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 utilizaçãoInfo
- 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
Links
- 108091034117 Oligonucleotide Proteins 0.000 title abstract 5
- 102000007338 Fragile X Mental Retardation Protein Human genes 0.000 title abstract 3
- 108010032606 Fragile X Mental Retardation Protein Proteins 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 3
- 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 abstract 2
- 230000002452 interceptive effect Effects 0.000 title abstract 2
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 3
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 3
- 208000028774 intestinal disease Diseases 0.000 abstract 3
- 206010009900 Colitis ulcerative Diseases 0.000 abstract 1
- 206010009944 Colon cancer Diseases 0.000 abstract 1
- 208000001333 Colorectal Neoplasms Diseases 0.000 abstract 1
- 208000011231 Crohn disease Diseases 0.000 abstract 1
- 208000022559 Inflammatory bowel disease Diseases 0.000 abstract 1
- 201000006704 Ulcerative Colitis Diseases 0.000 abstract 1
- 239000003814 drug Substances 0.000 abstract 1
- 230000000694 effects Effects 0.000 abstract 1
- 238000004519 manufacturing process Methods 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
Classifications
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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 N.A.
-
- 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)
Abstract
oligonucleotídeos de interferência com proteína de retardo mental do x frágil e métodos de sua utilização. são reveladas nesse relatório descritivo sequências de oligonucleotídeo anti-senso contra proteína de retardo mental do x frágil (fmrp) e métodos de sua utilização para o tratamento de doenças intestinais como, por exemplo, câncer cólon-retal e doença intestinal inflamatória (por exemplo, doença de crohn e colite ulcerativa), associadas com atividade ou expressão elevada de fmrp. também são reveladas composições farmacêuticas que contêm um oligonucleotídeo anti-senso fmrp útil para o tratamento de uma doença intestinal e fabricação de medicamentos que contêm um oligonucleotídeo anti-senso fmrp revelado para ser usado no tratamento de uma doença intestinal.
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 (pt) |
| EP (1) | EP3931324A1 (pt) |
| JP (1) | JP2022521502A (pt) |
| KR (1) | KR20210132678A (pt) |
| CN (1) | CN113748207A (pt) |
| AU (1) | AU2020228134A1 (pt) |
| BR (1) | BR112021016907A2 (pt) |
| CA (1) | CA3130854A1 (pt) |
| EA (1) | EA202192330A1 (pt) |
| MX (1) | MX2021010255A (pt) |
| SG (1) | SG11202109112WA (pt) |
| WO (1) | WO2020174023A1 (pt) |
Families Citing this family (1)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2023122800A1 (en) * | 2021-12-23 | 2023-06-29 | University Of Massachusetts | Therapeutic treatment for fragile x-associated disorder |
Family Cites Families (7)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| AU2009315898B2 (en) | 2008-11-13 | 2015-07-23 | Nogra Pharma Limited | Antisense compositions and methods of making and using same |
| EP2411532B1 (en) * | 2009-03-24 | 2017-04-05 | Asuragen, Inc. | Pcr methods for characterizing the 5' untranslated region of the fmr1 and fmr2 genes |
| WO2011056930A2 (en) * | 2009-11-06 | 2011-05-12 | Emory University | Fragile x mental retardation protein (fmrp), compositions, and methods related thereto |
| US20130149297A1 (en) * | 2010-07-02 | 2013-06-13 | Vib Vzw | 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 |
| JP2018528968A (ja) * | 2015-09-17 | 2018-10-04 | ユニバーシティ オブ マサチューセッツ | Ｆｍｒ１発現を調節するための組成物および方法 |
| EP3368089A4 (en) * | 2015-10-26 | 2019-05-29 | Translate Bio Ma, Inc. | NANOPARTICLE FORMULATIONS FOR ADMINISTRATION OF NUCLEIC ACID COMPLEXES |
-
2020
- 2020-02-26 KR KR1020217030136A patent/KR20210132678A/ko unknown
- 2020-02-26 CA CA3130854A patent/CA3130854A1/en active Pending
- 2020-02-26 SG SG11202109112WA patent/SG11202109112WA/en unknown
- 2020-02-26 WO PCT/EP2020/055071 patent/WO2020174023A1/en unknown
- 2020-02-26 AU AU2020228134A patent/AU2020228134A1/en active Pending
- 2020-02-26 EP EP20707610.0A patent/EP3931324A1/en active Pending
- 2020-02-26 BR BR112021016907A patent/BR112021016907A2/pt unknown
- 2020-02-26 US US17/433,528 patent/US20220145303A1/en active Pending
- 2020-02-26 EA EA202192330A patent/EA202192330A1/ru unknown
- 2020-02-26 MX MX2021010255A patent/MX2021010255A/es unknown
- 2020-02-26 JP JP2021547879A patent/JP2022521502A/ja active Pending
- 2020-02-26 CN CN202080031598.4A patent/CN113748207A/zh active Pending
Also Published As
| Publication number | Publication date |
|---|---|
| CN113748207A (zh) | 2021-12-03 |
| CA3130854A1 (en) | 2020-09-03 |
| AU2020228134A1 (en) | 2021-10-14 |
| EP3931324A1 (en) | 2022-01-05 |
| EA202192330A1 (ru) | 2021-12-13 |
| MX2021010255A (es) | 2021-12-15 |
| JP2022521502A (ja) | 2022-04-08 |
| KR20210132678A (ko) | 2021-11-04 |
| SG11202109112WA (en) | 2021-09-29 |
| WO2020174023A1 (en) | 2020-09-03 |
| US20220145303A1 (en) | 2022-05-12 |
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