BR112021016907A2 - Oligonucleotídeos de interferência com proteína de retardo mental do x frágil e métodos de sua utilização - Google Patents

Oligonucleotídeos de interferência com proteína de retardo mental do x frágil e métodos de sua utilização

Info

Publication number
BR112021016907A2
BR112021016907A2 BR112021016907A BR112021016907A BR112021016907A2 BR 112021016907 A2 BR112021016907 A2 BR 112021016907A2 BR 112021016907 A BR112021016907 A BR 112021016907A BR 112021016907 A BR112021016907 A BR 112021016907A BR 112021016907 A2 BR112021016907 A2 BR 112021016907A2
Authority
BR
Brazil
Prior art keywords
methods
fragile
fmrp
mental retardation
retardation protein
Prior art date
Application number
BR112021016907A
Other languages
English (en)
Portuguese (pt)
Inventor
Francesca Viti
Salvatore Bellinvia
Original Assignee
Nogra Pharma Ltd
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 Nogra Pharma Ltd filed Critical Nogra Pharma Ltd
Publication of BR112021016907A2 publication Critical patent/BR112021016907A2/pt

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    • 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
    • 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
    • C12N15/1135Non-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
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00Drugs for disorders of the alimentary tract or the digestive system
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P29/00Non-central analgesic, antipyretic or antiinflammatory agents, e.g. antirheumatic agents; Non-steroidal antiinflammatory drugs [NSAID]
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P35/00Antineoplastic agents
    • 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
    • C12N2310/11Antisense
    • 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
    • C12N2310/30Chemical structure
    • C12N2310/31Chemical structure of the backbone
    • C12N2310/312Phosphonates
    • C12N2310/3125Methylphosphonates
    • 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
    • C12N2310/33Chemical structure of the base
    • C12N2310/334Modified C
    • C12N2310/33415-Methylcytosine

Landscapes

  • 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)
BR112021016907A 2019-02-26 2020-02-26 Oligonucleotídeos de interferência com proteína de retardo mental do x frágil e métodos de sua utilização BR112021016907A2 (pt)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201962810697P 2019-02-26 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
BR112021016907A2 true BR112021016907A2 (pt) 2021-11-03

Family

ID=69723929

Family Applications (1)

Application Number Title Priority Date Filing Date
BR112021016907A BR112021016907A2 (pt) 2019-02-26 2020-02-26 Oligonucleotídeos de interferência com proteína de retardo mental do x frágil e métodos de sua utilização

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
CA3130854A1 (en) 2020-09-03
EP3931324A1 (en) 2022-01-05
MX2021010255A (es) 2021-12-15
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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B08F Application dismissed because of non-payment of annual fees [chapter 8.6 patent gazette]

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B08K Patent lapsed as no evidence of payment of the annual fee has been furnished to inpi [chapter 8.11 patent gazette]

Free format text: EM VIRTUDE DO ARQUIVAMENTO PUBLICADO NA RPI 2868 DE 23-12-2025 E CONSIDERANDO AUSENCIA DE MANIFESTACAO DENTRO DOS PRAZOS LEGAIS, INFORMO QUE CABE SER MANTIDO O ARQUIVAMENTO DO PEDIDO DE PATENTE, CONFORME O DISPOSTO NO ARTIGO 12, DA RESOLUCAO 113/2013.