CA3130854A1 - Fragile x mental retardation protein interfering oligonucleotides and methods of using same - Google Patents

Fragile x mental retardation protein interfering oligonucleotides and methods of using same Download PDF

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Publication number
CA3130854A1
CA3130854A1 CA3130854A CA3130854A CA3130854A1 CA 3130854 A1 CA3130854 A1 CA 3130854A1 CA 3130854 A CA3130854 A CA 3130854A CA 3130854 A CA3130854 A CA 3130854A CA 3130854 A1 CA3130854 A1 CA 3130854A1
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Canada
Prior art keywords
antisense oligonucleotide
linkage
fmrp
seq
sirna
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
CA3130854A
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English (en)
French (fr)
Inventor
Francesca Viti
Salvatore Bellinvia
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.)
Nogra Pharma Ltd
Original Assignee
Nogra Pharma Ltd
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Filing date
Publication date
Application filed by Nogra Pharma Ltd filed Critical Nogra Pharma Ltd
Publication of CA3130854A1 publication Critical patent/CA3130854A1/en
Pending legal-status Critical Current

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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
    • 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
    • C12N15/1135—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 against oncogenes or tumor suppressor genes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00—Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00—Antineoplastic agents
    • 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
    • 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
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/312—Phosphonates
    • C12N2310/3125—Methylphosphonates
    • 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
    • C12N2310/33—Chemical structure of the base
    • C12N2310/334—Modified C
    • C12N2310/3341—5-Methylcytosine

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Medicinal Chemistry (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Biochemistry (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Pain & Pain Management (AREA)
  • Rheumatology (AREA)
  • Oncology (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA3130854A 2019-02-26 2020-02-26 Fragile x mental retardation protein interfering oligonucleotides and methods of using same Pending CA3130854A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962810697P 2019-02-26 2019-02-26
US62/810,697 2019-02-26
PCT/EP2020/055071 WO2020174023A1 (en) 2019-02-26 2020-02-26 Fragile x mental retardation protein interfering oligonucleotides and methods of using same

Publications (1)

Publication Number Publication Date
CA3130854A1 true CA3130854A1 (en) 2020-09-03

Family

ID=69723929

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3130854A Pending CA3130854A1 (en) 2019-02-26 2020-02-26 Fragile x mental retardation protein interfering oligonucleotides and methods of using same

Country Status (12)

Country Link
US (1) US20220145303A1 (https=)
EP (1) EP3931324A1 (https=)
JP (1) JP2022521502A (https=)
KR (1) KR20210132678A (https=)
CN (1) CN113748207A (https=)
AU (1) AU2020228134A1 (https=)
BR (1) BR112021016907A2 (https=)
CA (1) CA3130854A1 (https=)
EA (1) EA202192330A1 (https=)
MX (1) MX2021010255A (https=)
SG (1) SG11202109112WA (https=)
WO (1) WO2020174023A1 (https=)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2025501755A (ja) * 2021-12-23 2025-01-23 ユニバーシティー オブ マサチューセッツ 脆弱ｘ関連障害に対する治療的処置
CN121109315A (zh) * 2025-08-28 2025-12-12 同济大学 脆性x智力障碍1号基因在治疗或缓解骨关节炎中的应用

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
RU2593939C2 (ru) 2008-11-13 2016-08-10 Ногра Фарма Лимитед Антисмысловые композиции и способы их получения и применения
US8679757B2 (en) * 2009-03-24 2014-03-25 Asuragen, Inc. PCR methods for characterizing the 5′ untranslated region of the FMR1 and FMR2 genes
EP2488665B1 (en) * 2009-10-16 2017-12-13 National University of Singapore Screening method for trinucleotide repeat sequences
US20120231015A1 (en) * 2009-11-06 2012-09-13 Emory University Fragile x mental retardation protein (fmrp), compositions, and methods related thereto
AU2011273360A1 (en) * 2010-07-02 2013-02-07 Vib Vzw The role of Fragile X mental retardation gene and protein in cancer metastasis
CN103981253A (zh) * 2014-03-27 2014-08-13 江苏佰龄全基因生物医学技术有限公司 一种用于检测脆性x染色体综合征的cgg重复数及agg插入信息的pcr试剂盒
WO2017049192A1 (en) * 2015-09-17 2017-03-23 University Of Massachusetts Compositions and methods for modulating fmr1 expression
EP3368089B1 (en) * 2015-10-26 2025-11-05 Translate Bio Ma, Inc. Nanoparticle formulations for delivery of nucleic acid complexes
WO2019154884A1 (en) * 2018-02-07 2019-08-15 Ecole Polytechnique Federale De Lausanne (Epfl) Method for determining cancer invasiveness and patient prognosis
CN108531576A (zh) * 2018-04-12 2018-09-14 北京信诺佰世医学检验所有限公司 检测脆性x染色体综合征的试剂盒和系统

Also Published As

Publication number Publication date
WO2020174023A1 (en) 2020-09-03
EA202192330A1 (ru) 2021-12-13
EP3931324A1 (en) 2022-01-05
MX2021010255A (es) 2021-12-15
BR112021016907A2 (pt) 2021-11-03
CN113748207A (zh) 2021-12-03
US20220145303A1 (en) 2022-05-12
JP2022521502A (ja) 2022-04-08
SG11202109112WA (en) 2021-09-29
AU2020228134A1 (en) 2021-10-14
KR20210132678A (ko) 2021-11-04

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