RU2020121752A - Средство для лечения синдрома ангельмана на основе антисмысловой нуклеиновой кислоты - Google Patents
Средство для лечения синдрома ангельмана на основе антисмысловой нуклеиновой кислоты Download PDFInfo
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- RU2020121752A RU2020121752A RU2020121752A RU2020121752A RU2020121752A RU 2020121752 A RU2020121752 A RU 2020121752A RU 2020121752 A RU2020121752 A RU 2020121752A RU 2020121752 A RU2020121752 A RU 2020121752A RU 2020121752 A RU2020121752 A RU 2020121752A
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- oligonucleotide
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- oligonucleotide according
- rna
- nucleic acid
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- 208000009575 Angelman syndrome Diseases 0.000 title claims 3
- 150000007523 nucleic acids Chemical class 0.000 title claims 3
- 230000000692 anti-sense effect Effects 0.000 title 1
- 102000039446 nucleic acids Human genes 0.000 title 1
- 108020004707 nucleic acids Proteins 0.000 title 1
- 108091034117 Oligonucleotide Proteins 0.000 claims 17
- 125000003835 nucleoside group Chemical group 0.000 claims 4
- 239000002777 nucleoside Substances 0.000 claims 3
- 239000002773 nucleotide Substances 0.000 claims 3
- 125000003729 nucleotide group Chemical group 0.000 claims 3
- 108091028043 Nucleic acid sequence Proteins 0.000 claims 2
- FWMNVWWHGCHHJJ-SKKKGAJSSA-N 4-amino-1-[(2r)-6-amino-2-[[(2r)-2-[[(2r)-2-[[(2r)-2-amino-3-phenylpropanoyl]amino]-3-phenylpropanoyl]amino]-4-methylpentanoyl]amino]hexanoyl]piperidine-4-carboxylic acid Chemical compound C([C@H](C(=O)N[C@H](CC(C)C)C(=O)N[C@H](CCCCN)C(=O)N1CCC(N)(CC1)C(O)=O)NC(=O)[C@H](N)CC=1C=CC=CC=1)C1=CC=CC=C1 FWMNVWWHGCHHJJ-SKKKGAJSSA-N 0.000 claims 1
- 102100034343 Integrase Human genes 0.000 claims 1
- 101710203526 Integrase Proteins 0.000 claims 1
- -1 LNA nucleoside Chemical class 0.000 claims 1
- 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 claims 1
- 239000002253 acid Substances 0.000 claims 1
- 239000002671 adjuvant Substances 0.000 claims 1
- 239000000074 antisense oligonucleotide Substances 0.000 claims 1
- 238000012230 antisense oligonucleotides Methods 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 238000000034 method Methods 0.000 claims 1
- 239000000203 mixture Substances 0.000 claims 1
- 150000003833 nucleoside derivatives Chemical class 0.000 claims 1
- 239000008194 pharmaceutical composition Substances 0.000 claims 1
- 150000003839 salts Chemical class 0.000 claims 1
- 239000002904 solvent Substances 0.000 claims 1
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical compound [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 1
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- 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
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- 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
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- 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
- A61K48/005—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'active' part of the composition delivered, i.e. the nucleic acid delivered
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- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
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- C07H21/04—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with deoxyribosyl as saccharide radical
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- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C12N2310/35—Nature of the modification
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- C12N2310/3521—Methyl
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- C12N2310/35—Nature of the modification
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- C12N2310/3525—MOE, methoxyethoxy
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
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- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
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- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
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- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
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- Neurosurgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762593431P | 2017-12-01 | 2017-12-01 | |
| US62/593,431 | 2017-12-01 | ||
| US201862676034P | 2018-05-24 | 2018-05-24 | |
| US62/676,034 | 2018-05-24 | ||
| PCT/US2018/063416 WO2019109001A1 (en) | 2017-12-01 | 2018-11-30 | Angelman syndrome antisense treatment |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| RU2020121752A true RU2020121752A (ru) | 2021-12-30 |
Family
ID=66664231
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| RU2020121752A RU2020121752A (ru) | 2017-12-01 | 2018-11-30 | Средство для лечения синдрома ангельмана на основе антисмысловой нуклеиновой кислоты |
Country Status (26)
| Country | Link |
|---|---|
| US (5) | US11198869B2 (enExample) |
| EP (2) | EP3717646B1 (enExample) |
| JP (3) | JP7297320B2 (enExample) |
| KR (2) | KR102723989B1 (enExample) |
| CN (2) | CN111433361B (enExample) |
| AU (1) | AU2018375807B2 (enExample) |
| BR (1) | BR112020009431A2 (enExample) |
| CA (1) | CA3079755A1 (enExample) |
| CL (1) | CL2020001344A1 (enExample) |
| CO (1) | CO2020006361A2 (enExample) |
| DK (1) | DK3717646T3 (enExample) |
| ES (1) | ES3032315T3 (enExample) |
| FI (1) | FI3717646T3 (enExample) |
| HR (1) | HRP20250804T1 (enExample) |
| IL (1) | IL274146B2 (enExample) |
| LT (1) | LT3717646T (enExample) |
| MX (2) | MX2020005680A (enExample) |
| PE (1) | PE20211238A1 (enExample) |
| PL (1) | PL3717646T3 (enExample) |
| PT (1) | PT3717646T (enExample) |
| RS (1) | RS66986B1 (enExample) |
| RU (1) | RU2020121752A (enExample) |
| SG (1) | SG11202003596WA (enExample) |
| SI (1) | SI3717646T1 (enExample) |
| SM (1) | SMT202500334T1 (enExample) |
| WO (1) | WO2019109001A1 (enExample) |
Families Citing this family (9)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| PL3374509T3 (pl) | 2015-11-12 | 2021-05-04 | F. Hoffmann-La Roche Ag | Oligonukleotydy do indukowania ojcowskiej ekspresji UBE3A |
| WO2019168950A1 (en) * | 2018-02-27 | 2019-09-06 | The University Of North Carolina At Chapel Hill | Methods and compositions for treating angelman syndrome |
| TWI874376B (zh) | 2019-03-29 | 2025-03-01 | 美商Ionis製藥公司 | 用於調節ube3a-ats 之化合物及方法 |
| TW202216996A (zh) * | 2020-06-29 | 2022-05-01 | 美商Ionis製藥公司 | 調節ｐｌｐ１之化合物及方法 |
| CA3197664A1 (en) * | 2020-10-06 | 2022-04-14 | University Of Maryland, Baltimore | Rapid diagnostic electrochemical biosensing targeted with antisense oligonucleotides |
| EP4473116A2 (en) * | 2022-03-07 | 2024-12-11 | University of Connecticut | Shrna targeting snord115 locations to restore paternal ube3a gene expression in angelman syndrome |
| CN120476156A (zh) | 2023-01-31 | 2025-08-12 | 富士胶片株式会社 | 固化性组合物、固化物、光学材料、衍射光学元件及化合物 |
| WO2025100980A1 (ko) * | 2023-11-08 | 2025-05-15 | 한국과학기술원 | Ube3a를 표적으로 하여 발현증강을 유도하는 안티센스 올리고뉴클레오티드 및 이의 용도 |
| WO2025217466A1 (en) * | 2024-04-11 | 2025-10-16 | Ultragenyx Pharmaceutical Inc. | Dosing regimens for antisense treatment of angelman syndrome |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CA2122030C (en) | 1991-10-24 | 1997-03-04 | Muthiah Manoharan | Derivatized oligonucleotides having improved uptake and other properties |
| CZ302719B6 (cs) * | 2000-12-01 | 2011-09-21 | MAX-PLANCK-Gesellschaft zur Förderung der Wissenschaften e.V. | Izolovaná molekula dvouretezcové RNA, zpusob její výroby a její použití |
| EP1799859B1 (en) | 2004-09-17 | 2014-07-02 | Isis Pharmaceuticals, Inc. | Enhanced antisense oligonucleotides |
| EP1676834A1 (en) | 2004-12-30 | 2006-07-05 | Sanofi-Aventis Deutschland GmbH | Fused bicyclic carboxamide derivates for use as CXCR2 inhibitors in the treatment of inflammation |
| WO2007031091A2 (en) | 2005-09-15 | 2007-03-22 | Santaris Pharma A/S | Rna antagonist compounds for the modulation of p21 ras expression |
| US8178503B2 (en) * | 2006-03-03 | 2012-05-15 | International Business Machines Corporation | Ribonucleic acid interference molecules and binding sites derived by analyzing intergenic and intronic regions of genomes |
| US20080299659A1 (en) * | 2007-03-02 | 2008-12-04 | Nastech Pharmaceutical Company Inc. | Nucleic acid compounds for inhibiting apob gene expression and uses thereof |
| DK2149605T3 (da) | 2007-03-22 | 2013-09-30 | Santaris Pharma As | Korte RNA antagonist forbindelser til modulering af det ønskede mRNA |
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