JP2022531177A - 神経変性障害を治療するための方法 - Google Patents
神経変性障害を治療するための方法 Download PDFInfo
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- JP2022531177A JP2022531177A JP2021564246A JP2021564246A JP2022531177A JP 2022531177 A JP2022531177 A JP 2022531177A JP 2021564246 A JP2021564246 A JP 2021564246A JP 2021564246 A JP2021564246 A JP 2021564246A JP 2022531177 A JP2022531177 A JP 2022531177A
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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
- A61K31/7028—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages
- A61K31/7032—Compounds having saccharide radicals attached to non-saccharide compounds by glycosidic linkages attached to a polyol, i.e. compounds having two or more free or esterified hydroxy groups, including the hydroxy group involved in the glycosidic linkage, e.g. monoglucosyldiacylglycerides, lactobionic acid, gangliosides
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/45—Transferases (2)
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- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
- A61K38/00—Medicinal preparations containing peptides
- A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
- A61K38/43—Enzymes; Proenzymes; Derivatives thereof
- A61K38/46—Hydrolases (3)
- A61K38/47—Hydrolases (3) acting on glycosyl compounds (3.2), e.g. cellulases, lactases
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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
- A61P25/14—Drugs for disorders of the nervous system for treating abnormal movements, e.g. chorea, dyskinesia
- A61P25/16—Anti-Parkinson drugs
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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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- 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
- C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
- C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
- C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
- C12N15/86—Viral vectors
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y204/00—Glycosyltransferases (2.4)
- C12Y204/01—Hexosyltransferases (2.4.1)
- C12Y204/01062—Ganglioside galactosyltransferase (2.4.1.62)
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12Y—ENZYMES
- C12Y302/00—Hydrolases acting on glycosyl compounds, i.e. glycosylases (3.2)
- C12Y302/01—Glycosidases, i.e. enzymes hydrolysing O- and S-glycosyl compounds (3.2.1)
- C12Y302/01018—Exo-alpha-sialidase (3.2.1.18), i.e. trans-sialidase
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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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14141—Use of virus, viral particle or viral elements as a vector
- C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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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
- C12N2750/00—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
- C12N2750/00011—Details
- C12N2750/14011—Parvoviridae
- C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
- C12N2750/14171—Demonstrated in vivo effect
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Chemical & Material Sciences (AREA)
- Engineering & Computer Science (AREA)
- Bioinformatics & Cheminformatics (AREA)
- General Health & Medical Sciences (AREA)
- Organic Chemistry (AREA)
- Biomedical Technology (AREA)
- Veterinary Medicine (AREA)
- Medicinal Chemistry (AREA)
- Public Health (AREA)
- Pharmacology & Pharmacy (AREA)
- Animal Behavior & Ethology (AREA)
- Genetics & Genomics (AREA)
- Neurosurgery (AREA)
- Neurology (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Epidemiology (AREA)
- Biochemistry (AREA)
- Molecular Biology (AREA)
- General Chemical & Material Sciences (AREA)
- Chemical Kinetics & Catalysis (AREA)
- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
- Proteomics, Peptides & Aminoacids (AREA)
- Biotechnology (AREA)
- Immunology (AREA)
- Gastroenterology & Hepatology (AREA)
- Hospice & Palliative Care (AREA)
- Psychiatry (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Biophysics (AREA)
- Physics & Mathematics (AREA)
- Virology (AREA)
- Psychology (AREA)
- Medicines Containing Material From Animals Or Micro-Organisms (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
Applications Claiming Priority (3)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201962840235P | 2019-04-29 | 2019-04-29 | |
| US62/840,235 | 2019-04-29 | ||
| PCT/US2020/030409 WO2020223310A2 (en) | 2019-04-29 | 2020-04-29 | Methods for treating neurodegenerative disorders |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2022531177A true JP2022531177A (ja) | 2022-07-06 |
| JP2022531177A5 JP2022531177A5 (https=) | 2023-05-10 |
Family
ID=73029186
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2021564246A Pending JP2022531177A (ja) | 2019-04-29 | 2020-04-29 | 神経変性障害を治療するための方法 |
Country Status (5)
| Country | Link |
|---|---|
| US (1) | US20220226357A1 (https=) |
| EP (1) | EP3962492A4 (https=) |
| JP (1) | JP2022531177A (https=) |
| CN (1) | CN114761021A (https=) |
| WO (1) | WO2020223310A2 (https=) |
Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2007018275A1 (ja) * | 2005-08-11 | 2007-02-15 | National Institute Of Advanced Industrial Science And Technology | ローリングモデルに基づく、送達媒体の設計 |
| WO2009104649A1 (ja) * | 2008-02-22 | 2009-08-27 | 片山化学工業株式会社 | 合成糖脂質含有リポソーム |
| JP2013508366A (ja) * | 2009-10-19 | 2013-03-07 | アミカス セラピューティックス インコーポレイテッド | 中枢神経系の変性障害を予防および/または治療するための新規の組成物 |
| JP2014523881A (ja) * | 2011-06-22 | 2014-09-18 | ザ ジェネラル ホスピタル コーポレイション | プロテイノパチーの処置方法 |
| WO2017009312A1 (en) * | 2015-07-13 | 2017-01-19 | H. Lundbeck A/S | Agents, uses and methods for the treatment of synucleinopathy |
| JP2017514515A (ja) * | 2014-04-08 | 2017-06-08 | プロセナ・バイオサイエンシズ・リミテッド | α−シヌクレインを認識する抗体を含む血液脳関門シャトル |
Family Cites Families (3)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2473174A4 (en) * | 2009-09-01 | 2014-02-26 | Lz Therapeutics Inc | TRANSMUCOSAL GANGLIOSIDE FORMULATIONS |
| US20140187507A1 (en) * | 2012-12-14 | 2014-07-03 | Rutgers, The State University Of New Jersey | Treatment of parkinsons disease with glycolipids |
| WO2017015491A1 (en) * | 2015-07-21 | 2017-01-26 | Thomas Jefferson University | Gene therapies for neurodegenerative disorders targeting ganglioside biosynthetic pathways |
-
2020
- 2020-04-29 JP JP2021564246A patent/JP2022531177A/ja active Pending
- 2020-04-29 CN CN202080044151.0A patent/CN114761021A/zh active Pending
- 2020-04-29 EP EP20798336.2A patent/EP3962492A4/en active Pending
- 2020-04-29 WO PCT/US2020/030409 patent/WO2020223310A2/en not_active Ceased
- 2020-04-29 US US17/607,294 patent/US20220226357A1/en active Pending
Patent Citations (6)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| WO2007018275A1 (ja) * | 2005-08-11 | 2007-02-15 | National Institute Of Advanced Industrial Science And Technology | ローリングモデルに基づく、送達媒体の設計 |
| WO2009104649A1 (ja) * | 2008-02-22 | 2009-08-27 | 片山化学工業株式会社 | 合成糖脂質含有リポソーム |
| JP2013508366A (ja) * | 2009-10-19 | 2013-03-07 | アミカス セラピューティックス インコーポレイテッド | 中枢神経系の変性障害を予防および/または治療するための新規の組成物 |
| JP2014523881A (ja) * | 2011-06-22 | 2014-09-18 | ザ ジェネラル ホスピタル コーポレイション | プロテイノパチーの処置方法 |
| JP2017514515A (ja) * | 2014-04-08 | 2017-06-08 | プロセナ・バイオサイエンシズ・リミテッド | α−シヌクレインを認識する抗体を含む血液脳関門シャトル |
| WO2017009312A1 (en) * | 2015-07-13 | 2017-01-19 | H. Lundbeck A/S | Agents, uses and methods for the treatment of synucleinopathy |
Non-Patent Citations (7)
| Title |
|---|
| GUSHENG WU ET AL.: "Deficiency of Ganglioside GM1 Correlates With Parkinson’s Disease in Mice and Humans", JOURNAL OF NEUROSCIENCE RESEARCH, vol. 90, JPN6024015348, 2012, pages 1997 - 2008, ISSN: 0005473055 * |
| JAY S. SCHNEIDER ET AL.: "GM1 Ganglioside Modifies α-Synuclein Toxicity and is Neuroprotective in a Rat α-Synuclein Model of", SCIENTIFIC REPORTS, vol. 9, JPN7024001614, 2019, pages 1 - 12, ISSN: 0005309628 * |
| JIANSHE WE ET AL.: "Gangliosides protection against lysosomal pathology of synucleinopathies", AUTOPHAGY, vol. 5, JPN7024001613, 2009, pages 860 - 861, ISSN: 0005309629 * |
| JIANSHE WEI ET AL.: "Protective Role of Endogenous Gangliosides for Lysosomal Pathology in a Cellular Model of Synucleino", THE AMERICAN JOURNAL OF PATHOLOGY, vol. 174, JPN7024001612, 2009, pages 1891 - 1909, ISSN: 0005309630 * |
| ZAK MARTINEZ ET AL.: "GM1 Specifically Interacts with R-Synuclein and Inhibits Fibrillation", BIOCHEMISTRY, vol. 46, JPN6024015347, 2007, pages 1868 - 1877, ISSN: 0005473056 * |
| ZAK MARTINEZ ET AL.: "GM1 Specifically Interacts with α-Synuclein and Inhibits Fibrillation", BIOCHEMISTRY, vol. 46, JPN6024048566, 2007, pages 1868 - 1877, ISSN: 0005473057 * |
| 高田 昌彦: "アルファシヌクレイン発現によるパーキンソン病サルモデルの開発", 日本生物学的精神医学会誌, vol. 24, JPN6024048567, 2013, pages 57 - 61, ISSN: 0005473058 * |
Also Published As
| Publication number | Publication date |
|---|---|
| WO2020223310A3 (en) | 2020-12-10 |
| EP3962492A4 (en) | 2023-05-31 |
| WO2020223310A2 (en) | 2020-11-05 |
| EP3962492A2 (en) | 2022-03-09 |
| US20220226357A1 (en) | 2022-07-21 |
| CN114761021A (zh) | 2022-07-15 |
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