CL2020001344A1 - Tratamiento antisentido del síndrome de angelman. - Google Patents
Tratamiento antisentido del síndrome de angelman.Info
- Publication number
- CL2020001344A1 CL2020001344A1 CL2020001344A CL2020001344A CL2020001344A1 CL 2020001344 A1 CL2020001344 A1 CL 2020001344A1 CL 2020001344 A CL2020001344 A CL 2020001344A CL 2020001344 A CL2020001344 A CL 2020001344A CL 2020001344 A1 CL2020001344 A1 CL 2020001344A1
- Authority
- CL
- Chile
- Prior art keywords
- ube3a
- angelman syndrome
- antisense treatment
- treatment
- oligonucleotides
- Prior art date
Links
- 208000009575 Angelman syndrome Diseases 0.000 title abstract 2
- 230000000692 anti-sense effect Effects 0.000 title 1
- 108700028369 Alleles Proteins 0.000 abstract 2
- 108020000948 Antisense Oligonucleotides Proteins 0.000 abstract 1
- 108091034117 Oligonucleotide Proteins 0.000 abstract 1
- 108091046869 Telomeric non-coding RNA Proteins 0.000 abstract 1
- 102000006275 Ubiquitin-Protein Ligases Human genes 0.000 abstract 1
- 108010083111 Ubiquitin-Protein Ligases Proteins 0.000 abstract 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 abstract 1
- 239000000074 antisense oligonucleotide Substances 0.000 abstract 1
- 238000012230 antisense oligonucleotides Methods 0.000 abstract 1
- 238000009396 hybridization Methods 0.000 abstract 1
- 230000001939 inductive effect Effects 0.000 abstract 1
- 238000000034 method Methods 0.000 abstract 1
- 210000002569 neuron Anatomy 0.000 abstract 1
- 230000008775 paternal effect Effects 0.000 abstract 1
- 239000008194 pharmaceutical composition Substances 0.000 abstract 1
Classifications
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- C—CHEMISTRY; METALLURGY
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- 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
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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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- A—HUMAN NECESSITIES
- 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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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- C07H21/02—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 ribosyl as saccharide radical
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- C07—ORGANIC CHEMISTRY
- C07H—SUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
- C07H21/00—Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
- 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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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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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
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
- C12N2310/113—Antisense targeting other non-coding nucleic acids, e.g. antagomirs
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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- C12N2310/00—Structure or type of the nucleic acid
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- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
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- 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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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/341—Gapmers, i.e. of the type ===---===
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/34—Spatial arrangement of the modifications
- C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3521—Methyl
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
- C12N2310/3525—MOE, methoxyethoxy
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- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
Landscapes
- Health & Medical Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Molecular Biology (AREA)
- Organic Chemistry (AREA)
- Biochemistry (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biomedical Technology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Zoology (AREA)
- Wood Science & Technology (AREA)
- General Engineering & Computer Science (AREA)
- Plant Pathology (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Veterinary Medicine (AREA)
- Animal Behavior & Ethology (AREA)
- Epidemiology (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Neurology (AREA)
- Neurosurgery (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
- Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
- Measuring Or Testing Involving Enzymes Or Micro-Organisms (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201762593431P | 2017-12-01 | 2017-12-01 | |
| US201862676034P | 2018-05-24 | 2018-05-24 |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| CL2020001344A1 true CL2020001344A1 (es) | 2020-09-21 |
Family
ID=66664231
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| CL2020001344A CL2020001344A1 (es) | 2017-12-01 | 2020-05-20 | Tratamiento antisentido del síndrome de angelman. |
Country Status (27)
| Country | Link |
|---|---|
| US (5) | US11198869B2 (enExample) |
| EP (2) | EP3717646B1 (enExample) |
| JP (4) | 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) |
| HU (1) | HUE072645T2 (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 (10)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| HK1258434A1 (zh) | 2015-11-12 | 2019-11-08 | F. Hoffmann-La Roche Ag | 来自灵长类物种的标准化神经元细胞测定试验 |
| EP3758714A4 (en) * | 2018-02-27 | 2021-12-01 | The University of North Carolina at Chapel Hill | METHODS AND COMPOSITIONS FOR TREATMENT OF ANGELMAN'S SYNDROME |
| TW202536179A (zh) | 2019-03-29 | 2025-09-16 | 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 |
| CN119384504A (zh) * | 2022-03-07 | 2025-01-28 | 康涅狄格大学 | 靶向SNORD115位置以恢复快乐木偶综合征中父系UBE3A基因表达的shRNA |
| KR20250107262A (ko) | 2023-01-31 | 2025-07-11 | 후지필름 가부시키가이샤 | 경화성 조성물, 경화물, 광학 재료, 회절 광학 소자 및 화합물 |
| 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 |
| CN121992041A (zh) * | 2024-11-06 | 2026-05-08 | 中国科学院脑科学与智能技术卓越创新中心 | 靶向ube3a等位基因建立天使综合征动物模型的方法和应用 |
Family Cites Families (29)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| JP2823959B2 (ja) | 1991-10-24 | 1998-11-11 | アイシス・ファーマシューティカルス・インコーポレーテッド | 改良された取り込みおよびその他の性質を持つ誘導化オリゴヌクレオチド |
| CN100523215C (zh) * | 2000-12-01 | 2009-08-05 | 马普科技促进协会 | 介导rna干涉的小rna分子 |
| 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 |
| US8580756B2 (en) | 2007-03-22 | 2013-11-12 | Santaris Pharma A/S | Short oligomer antagonist compounds for the modulation of target mRNA |
| US9290534B2 (en) | 2008-04-04 | 2016-03-22 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds having at least one neutrally linked terminal bicyclic nucleoside |
| BRPI0923225A2 (pt) | 2008-12-02 | 2016-10-04 | Chiralgen Ltd | metodo para sintese de acidos nucleicos modificados no atomo de fosforo |
| EP2462153B1 (en) | 2009-08-06 | 2015-07-29 | Isis Pharmaceuticals, Inc. | Bicyclic cyclohexose nucleic acid analogs |
| RU2013150331A (ru) | 2011-04-20 | 2015-05-27 | Рош Гликарт Аг | СПОСОБ И УСТРОЙСТВА ДЛЯ рН-ЗАВИСИМОГО ПРОХОЖДЕНИЯ ГЕМАТОЭНЦЕФАЛИЧЕСКОГО БАРЬЕРА |
| US10023861B2 (en) | 2011-08-29 | 2018-07-17 | Ionis Pharmaceuticals, Inc. | Oligomer-conjugate complexes and their use |
| EP2850092B1 (en) | 2012-04-09 | 2017-03-01 | Ionis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
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