JP7758671B2 - パーキンソン病を治療するための方法 - Google Patents

パーキンソン病を治療するための方法

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Publication number
JP7758671B2
JP7758671B2 JP2022535694A JP2022535694A JP7758671B2 JP 7758671 B2 JP7758671 B2 JP 7758671B2 JP 2022535694 A JP2022535694 A JP 2022535694A JP 2022535694 A JP2022535694 A JP 2022535694A JP 7758671 B2 JP7758671 B2 JP 7758671B2
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Prior art keywords
gad
vector
nucleic acid
subject
pharmaceutical composition
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JP2022535694A
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English (en)
Japanese (ja)
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JP2023506007A5 (enExample
JP2023506007A (ja
Inventor
フォーブズ アレクサンドリア
デュリング マシュー
Original Assignee
メイラグティーエックス ユーケー アイアイ リミティド
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Publication of JP2023506007A publication Critical patent/JP2023506007A/ja
Publication of JP2023506007A5 publication Critical patent/JP2023506007A5/ja
Priority to JP2025171111A priority Critical patent/JP2025188191A/ja
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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)
    • A61K38/48—Hydrolases (3) acting on peptide bonds (3.4)
    • A61K38/4813—Exopeptidases (3.4.11. to 3.4.19)
    • 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
    • A61K38/51—Lyases (4)
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A61K48/0083—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 administration regime
    • 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
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y401/00—Carbon-carbon lyases (4.1)
    • C12Y401/01—Carboxy-lyases (4.1.1)
    • C12Y401/01015—Glutamate decarboxylase (4.1.1.15)
    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Medicinal Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Epidemiology (AREA)
  • Genetics & Genomics (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Organic Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Psychology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Acyclic And Carbocyclic Compounds In Medicinal Compositions (AREA)
  • Medicines Containing Plant Substances (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
JP2022535694A 2019-12-12 2020-12-14 パーキンソン病を治療するための方法 Active JP7758671B2 (ja)

Priority Applications (1)

Application Number Priority Date Filing Date Title
JP2025171111A JP2025188191A (ja) 2019-12-12 2025-10-09 パーキンソン病を治療するための方法

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962947418P 2019-12-12 2019-12-12
US62/947,418 2019-12-12
PCT/US2020/064928 WO2021119615A1 (en) 2019-12-12 2020-12-14 Methods for treating parkinson's disease

Related Child Applications (1)

Application Number Title Priority Date Filing Date
JP2025171111A Division JP2025188191A (ja) 2019-12-12 2025-10-09 パーキンソン病を治療するための方法

Publications (3)

Publication Number Publication Date
JP2023506007A JP2023506007A (ja) 2023-02-14
JP2023506007A5 JP2023506007A5 (enExample) 2023-11-30
JP7758671B2 true JP7758671B2 (ja) 2025-10-22

Family

ID=76330812

Family Applications (2)

Application Number Title Priority Date Filing Date
JP2022535694A Active JP7758671B2 (ja) 2019-12-12 2020-12-14 パーキンソン病を治療するための方法
JP2025171111A Pending JP2025188191A (ja) 2019-12-12 2025-10-09 パーキンソン病を治療するための方法

Family Applications After (1)

Application Number Title Priority Date Filing Date
JP2025171111A Pending JP2025188191A (ja) 2019-12-12 2025-10-09 パーキンソン病を治療するための方法

Country Status (10)

Country Link
US (1) US20230050187A1 (enExample)
EP (1) EP4072568A4 (enExample)
JP (2) JP7758671B2 (enExample)
KR (1) KR20220114028A (enExample)
AU (1) AU2020403266A1 (enExample)
BR (1) BR112022011584A2 (enExample)
CA (1) CA3161322A1 (enExample)
IL (1) IL293776A (enExample)
MX (1) MX2022007193A (enExample)
WO (1) WO2021119615A1 (enExample)

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018232055A1 (en) 2017-06-15 2018-12-20 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2409674C (en) * 2000-05-23 2010-05-11 Neurologix, Inc. Glutamic acid decarboxylase (gad) based delivery systems
US7695959B2 (en) * 2000-05-23 2010-04-13 Neurologix, Inc. Glutamic acid decarboxylase (GAD) based delivery systems
US20030087264A1 (en) * 2001-05-22 2003-05-08 Kaplitt Michael G. Transcriptional regulation of target genes
CA2610164A1 (en) * 2005-05-31 2006-12-07 Neurologix, Inc. Novel glutamic acid decarboxylase (gad) chimera and methods of use

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018232055A1 (en) 2017-06-15 2018-12-20 Voyager Therapeutics, Inc. Aadc polynucleotides for the treatment of parkinson's disease

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
The Lancet，2007年，Vol.369, No.9579，p.2097-2105

Also Published As

Publication number Publication date
EP4072568A1 (en) 2022-10-19
CA3161322A1 (en) 2021-06-17
IL293776A (en) 2022-08-01
AU2020403266A1 (en) 2022-08-04
KR20220114028A (ko) 2022-08-17
BR112022011584A2 (pt) 2022-12-13
JP2025188191A (ja) 2025-12-25
JP2023506007A (ja) 2023-02-14
MX2022007193A (es) 2022-09-19
WO2021119615A1 (en) 2021-06-17
EP4072568A4 (en) 2024-02-07
US20230050187A1 (en) 2023-02-16

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