EA202192330A1 - Олигонуклеотиды, препятствующие экспрессии белка, ассоциированного с синдромом ломкой х-хромосомы, и способы их применения - Google Patents
Олигонуклеотиды, препятствующие экспрессии белка, ассоциированного с синдромом ломкой х-хромосомы, и способы их примененияInfo
- Publication number
- EA202192330A1 EA202192330A1 EA202192330A EA202192330A EA202192330A1 EA 202192330 A1 EA202192330 A1 EA 202192330A1 EA 202192330 A EA202192330 A EA 202192330A EA 202192330 A EA202192330 A EA 202192330A EA 202192330 A1 EA202192330 A1 EA 202192330A1
- Authority
- EA
- Eurasian Patent Office
- Prior art keywords
- fmrp
- methods
- fragile
- syndrome
- treatment
- Prior art date
Links
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 3
- 208000001914 Fragile X syndrome Diseases 0.000 title abstract 2
- 238000000034 method Methods 0.000 title abstract 2
- 102000004169 proteins and genes Human genes 0.000 title abstract 2
- 108090000623 proteins and genes Proteins 0.000 title abstract 2
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 title 1
- 230000002452 interceptive effect Effects 0.000 title 1
- 102000007338 Fragile X Mental Retardation Protein Human genes 0.000 abstract 4
- 108010032606 Fragile X Mental Retardation Protein Proteins 0.000 abstract 4
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 3
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 3
- 201000010099 disease Diseases 0.000 abstract 3
- 208000037265 diseases, disorders, signs and symptoms Diseases 0.000 abstract 3
- 108020000948 Antisense Oligonucleotides Proteins 0.000 abstract 1
- 206010009900 Colitis ulcerative Diseases 0.000 abstract 1
- 206010009944 Colon cancer Diseases 0.000 abstract 1
- 208000011231 Crohn disease Diseases 0.000 abstract 1
- 208000022559 Inflammatory bowel disease Diseases 0.000 abstract 1
- 208000015634 Rectal Neoplasms Diseases 0.000 abstract 1
- 201000006704 Ulcerative Colitis Diseases 0.000 abstract 1
- 210000001072 colon Anatomy 0.000 abstract 1
- 208000029742 colonic neoplasm Diseases 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 210000000936 intestine Anatomy 0.000 abstract 1
- 238000004519 manufacturing process Methods 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
- 206010038038 rectal cancer Diseases 0.000 abstract 1
- 201000001275 rectum cancer Diseases 0.000 abstract 1
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
-
- 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
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)
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 |
|---|---|
| EA202192330A1 true EA202192330A1 (ru) | 2021-12-13 |
Family
ID=69723929
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| EA202192330A EA202192330A1 (ru) | 2019-02-26 | 2020-02-26 | Олигонуклеотиды, препятствующие экспрессии белка, ассоциированного с синдромом ломкой х-хромосомы, и способы их применения |
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)
| 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)
| 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 |
-
2020
- 2020-02-26 CA CA3130854A patent/CA3130854A1/en active Pending
- 2020-02-26 SG SG11202109112WA patent/SG11202109112WA/en unknown
- 2020-02-26 EP EP20707610.0A patent/EP3931324A1/en not_active Withdrawn
- 2020-02-26 KR KR1020217030136A patent/KR20210132678A/ko not_active Abandoned
- 2020-02-26 MX MX2021010255A patent/MX2021010255A/es unknown
- 2020-02-26 US US17/433,528 patent/US20220145303A1/en not_active Abandoned
- 2020-02-26 CN CN202080031598.4A patent/CN113748207A/zh active Pending
- 2020-02-26 AU AU2020228134A patent/AU2020228134A1/en not_active Abandoned
- 2020-02-26 JP JP2021547879A patent/JP2022521502A/ja not_active Ceased
- 2020-02-26 WO PCT/EP2020/055071 patent/WO2020174023A1/en not_active Ceased
- 2020-02-26 EA EA202192330A patent/EA202192330A1/ru unknown
- 2020-02-26 BR BR112021016907A patent/BR112021016907A2/pt not_active IP Right Cessation
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020174023A1 (en) | 2020-09-03 |
| CA3130854A1 (en) | 2020-09-03 |
| 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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