2023-08-29 Stichting Katholieke Universiteit Antisense oligonucleotides rescue aberrant splicing of ABCA4
US20220281911A1 (en) 2019-08-19 2022-09-08 Mina Therapeutics Limited Oligonucleotide conjugate compositions and methods of use
CA3152513A1 (en) * 2019-08-30 2021-03-04 Baylor College Of Medicine System for regulating gene expression
EP3822370A1 (en) * 2019-11-15 2021-05-19 Curiosity Diagnostics Sp. z o.o. Method of determining the presence of a hyper-virulent clostridioides difficile strain of the b1/nap1/027 group in a sample
WO2021126997A1 (en) * 2019-12-18 2021-06-24 Petdx, Inc. Methods and compositions for cancer detection, characterization or management in companion animals
AU2021212949A1 (en) * 2020-01-31 2022-09-01 Nissan Chemical Corporation Antisense oligonucleotide of ATN1
EP4116419A1 (en) * 2020-03-04 2023-01-11 Nissan Chemical Corporation Antisense oligonucleotide of calm2
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
US20230272387A1 (en) * 2020-07-22 2023-08-31 The Florey Institute Of Neuroscience And Mental Health Compositions and methods for treating disorders associated with loss-of-function mutations in scn2a
US12384814B2 (en) 2020-07-28 2025-08-12 Ionis Pharmaceuticals, Inc. Compounds and methods for reducing app expression
EP4015040A1 (en) * 2020-12-16 2022-06-22 Fundación para la Formación e Investigación Sanitarias de la Región de Murcia Method for treating blood diseases
CA3205040A1 (en) 2020-12-18 2022-06-23 Ionis Pharmaceuticals, Inc. Compounds and methods for modulating factor xii
US20220370599A1 (en) * 2021-04-30 2022-11-24 Greenlight Biosciences, Inc. Messenger rna therapeutics and compositions
KR20240023602A (ko) 2021-06-18 2024-02-22 아이오니스 파마수티컬즈, 인코포레이티드 Ifnar1 발현을 감소시키기 위한 화합물 및 방법
KR20240095157A (ko) 2021-07-21 2024-06-25 아큐라스템 인코포레이티드 Unc13a 안티센스 올리고뉴클레오타이드
EP4396352A4 (en) 2021-09-01 2025-10-01 Ionis Pharmaceuticals Inc COMPOUNDS AND METHODS FOR REDUCING DMPK EXPRESSION
WO2023111337A1 (en) * 2021-12-17 2023-06-22 F. Hoffmann-La Roche Ag Antisense oligonucleotide
WO2023139360A1 (en) * 2022-01-19 2023-07-27 Autolus Limited Nucleic acid construct
EP4590841A2 (en) * 2022-09-20 2025-07-30 Aldevron, LLC Best1 vectors and uses thereof
GB202312098D0 (en) * 2023-08-08 2023-09-20 Ucl Business Ltd Therapeutic molecules
WO2025056973A2 (en) * 2023-09-13 2025-03-20 Haya Therapeutics Sa Compositions and methods for modulating wisp2 super-enhancer-associated rna
WO2025072672A2 (en) * 2023-09-27 2025-04-03 Judo Bio, Inc. Slc6a19-targeting modulatory nucleic acid agents
GB202401412D0 (en) * 2024-02-02 2024-03-20 Harness Therapeutics Ltd Functional nucleic acid
WO2026033502A1 (en) * 2024-08-09 2026-02-12 Associação Biopolis Kit, method, and uses thereof

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH04320700A (ja) * 1991-04-17 1992-11-11 Nippon Shoji Kk アポリポプロテインe遺伝子タイプの検査方法及びその検査に好適なプライマー及びプローブ
WO2008103763A2 (en) * 2007-02-20 2008-08-28 Sequenom, Inc. Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation
JP2010516256A (ja) * 2007-01-19 2010-05-20 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン Adrb2癌マーカー
US20120004278A1 (en) * 2010-06-18 2012-01-05 The Board Of Trustees Of The Leland Stanford Junior University Linc rnas in cancer diagnosis and treatment

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2008025069A1 (en) * 2006-08-28 2008-03-06 The Walter And Eliza Hall Institute Of Medical Research Methods of modulating cellular activity and compositions therefor
JP2012503651A (ja) * 2008-09-26 2012-02-09 エージェンシー フォー サイエンス,テクノロジー アンド リサーチ 3−デアザネプラノシン誘導体

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JPH04320700A (ja) * 1991-04-17 1992-11-11 Nippon Shoji Kk アポリポプロテインe遺伝子タイプの検査方法及びその検査に好適なプライマー及びプローブ
JP2010516256A (ja) * 2007-01-19 2010-05-20 ザ リージェンツ オブ ザ ユニバーシティ オブ ミシガン Adrb2癌マーカー
WO2008103763A2 (en) * 2007-02-20 2008-08-28 Sequenom, Inc. Methods and compositions for cancer diagnosis and treatment based on nucleic acid methylation
US20120004278A1 (en) * 2010-06-18 2012-01-05 The Board Of Trustees Of The Leland Stanford Junior University Linc rnas in cancer diagnosis and treatment

Non-Patent Citations (5)

* Cited by examiner, † Cited by third party
Title
KANHERE A. ET AL., MOLECULAR CELL, vol. 38(2010), JPN6017005739, pages 675 - 688, ISSN: 0003506431 *
PRENSNER J.R. ET AL., NAT. BIOTECHNOL., vol. 29(8)(2011), JPN6017005745, pages 742 - 749, ISSN: 0003506433 *
SARMA K. ET AL., PROC. NATL. ACAD. USA, vol. 107(51)(2010), JPN6017005742, pages 22196 - 22201, ISSN: 0003506430 *
ZHAO J. ET AL., SCIENCE, vol. 322(5902)(2008), JPN6017005741, pages 750 - 756, ISSN: 0003506429 *
神武洋二郎 他: "長鎖ncRNAによるクロマチン修飾因子のリクルートメント機構", 実験医学, vol. Vol.29 No.11(2011), JPN7017000587, pages 1716 - 1721, ISSN: 0003506432 *

Cited By (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2021514182A (ja) * 2018-02-09 2021-06-10 ジェネンテック, インコーポレイテッド Tmem106b発現を調節するためのオリゴヌクレオチド
JP7281474B2 (ja) 2018-02-09 2023-05-25 ジェネンテック, インコーポレイテッド Tmem106b発現を調節するためのオリゴヌクレオチド
JP2023503202A (ja) * 2020-02-12 2023-01-26 アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー,リミテッド アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用
JP7321614B2 (ja) 2020-02-12 2023-08-07 アソキュラ・ファーマシューティカルズ・スージョウ・カンパニー,リミテッド アンチセンスオリゴヌクレオチドおよびペンドレッド症候群を処置するためのそれらの使用
JP2023523622A (ja) * 2020-04-29 2023-06-06 サリオジェン セラピューティクス インコーポレイテッド 遺伝性黄斑変性症の治療のための組成物及び方法

Also Published As

Publication number Publication date
US20150141320A1 (en) 2015-05-21
WO2013173637A1 (en) 2013-11-21
EP2850184A4 (en) 2016-01-27
EP2850184A1 (en) 2015-03-25
WO2013173637A8 (en) 2014-12-24

Similar Documents

Publication Publication Date Title
JP2016522674A (ja) 遺伝子発現を調節するための組成物及び方法
JP2016528873A (ja) 遺伝子発現を調節するための組成物及び方法
JP2015518710A (ja) ヘモグロビン遺伝子ファミリー発現を調節するための組成物及び方法
JP2015518714A (ja) 遺伝子発現を調節するための組成物及び方法
JP2015523853A (ja) Atp2a2発現を調節するための組成物及び方法
JP2015518712A (ja) Mecp2発現を調節するための組成物及び方法
JP2015518713A (ja) Utrn発現を調節するための組成物及び方法
JP2015519057A (ja) Pten発現を調節するための組成物及び方法
JP2015523855A (ja) Apoa1及びabca1発現を調節するための組成物及び方法
ES2586593T3 (es) Liposomas de retinoide para potenciar la modulación de la expresión de hsp47
US10041074B2 (en) Euchromatic region targeting methods for modulating gene expression
JP2015518711A (ja) Bdnf発現を調節するための組成物及び方法
JP6129844B2 (ja) 多量体オリゴヌクレオチド化合物
JP2015523854A (ja) Smn遺伝子ファミリー発現を調節するための組成物及び方法
JP2016521556A (ja) Foxp3発現を調節するための組成物及び方法
US20150225722A1 (en) Methods for selective targeting of heterochromatin forming non-coding rna
JP2016534035A (ja) 筋萎縮性側索硬化症を治療するための組成物及び方法
US11464873B2 (en) RNA-modulating agents
US20180312839A1 (en) Methods and compositions for increasing smn expression
CN105658797A (zh) 用于调节rna的组合物和方法
JP2014527819A5 (enExample)
US20180055869A1 (en) Compositions and methods for modulating rna
KR20180057608A (ko) 치료용 올리고뉴클레오타이드
US20200054746A1 (en) Methods for increasing neuronal survival
WO2022026648A1 (en) Inhibition of incexact1 to treat heart disease

Legal Events

Date Code Title Description
A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20150428

A521 Request for written amendment filed

Free format text: JAPANESE INTERMEDIATE CODE: A523

Effective date: 20160513

A621 Written request for application examination

Free format text: JAPANESE INTERMEDIATE CODE: A621

Effective date: 20160513

A131 Notification of reasons for refusal

Free format text: JAPANESE INTERMEDIATE CODE: A131

Effective date: 20170228

A02 Decision of refusal

Free format text: JAPANESE INTERMEDIATE CODE: A02

Effective date: 20171012