CA2979825A1 - Mirna mimetics and their use in treating sensory conditions - Google Patents

Mirna mimetics and their use in treating sensory conditions Download PDF

Info

Publication number
CA2979825A1
CA2979825A1 CA2979825A CA2979825A CA2979825A1 CA 2979825 A1 CA2979825 A1 CA 2979825A1 CA 2979825 A CA2979825 A CA 2979825A CA 2979825 A CA2979825 A CA 2979825A CA 2979825 A1 CA2979825 A1 CA 2979825A1
Authority
CA
Canada
Prior art keywords
mir
strand
sequence
seq
mimetic compound
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
CA2979825A
Other languages
English (en)
French (fr)
Inventor
Aimee Jackson
Christina Dalby
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.)
Viridian Therapeutics Inc
Original Assignee
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 Miragen Therapeutics Inc filed Critical Miragen Therapeutics Inc
Publication of CA2979825A1 publication Critical patent/CA2979825A1/en
Abandoned 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
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/70Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088Compounds having three or more nucleosides or nucleotides
    • A61K31/7105Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/02Ophthalmic agents
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00Drugs for disorders of the senses
    • A61P27/16Otologicals
    • 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
    • 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
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • 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
    • C12N2310/14Type of nucleic acid interfering nucleic acids [NA]
    • C12N2310/141MicroRNAs, miRNAs
    • 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
    • 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
    • C12N2310/35Nature of the modification
    • C12N2310/351Conjugate
    • C12N2310/3515Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/00Applications; Uses
    • C12N2320/30Special therapeutic applications
    • C12N2320/31Combination therapy

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Molecular Biology (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Organic Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Wood Science & Technology (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Ophthalmology & Optometry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Peptides Or Proteins (AREA)
CA2979825A 2015-03-16 2016-03-16 Mirna mimetics and their use in treating sensory conditions Abandoned CA2979825A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201562133590P 2015-03-16 2015-03-16
US62/133,590 2015-03-16
PCT/US2016/022645 WO2016149370A1 (en) 2015-03-16 2016-03-16 Mirna mimetics and their use in treating sensory conditions

Publications (1)

Publication Number Publication Date
CA2979825A1 true CA2979825A1 (en) 2016-09-22

Family

ID=56919428

Family Applications (1)

Application Number Title Priority Date Filing Date
CA2979825A Abandoned CA2979825A1 (en) 2015-03-16 2016-03-16 Mirna mimetics and their use in treating sensory conditions

Country Status (7)

Country Link
US (1) US20180080023A1 (enExample)
EP (1) EP3271464A4 (enExample)
JP (1) JP2018509912A (enExample)
CN (1) CN107532181A (enExample)
AU (1) AU2016233311A1 (enExample)
CA (1) CA2979825A1 (enExample)
WO (1) WO2016149370A1 (enExample)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2019195765A1 (en) 2018-04-06 2019-10-10 University Of Massachusetts Mlk-regulated micrornas in angiogenesis and tumor development
CN110013486B (zh) * 2019-03-27 2021-08-17 浙江大学 mmu-miR-183-5p在制备抑制胚胎着床的药物中的应用

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP2530157B1 (en) * 2003-07-31 2016-09-28 Regulus Therapeutics Inc. Oligomeric compounds and compositions for use in modulation of miRNAs
ES2873350T3 (es) * 2007-08-27 2021-11-03 1Globe Health Inst Llc Composiciones de ARN interferente asimétrico y usos de las mismas
WO2009137807A2 (en) * 2008-05-08 2009-11-12 Asuragen, Inc. Compositions and methods related to mirna modulation of neovascularization or angiogenesis
WO2010027838A1 (en) * 2008-08-27 2010-03-11 The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Mir 204, mir 211, their anti-mirs, and therapeutic uses of same
US9096850B2 (en) * 2009-08-24 2015-08-04 Sirna Therapeutics, Inc. Segmented micro RNA mimetics
WO2014100252A1 (en) * 2012-12-18 2014-06-26 University Of Washington Through Its Center For Commercialization Methods and compositions to modulate rna processing
US20150051267A1 (en) * 2013-08-16 2015-02-19 Purdue Research Foundation BICISTRONIC GENE TRANSFER TOOLS FOR DELIVERY OF miRNAS AND PROTEIN CODING SEQUENCES
US20160304865A1 (en) * 2013-09-26 2016-10-20 Friedrich Miescher Institute For Biomedical Research Tools and methods using mirna 182, 96 and/or 183 for treating pathologies
GB201400598D0 (en) * 2014-01-14 2014-03-05 Univ Glasgow Materials and methods for modulation of tendon healing

Also Published As

Publication number Publication date
WO2016149370A1 (en) 2016-09-22
AU2016233311A1 (en) 2017-10-05
JP2018509912A (ja) 2018-04-12
US20180080023A1 (en) 2018-03-22
EP3271464A4 (en) 2018-10-31
CN107532181A (zh) 2018-01-02
EP3271464A1 (en) 2018-01-24

Similar Documents

Publication Publication Date Title
TWI833770B (zh) 用於減少 lrrk2 表現之化合物及方法
CA3099522C (en) GAPMERES AND METHODS OF USING THESE FOR THE TREATMENT OF MUSCULAR DYSTROPHY
JP5916660B2 (ja) ハンチンチン対する、組成物及びその使用
ES2984631T3 (es) Composiciones y procedimientos para disminuir la expresión de tau
JP2023529316A (ja) ゲノム編集のための組成物及び方法
EA035433B1 (ru) Модуляторы фактора в комплемента
BR112014008925A2 (pt) micro rnas em distúrbios de neurodegenerativos
TR201816256T4 (tr) Bir süjede smn2 uç birleştirmesinin modülasyonu için bileşimler ve yöntemler.
TW201143780A (en) Treatment of Colony-stimulating factor 3 (CSF3) related diseases by inhibition of natural antisense transcript to CSF3
JP2016013125A (ja) サーチュイン(sirt)に対する天然アンチセンス転写物の阻害によるサーチュイン(sirt)関連疾患の治療
AU2020252710B8 (en) Compound, method and pharmaceutical composition for modulating expression of dux4
TW201219569A (en) Treatment of Sialidase 4 (NEU4) related diseases by inhibition of natural antisense transcript to NEU4
US11629347B2 (en) Anti-C9ORF72 oligonucleotides and related methods
JP2024516356A (ja) ケトヘキソキナーゼ(khk)を阻害するための組成物及び方法
JP7560616B2 (ja) 膜貫通型セリンプロテアーゼ6(tmprss6)発現を阻害するための組成物および方法
KR20230076823A (ko) 시신경 위축의 치료
CN115397436A (zh) 用于抑制PNPLA3表达的RNAi剂、其药物组合物和使用方法
JP2025100729A (ja) 補体成分3の発現を阻害するための組成物及び方法
ES2909308T3 (es) Métodos para modular la expresión de MECP2
BR112020016170A2 (pt) Oligonucleotídeos terapêutico e antissenso, conjugado, sal farmaceuticamente aceitável, composição farmacêutica, método in vitro ou in vivo para modular a expressão de tmem106b, método para tratar ou prevenir uma doença, uso do oligonucleotídeo e uso ou método
US8450473B2 (en) Compositions and methods for therapy of macular degeneration
US20180080023A1 (en) Mirna mimetics and their use in treating sensory conditions
Guncay Evaluation of LNA Gapmer efficacy in FSHD patients' muscle cells
HK40084771A (en) Rnai agents for inhibiting expression of pnpla3, pharmaceutical compositions thereof, and methods of use

Legal Events

Date Code Title Description
FZDE Discontinued

Effective date: 20220607

FZDE Discontinued

Effective date: 20220607