MX2022011536A - Oligonucleotidos para la modulacion de snca. - Google Patents
Oligonucleotidos para la modulacion de snca.Info
- Publication number
- MX2022011536A MX2022011536A MX2022011536A MX2022011536A MX2022011536A MX 2022011536 A MX2022011536 A MX 2022011536A MX 2022011536 A MX2022011536 A MX 2022011536A MX 2022011536 A MX2022011536 A MX 2022011536A MX 2022011536 A MX2022011536 A MX 2022011536A
- Authority
- MX
- Mexico
- Prior art keywords
- snca
- oligonucleotides
- modulation
- novel
- targeting
- Prior art date
Links
- 108091034117 Oligonucleotide 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 abstract 2
- 101150110423 SNCA gene Proteins 0.000 title 1
- 102100026882 Alpha-synuclein Human genes 0.000 abstract 2
- 101000834898 Homo sapiens Alpha-synuclein Proteins 0.000 abstract 2
- 230000008685 targeting Effects 0.000 abstract 2
- 230000004770 neurodegeneration Effects 0.000 abstract 1
- 208000015122 neurodegenerative disease Diseases 0.000 abstract 1
Classifications
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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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- A61K31/70—Carbohydrates; Sugars; Derivatives thereof
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- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
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- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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- A61K47/55—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds
- A61K47/551—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being also a pharmacologically or therapeutically active agent, i.e. the entire conjugate being a codrug, i.e. a dimer, oligomer or polymer of pharmacologically or therapeutically active compounds one of the codrug's components being a vitamin, e.g. niacinamide, vitamin B3, cobalamin, vitamin B12, folate, vitamin A or retinoic acid
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- A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
- A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
- A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
- A61K47/554—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound the modifying agent being a steroid plant sterol, glycyrrhetic acid, enoxolone or bile acid
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- 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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- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P25/00—Drugs for disorders of the nervous system
- A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
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Abstract
Esta descripción se refiere a secuencias dirigidas a SNCA nuevas. También se proporcionan oligonucleótidos dirigidos a SNCA nuevos para el tratamiento de enfermedades neurodegenerativas.
Applications Claiming Priority (3)
Application Number | Priority Date | Filing Date | Title |
---|---|---|---|
US202062991406P | 2020-03-18 | 2020-03-18 | |
US202063071115P | 2020-08-27 | 2020-08-27 | |
PCT/US2021/022748 WO2021188661A1 (en) | 2020-03-18 | 2021-03-17 | Oligonucleotides for snca modulation |
Publications (1)
Publication Number | Publication Date |
---|---|
MX2022011536A true MX2022011536A (es) | 2023-01-04 |
Family
ID=77772170
Family Applications (1)
Application Number | Title | Priority Date | Filing Date |
---|---|---|---|
MX2022011536A MX2022011536A (es) | 2020-03-18 | 2021-03-17 | Oligonucleotidos para la modulacion de snca. |
Country Status (10)
Country | Link |
---|---|
US (1) | US20210363524A1 (es) |
EP (1) | EP4121538A4 (es) |
JP (1) | JP2023518268A (es) |
KR (1) | KR20230002327A (es) |
CN (1) | CN115777021A (es) |
AU (1) | AU2021238326A1 (es) |
BR (1) | BR112022018475A2 (es) |
CA (1) | CA3171757A1 (es) |
MX (1) | MX2022011536A (es) |
WO (1) | WO2021188661A1 (es) |
Families Citing this family (4)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
US20230416735A1 (en) * | 2022-04-14 | 2023-12-28 | University Of Massachusetts | Oligonucleotides for atn1 modulation |
WO2023215390A2 (en) * | 2022-05-04 | 2023-11-09 | University Of Massachusetts | Oligonucleotides for pms1 modulation |
WO2024073609A2 (en) * | 2022-09-29 | 2024-04-04 | Atalanta Therapeutics, Inc. | Sirna compositions and methods targeting alpha-synuclein nucleic acids |
WO2024166019A1 (en) * | 2023-02-08 | 2024-08-15 | Biorchestra Co., Ltd. | Sirna targeting alpha-synuclein and uses thereof |
Family Cites Families (7)
Publication number | Priority date | Publication date | Assignee | Title |
---|---|---|---|---|
EP1675948A2 (en) * | 2003-10-23 | 2006-07-05 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF PARKINSON DISEASE USING SHORT INTERERING NUCLEIC ACID (siNA) |
WO2009079399A2 (en) * | 2007-12-14 | 2009-06-25 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
JP5875976B2 (ja) * | 2009-06-01 | 2016-03-02 | ヘイロー−バイオ アールエヌエーアイ セラピューティクス, インコーポレイテッド | 多価rna干渉のためのポリヌクレオチド、組成物およびそれらの使用方法 |
WO2012027713A2 (en) * | 2010-08-26 | 2012-03-01 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibition of snca |
CA3077910A1 (en) * | 2010-11-17 | 2012-05-24 | Ionis Pharmaceuticals, Inc. | Modulation of alpha synuclein expression |
EP3408391A4 (en) * | 2016-01-31 | 2019-08-28 | University of Massachusetts | BRANCHED OLIGONUCLEOTIDES |
MX2021001395A (es) * | 2018-08-03 | 2021-08-11 | Genzyme Corp | Arni variante contra alfa-sinucleína. |
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2021
- 2021-03-17 CN CN202180035697.4A patent/CN115777021A/zh active Pending
- 2021-03-17 MX MX2022011536A patent/MX2022011536A/es unknown
- 2021-03-17 EP EP21771103.5A patent/EP4121538A4/en active Pending
- 2021-03-17 WO PCT/US2021/022748 patent/WO2021188661A1/en unknown
- 2021-03-17 JP JP2022556250A patent/JP2023518268A/ja active Pending
- 2021-03-17 BR BR112022018475A patent/BR112022018475A2/pt unknown
- 2021-03-17 US US17/204,483 patent/US20210363524A1/en active Pending
- 2021-03-17 CA CA3171757A patent/CA3171757A1/en active Pending
- 2021-03-17 KR KR1020227032663A patent/KR20230002327A/ko unknown
- 2021-03-17 AU AU2021238326A patent/AU2021238326A1/en active Pending
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AU2021238326A1 (en) | 2022-10-13 |
CA3171757A1 (en) | 2021-09-23 |
US20210363524A1 (en) | 2021-11-25 |
EP4121538A4 (en) | 2024-07-31 |
CN115777021A (zh) | 2023-03-10 |
KR20230002327A (ko) | 2023-01-05 |
WO2021188661A1 (en) | 2021-09-23 |
BR112022018475A2 (pt) | 2022-11-29 |
JP2023518268A (ja) | 2023-04-28 |
EP4121538A1 (en) | 2023-01-25 |
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