CA2986913A1 - Mir-155 inhibitors for treating amyotrophic lateral sclerosis (als) - Google Patents

Mir-155 inhibitors for treating amyotrophic lateral sclerosis (als)

Info

Publication number
CA2986913A1
CA2986913A1 CA2986913A CA2986913A CA2986913A1 CA 2986913 A1 CA2986913 A1 CA 2986913A1 CA 2986913 A CA2986913 A CA 2986913A CA 2986913 A CA2986913 A CA 2986913A CA 2986913 A1 CA2986913 A1 CA 2986913A1
Authority
CA
Canada
Prior art keywords
oligonucleotide inhibitor
mir
cells
nucleotide
cns
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.)
Abandoned
Application number
CA2986913A
Other languages
English (en)
French (fr)
Inventor
Aimee Jackson
Christina Dalby
Howard L. Weiner
Oleg Butovsky
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.)
Brigham and Womens Hospital Inc
Viridian Therapeutics Inc
Original Assignee
Brigham and Womens Hospital Inc
Miragen Therapeutics 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
Application filed by Brigham and Womens Hospital Inc, Miragen Therapeutics Inc filed Critical Brigham and Womens Hospital Inc
Publication of CA2986913A1 publication Critical patent/CA2986913A1/en
Abandoned legal-status Critical Current

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
    • 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
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • 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
    • C12N2310/30—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
    • C12N2310/323—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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Biomedical Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Biochemistry (AREA)
  • Organic Chemistry (AREA)
  • Epidemiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Zoology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Psychiatry (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Microbiology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
CA2986913A 2015-06-05 2016-06-03 Mir-155 inhibitors for treating amyotrophic lateral sclerosis (als) Abandoned CA2986913A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562171743P 2015-06-05 2015-06-05
US62/171,743 2015-06-05
PCT/US2016/035794 WO2016196978A1 (en) 2015-06-05 2016-06-03 Mir-155 inhibitors for treating amyotrophic lateral sclerosis (als)

Publications (1)

Publication Number Publication Date
CA2986913A1 true CA2986913A1 (en) 2016-12-08

Family

ID=57442041

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2986913A Abandoned CA2986913A1 (en) 2015-06-05 2016-06-03 Mir-155 inhibitors for treating amyotrophic lateral sclerosis (als)

Country Status (6)

Country Link
US (1) US20180161357A1 (enExample)
EP (1) EP3303589A4 (enExample)
JP (1) JP2018517704A (enExample)
CN (1) CN107922947A (enExample)
CA (1) CA2986913A1 (enExample)
WO (1) WO2016196978A1 (enExample)

Families Citing this family (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2013055865A1 (en) 2011-10-11 2013-04-18 The Brigham And Women's Hospital, Inc. Micrornas in neurodegenerative disorders
RU2718534C2 (ru) 2015-06-05 2020-04-08 Мираджен Терапьютикс, Инк. Ингибиторы mir-155 для лечения кожной t-клеточной лимфомы (ctcl)
EP3526319B1 (en) 2016-10-14 2025-12-10 Children's Medical Center Corporation Compositions and methods for treating diseases and disorders of the central nervous system
EP3571310A4 (en) 2017-01-17 2020-12-16 Children's Medical Center Corporation COMPOSITIONS AND METHODS FOR DIAGNOSIS AND TREATMENT OF PEROXYSOMAL DISEASES
CA3050691A1 (en) 2017-01-17 2018-07-26 Dana-Farber Cancer Institute, Inc. Compositions and methods for treating lysosomal storage diseases and disorders
US12144822B2 (en) 2018-04-03 2024-11-19 Academia Sinica Mir-17˜92 as therapeutic or diagnostic target of motor neuron (MN) degeneration diseases
JP7499754B2 (ja) * 2018-08-23 2024-06-14 ロシュ・イノベーション・センター・コペンハーゲン・アクティーゼルスカブ Ｍｉｃｒｏｒｎａ－１３４バイオマーカー
WO2021067613A1 (en) * 2019-10-01 2021-04-08 Children's Medical Center Corporation Compositions and methods for treating amyotrophic lateral sclerosis
CN114272378B (zh) * 2020-09-27 2023-06-23 四川大学华西医院 一种使cttnbp2nl功能缺失的试剂在制备治疗疾病的药物中的用途
CN112587662A (zh) * 2020-12-22 2021-04-02 上海市徐汇区中心医院 一种miR-155/PEA15信号通路抑制剂在病理性心脏重构药物中的应用

Family Cites Families (7)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN101437942A (zh) * 2006-04-03 2009-05-20 桑塔里斯制药公司 包含抗微小rna反义寡核苷酸的药物组合物
MX2008012219A (es) * 2006-04-03 2008-10-02 Santaris Pharma As Composicion farmaceutica que comprende oligonucleotidos antisentido anti-miarn.
ES2406686T3 (es) * 2007-10-04 2013-06-07 Santaris Pharma A/S Micromirs
EP2268811A1 (en) * 2008-03-07 2011-01-05 Santaris Pharma A/S Pharmaceutical compositions for treatment of microrna related diseases
US9493834B2 (en) * 2009-07-29 2016-11-15 Pharnext Method for detecting a panel of biomarkers
WO2013055865A1 (en) * 2011-10-11 2013-04-18 The Brigham And Women's Hospital, Inc. Micrornas in neurodegenerative disorders
WO2013134403A1 (en) * 2012-03-06 2013-09-12 The Washington University Method of treating neurodegenerative diseases with microrna regulators

Also Published As

Publication number Publication date
EP3303589A1 (en) 2018-04-11
WO2016196978A1 (en) 2016-12-08
US20180161357A1 (en) 2018-06-14
JP2018517704A (ja) 2018-07-05
CN107922947A (zh) 2018-04-17
EP3303589A4 (en) 2019-01-02

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Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20220301