MX2023003810A - Composiciones de arn de interferencia (arni) de alfa sinucleina (snca) y metodos de usos de las mismas para tratar o prevenir enfermedades neurodegenerativas asociadas con la snca. - Google Patents
Composiciones de arn de interferencia (arni) de alfa sinucleina (snca) y metodos de usos de las mismas para tratar o prevenir enfermedades neurodegenerativas asociadas con la snca.Info
- Publication number
- MX2023003810A MX2023003810A MX2023003810A MX2023003810A MX2023003810A MX 2023003810 A MX2023003810 A MX 2023003810A MX 2023003810 A MX2023003810 A MX 2023003810A MX 2023003810 A MX2023003810 A MX 2023003810A MX 2023003810 A MX2023003810 A MX 2023003810A
- Authority
- MX
- Mexico
- Prior art keywords
- snca
- methods
- compositions
- sinuclein
- rnai
- Prior art date
Links
- 102100026882 Alpha-synuclein Human genes 0.000 title abstract 3
- 101000834898 Homo sapiens Alpha-synuclein Proteins 0.000 title abstract 3
- 238000000034 method Methods 0.000 title abstract 3
- 239000000203 mixture Substances 0.000 title abstract 3
- 208000015122 neurodegenerative disease Diseases 0.000 title abstract 3
- 238000012228 RNA interference-mediated gene silencing Methods 0.000 title 2
- 230000009368 gene silencing by RNA Effects 0.000 title 2
- 208000009829 Lewy Body Disease Diseases 0.000 abstract 2
- 201000002832 Lewy body dementia Diseases 0.000 abstract 2
- 208000018737 Parkinson disease Diseases 0.000 abstract 2
- 101150110423 SNCA gene Proteins 0.000 abstract 2
- 239000003795 chemical substances by application Substances 0.000 abstract 2
- 208000001089 Multiple system atrophy Diseases 0.000 abstract 1
- 208000032859 Synucleinopathies Diseases 0.000 abstract 1
- 230000002401 inhibitory effect Effects 0.000 abstract 1
- 230000004770 neurodegeneration Effects 0.000 abstract 1
- 229920002477 rna polymer Polymers 0.000 abstract 1
- 230000008685 targeting Effects 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
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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
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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
- 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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- 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
- A61K31/713—Double-stranded nucleic acids or oligonucleotides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K47/00—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
- 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
- A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
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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
- 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]
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/312—Phosphonates
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/31—Chemical structure of the backbone
- C12N2310/315—Phosphorothioates
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
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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
- 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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- 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
- C12N2310/332—Abasic residue
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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
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3515—Lipophilic moiety, e.g. cholesterol
-
- 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
- C12N2310/35—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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- 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
- C12N2310/35—Nature of the modification
- C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
- C12N2310/3533—Halogen
Landscapes
- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Chemical & Material Sciences (AREA)
- Biomedical Technology (AREA)
- Genetics & Genomics (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biotechnology (AREA)
- Public Health (AREA)
- Animal Behavior & Ethology (AREA)
- Veterinary Medicine (AREA)
- Biochemistry (AREA)
- Wood Science & Technology (AREA)
- Pharmacology & Pharmacy (AREA)
- Zoology (AREA)
- Medicinal Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- Epidemiology (AREA)
- Plant Pathology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Microbiology (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Psychiatry (AREA)
- Hospice & Palliative Care (AREA)
- General Chemical & Material Sciences (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
- Micro-Organisms Or Cultivation Processes Thereof (AREA)
- Medicinal Preparation (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (2)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US202063086495P | 2020-10-01 | 2020-10-01 | |
| PCT/US2021/052580 WO2022072447A1 (en) | 2020-10-01 | 2021-09-29 | Snca irna compositions and methods of use thereof for treating or preventing snca-associated neurodegenerative diseases |
Publications (1)
| Publication Number | Publication Date |
|---|---|
| MX2023003810A true MX2023003810A (es) | 2023-06-12 |
Family
ID=80950831
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| MX2023003810A MX2023003810A (es) | 2020-10-01 | 2021-09-29 | Composiciones de arn de interferencia (arni) de alfa sinucleina (snca) y metodos de usos de las mismas para tratar o prevenir enfermedades neurodegenerativas asociadas con la snca. |
Country Status (12)
| Country | Link |
|---|---|
| US (1) | US20240011032A1 (https=) |
| EP (1) | EP4221838A4 (https=) |
| JP (1) | JP2023544385A (https=) |
| KR (1) | KR20230079405A (https=) |
| CN (1) | CN116710107A (https=) |
| AR (1) | AR123679A1 (https=) |
| AU (1) | AU2021353935A1 (https=) |
| CA (1) | CA3193830A1 (https=) |
| IL (1) | IL301712A (https=) |
| MX (1) | MX2023003810A (https=) |
| TW (1) | TW202229551A (https=) |
| WO (1) | WO2022072447A1 (https=) |
Families Citing this family (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| CN119630791A (zh) * | 2022-05-13 | 2025-03-14 | 迪克纳制药公司 | 用于抑制snca表达的组合物和方法 |
| US20260103703A1 (en) * | 2022-09-29 | 2026-04-16 | 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 |
| AU2024335806A1 (en) * | 2023-09-07 | 2026-04-23 | Janssen Pharmaceutica Nv | SNCA siRNA AND USES THEREOF |
| WO2025162214A1 (en) * | 2024-01-30 | 2025-08-07 | Shanghai Argo Biopharmaceutical Co., Ltd. | Compositions and methods for inhibiting expression of synuclein alpha (snca) gene |
| TW202547527A (zh) * | 2024-02-08 | 2025-12-16 | 日商泰爾茂股份有限公司 | 寡核苷酸 |
Family Cites Families (12)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2006006948A2 (en) * | 2002-11-14 | 2006-01-19 | Dharmacon, Inc. | METHODS AND COMPOSITIONS FOR SELECTING siRNA OF IMPROVED FUNCTIONALITY |
| EP2508608A1 (en) * | 2003-06-09 | 2012-10-10 | Alnylam Pharmaceuticals Inc. | Method of treating neurodegenerative disease |
| WO2005045034A2 (en) * | 2003-10-23 | 2005-05-19 | Sirna Therapeutics, Inc. | RNA INTERFERENCE MEDIATED TREATMENT OF PARKINSON DISEASE USING SHORT INTERERING NUCLEIC ACID (siNA) |
| US20080003570A1 (en) * | 2004-12-22 | 2008-01-03 | The General Hospital Corporation | Translation enhancer elements of genes encoding human Tau protein and human alpha-synuclein protein |
| US20100105134A1 (en) * | 2007-03-02 | 2010-04-29 | Mdrna, Inc. | Nucleic acid compounds for inhibiting gene expression and uses thereof |
| WO2009079399A2 (en) * | 2007-12-14 | 2009-06-25 | Alnylam Pharmaceuticals, Inc. | Method of treating neurodegenerative disease |
| 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 |
| CN108728533B (zh) * | 2017-04-20 | 2022-06-14 | 常青 | 用于髓母细胞瘤分子分型的基因群以及snca基因作为4型髓母细胞瘤的生物标志物的用途 |
| AU2019207859A1 (en) * | 2018-01-12 | 2020-07-02 | Roche Innovation Center Copenhagen A/S | Alpha-synuclein antisense oligonucleotides and uses thereof |
| WO2019217459A1 (en) * | 2018-05-07 | 2019-11-14 | Alnylam Pharmaceuticals, Inc. | Extrahepatic delivery |
| EP3830265A1 (en) * | 2018-08-03 | 2021-06-09 | Genzyme Corporation | Variant rnai against alpha-synuclein |
-
2021
- 2021-09-29 AU AU2021353935A patent/AU2021353935A1/en active Pending
- 2021-09-29 EP EP21876366.2A patent/EP4221838A4/en active Pending
- 2021-09-29 JP JP2023520289A patent/JP2023544385A/ja active Pending
- 2021-09-29 US US18/247,314 patent/US20240011032A1/en active Pending
- 2021-09-29 CN CN202180081220.XA patent/CN116710107A/zh active Pending
- 2021-09-29 KR KR1020237014415A patent/KR20230079405A/ko active Pending
- 2021-09-29 WO PCT/US2021/052580 patent/WO2022072447A1/en not_active Ceased
- 2021-09-29 IL IL301712A patent/IL301712A/en unknown
- 2021-09-29 CA CA3193830A patent/CA3193830A1/en active Pending
- 2021-09-29 MX MX2023003810A patent/MX2023003810A/es unknown
- 2021-09-30 TW TW110136531A patent/TW202229551A/zh unknown
- 2021-10-01 AR ARP210102736A patent/AR123679A1/es unknown
Also Published As
| Publication number | Publication date |
|---|---|
| JP2023544385A (ja) | 2023-10-23 |
| TW202229551A (zh) | 2022-08-01 |
| CA3193830A1 (en) | 2022-04-07 |
| CN116710107A (zh) | 2023-09-05 |
| KR20230079405A (ko) | 2023-06-07 |
| AU2021353935A1 (en) | 2023-05-25 |
| WO2022072447A1 (en) | 2022-04-07 |
| AR123679A1 (es) | 2023-01-04 |
| EP4221838A1 (en) | 2023-08-09 |
| US20240011032A1 (en) | 2024-01-11 |
| EP4221838A4 (en) | 2025-04-16 |
| IL301712A (en) | 2023-05-01 |
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