AU2016233311A1 - 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
AU2016233311A1
AU2016233311A1 AU2016233311A AU2016233311A AU2016233311A1 AU 2016233311 A1 AU2016233311 A1 AU 2016233311A1 AU 2016233311 A AU2016233311 A AU 2016233311A AU 2016233311 A AU2016233311 A AU 2016233311A AU 2016233311 A1 AU2016233311 A1 AU 2016233311A1
Authority
AU
Australia
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
AU2016233311A
Other languages
English (en)
Inventor
Christina Dalby
Aimee Jackson
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 AU2016233311A1 publication Critical patent/AU2016233311A1/en
Abandoned legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • A61K31/7105—Natural ribonucleic acids, i.e. containing only riboses attached to adenine, guanine, cytosine or uracil and having 3'-5' phosphodiester links
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • 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]
    • 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
    • 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
    • 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
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3515—Lipophilic moiety, e.g. cholesterol
    • 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
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination 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)
AU2016233311A 2015-03-16 2016-03-16 MiRNA mimetics and their use in treating sensory conditions Abandoned AU2016233311A1 (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
AU2016233311A1 true AU2016233311A1 (en) 2017-10-05

Family

ID=56919428

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2016233311A Abandoned AU2016233311A1 (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
CA2979825A1 (en) 2016-09-22
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
CA3099522C (en) GAPMERES AND METHODS OF USING THESE FOR THE TREATMENT OF MUSCULAR DYSTROPHY
TWI833770B (zh) 用於減少 lrrk2 表現之化合物及方法
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
EA035433B1 (ru) Модуляторы фактора в комплемента
JP2019500345A (ja) 肝臓病の処置のための組成物および方法
TW201143780A (en) Treatment of Colony-stimulating factor 3 (CSF3) related diseases by inhibition of natural antisense transcript to CSF3
BR112014008925A2 (pt) micro rnas em distúrbios de neurodegenerativos
TR201816256T4 (tr) Bir süjede smn2 uç birleştirmesinin modülasyonu için bileşimler ve yöntemler.
CA2761248A1 (en) Treatment of dystrophin family related diseases by inhibition of natural antisense transcript to dmd family
US12378554B2 (en) Anti-C9ORF72 oligonucleotides and related methods
JP2025100705A (ja) Ｄｕｘ４の発現を調節するための化合物、方法及び医薬組成物
TWI769197B (zh) 用於治療多囊腎病之組成物
TW202202153A (zh) 用於抑制ＰＮＰＬＡ３表現之ＲＮＡｉ劑、其醫藥組成物及使用方法
AU2016233311A1 (en) MiRNA mimetics and their use in treating sensory conditions
CN113891940B (zh) 视网膜色素变性的新治疗方法
JP2023528435A (ja) 遺伝子転写物のモジュレーターを使用する神経学的疾患の処置
US20260009032A1 (en) Anti-c9orf72 oligonucleotides and related methods
TW202430635A (zh) 用於抑制補體成分C3(C3)表現之RNAi藥劑、其醫藥組合物及使用方法
Guncay Evaluation of LNA Gapmer efficacy in FSHD patients' muscle cells
WO2021062251A2 (en) Targeting rlim to modulate body weight and obesity
HK40084771A (en) Rnai agents for inhibiting expression of pnpla3, pharmaceutical compositions thereof, and methods of use

Legal Events

Date Code Title Description
MK1 Application lapsed section 142(2)(a) - no request for examination in relevant period