CA3037288C - Modulation of micrornas against myotonic dystrophy type 1 and antagonists of micrornas therefor - Google Patents

Modulation of micrornas against myotonic dystrophy type 1 and antagonists of micrornas therefor Download PDF

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Publication number
CA3037288C
CA3037288C CA3037288A CA3037288A CA3037288C CA 3037288 C CA3037288 C CA 3037288C CA 3037288 A CA3037288 A CA 3037288A CA 3037288 A CA3037288 A CA 3037288A CA 3037288 C CA3037288 C CA 3037288C
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Prior art keywords
mir
oligoribonucleotide
seq
expression
molecule
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CA3037288A
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English (en)
French (fr)
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CA3037288A1 (en
Inventor
Ruben D. Artero Allepuz
Maria Beatriz Llamusi Troisi
Estefania Cerro Herreros
Juan M. FERNANDEZ COSTA
Veenu AISHWARYA
Thorleif Moller
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Universitat de Valencia
Aum Life Tech Inc
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Universitat de Valencia
Aum Life Tech Inc
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    • 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
    • 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
    • A61K31/7115—Nucleic acids or oligonucleotides having modified bases, i.e. other than adenine, guanine, cytosine, uracil or thymine
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P21/00—Drugs for disorders of the muscular or neuromuscular system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • 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
    • C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
    • 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
    • C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141—MicroRNAs, miRNAs

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biochemistry (AREA)
  • Biotechnology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Psychology (AREA)
  • Orthopedic Medicine & Surgery (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CA3037288A 2016-09-19 2017-09-19 Modulation of micrornas against myotonic dystrophy type 1 and antagonists of micrornas therefor Active CA3037288C (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
ES201631216A ES2659845B1 (es) 2016-09-19 2016-09-19 Modulación de microRNAs contra la distrofia miotónica tipo 1 y antagonistas de microRNAs para ello
ES201631216 2016-09-19
PCT/EP2017/073685 WO2018050930A1 (en) 2016-09-19 2017-09-19 Modulation of micrornas against myotonic dystrophy type 1 and antagonists of micrornas therefor

Publications (2)

Publication Number Publication Date
CA3037288A1 CA3037288A1 (en) 2018-03-22
CA3037288C true CA3037288C (en) 2024-01-16

Family

ID=59974404

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3037288A Active CA3037288C (en) 2016-09-19 2017-09-19 Modulation of micrornas against myotonic dystrophy type 1 and antagonists of micrornas therefor

Country Status (8)

Country Link
US (2) US11202794B2 (OSRAM)
EP (2) EP3516059A1 (OSRAM)
JP (1) JP6919098B2 (OSRAM)
AU (2) AU2017328728B2 (OSRAM)
CA (1) CA3037288C (OSRAM)
ES (1) ES2659845B1 (OSRAM)
IL (1) IL265461B2 (OSRAM)
WO (1) WO2018050930A1 (OSRAM)

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* Cited by examiner, † Cited by third party
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JP7221275B2 (ja) 2017-08-03 2023-02-13 ボイジャー セラピューティクス インコーポレイテッド Ａａｖを送達するための組成物および方法
JP7432929B2 (ja) * 2018-05-31 2024-02-19 コリア ユニバーシティ リサーチ アンド ビジネス ファウンデーション マイクロｒｎａの非正規標的を抑制するｒｎａ干渉誘導核酸およびその用途
US20210340537A1 (en) * 2020-04-15 2021-11-04 Duquesne University Of The Holy Spirit AN RNA G-QUADRUPLEX STRUCTURE IN PRE-miRNA-1229 AS A THERAPEUTIC TARGET FOR ALZHEIMER'S DISEASE AND VARIOUS CANCERS
ES2954008A1 (es) * 2022-04-06 2023-11-17 Univ Valencia Antagonistas de los microRNA humanos miR-100, miR-20, miR-222, miR-181, y miR-92 y usos de los mismos
EP4506010A1 (en) 2022-04-06 2025-02-12 Universitat de València Antagonists of human micro rna mir-100, mir-20, mir-222, mir-181 and mir-92 and uses of same
EP4282964A1 (en) * 2022-05-23 2023-11-29 Universitat de Valéncia Oligonucleotides conjugated to oleic acid and uses thereof
ES3014830A1 (es) * 2023-10-20 2025-04-25 Arthex Biotech S L Oligonucleotidos o analogos de los mismos para el tratamiento de la distrofia del endotelio corneal de fuchs
CN117467664B (zh) * 2023-11-07 2024-06-21 江苏省家禽科学研究所 一种靶向鸡mbnl1基因的反义寡核苷酸及其应用
WO2025114442A1 (en) * 2023-11-28 2025-06-05 Arthex Biotech S.L. Oligonucleotides conjugated to oleic acid and uses thereof

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US5981505A (en) 1993-01-26 1999-11-09 The Trustees Of The University Of Pennsylvania Compositions and methods for delivery of genetic material
US5837533A (en) 1994-09-28 1998-11-17 American Home Products Corporation Complexes comprising a nucleic acid bound to a cationic polyamine having an endosome disruption agent
US5840710A (en) 1994-12-09 1998-11-24 Genzyme Corporation Cationic amphiphiles containing ester or ether-linked lipophilic groups for intracellular delivery of therapeutic molecules
US6217900B1 (en) 1997-04-30 2001-04-17 American Home Products Corporation Vesicular complexes and methods of making and using the same
AU731909B2 (en) 1997-07-01 2001-04-05 Isis Pharmaceuticals, Inc. Compositions and methods for the delivery of oligonucleotides via the alimentary canal
EP1549352A4 (en) 2002-05-06 2005-07-27 Nucleonics Inc METHOD OF DISTRIBUTING NUCLEIC ACIDS
EP1648914A4 (en) * 2003-07-31 2009-12-16 Regulus Therapeutics Inc OLIGOMERIC COMPOUNDS AND COMPOSITIONS USEFUL FOR MODULATING SMALL NON-CODING RNA
CA2556435C (en) * 2004-02-13 2014-08-12 The Rockefeller University Anti-microrna oligonucleotide molecules
EP1838870A2 (en) * 2004-12-29 2007-10-03 Exiqon A/S NOVEL OLIGONUCLEOTIDE COMPOSITIONS AND PROBE SEQUENCES USEFUL FOR DETECTION AND ANALYSIS OF MICRORNAS AND THEIR TARGET MRNAs
AU2007211082B2 (en) * 2006-01-27 2012-09-27 Isis Pharmaceuticals, Inc. Oligomeric compounds and compositions for the use in modulation of microRNAs
CA3024953A1 (en) * 2006-04-03 2007-10-11 Roche Innovation Center Copenhagen A/S Pharmaceutical composition comprising anti-mirna antisense oligonucleotides
EP3034083B1 (en) * 2006-09-21 2020-12-09 University of Rochester Antisense oligonucleotides for use in treating myotonic dystrophy
WO2008116267A1 (en) * 2007-03-26 2008-10-02 Crc For Asthma And Airways Ltd Therapeutic targets and molecules
WO2009012263A2 (en) * 2007-07-18 2009-01-22 The Trustees Of Columbia University In The City Of New York Tissue-specific micrornas and compositions and uses thereof
AU2009256243A1 (en) * 2008-06-04 2009-12-10 The Board Of Regents Of The University Of Texas System Modulation of gene expression through endogenous small RNA targeting of gene promoters
EP2298359A1 (en) * 2008-06-04 2011-03-23 Kyowa Hakko Kirin Co., Ltd. Nucleic acid capable of controlling degranulation of mast cell
EP2346905A4 (en) * 2008-10-15 2012-03-07 4S3 Bioscience Inc METHODS AND COMPOSITIONS FOR TREATING MYOTONIC DYSTROPHY
US8852940B2 (en) 2009-04-01 2014-10-07 The Regents Of The University Of California Embryonic stem cell specific microRNAs promote induced pluripotency
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EP2275545A1 (en) * 2009-07-16 2011-01-19 Julius-Maximilians-Universität Würzburg Use of microRNA-24 and/or its targets for the treatment and prevention of ischemia and induction of angiogenesis
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Also Published As

Publication number Publication date
AU2017328728A1 (en) 2019-05-16
EP4269587A2 (en) 2023-11-01
ES2659845B1 (es) 2019-01-04
AU2021257890A1 (en) 2021-11-18
CA3037288A1 (en) 2018-03-22
JP6919098B2 (ja) 2021-08-18
IL265461B1 (en) 2024-02-01
EP3516059A1 (en) 2019-07-31
JP2019531092A (ja) 2019-10-31
US20220133774A1 (en) 2022-05-05
IL265461B2 (en) 2024-06-01
ES2659845A1 (es) 2018-03-19
US20190231809A1 (en) 2019-08-01
IL265461A (en) 2019-05-30
WO2018050930A1 (en) 2018-03-22
EP4269587A3 (en) 2024-07-10
AU2017328728B2 (en) 2021-07-29
US11202794B2 (en) 2021-12-21

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