JP2023529371A - シヌクレイノパチーの処置のための組成物および方法 - Google Patents

シヌクレイノパチーの処置のための組成物および方法 Download PDF

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JP2023529371A
JP2023529371A JP2022574541A JP2022574541A JP2023529371A JP 2023529371 A JP2023529371 A JP 2023529371A JP 2022574541 A JP2022574541 A JP 2022574541A JP 2022574541 A JP2022574541 A JP 2022574541A JP 2023529371 A JP2023529371 A JP 2023529371A
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fusion protein
synuclein
alpha
sequence
seq
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JP2023529371A5 (https=
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彰徳 菱谷
コヤ，ケイゾウ
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ソラ・バイオサイエンシズ・エルエルシー
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    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • 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
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/35—Fusion polypeptide containing a fusion for enhanced stability/folding during expression, e.g. fusions with chaperones or thioredoxin
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/70—Fusion polypeptide containing domain for protein-protein interaction
    • C07K2319/74—Fusion polypeptide containing domain for protein-protein interaction containing a fusion for binding to a cell surface receptor
    • 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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  • Health & Medical Sciences (AREA)
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  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
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  • Neurosurgery (AREA)
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  • Chemical Kinetics & Catalysis (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Wood Science & Technology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Plant Pathology (AREA)
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  • Physics & Mathematics (AREA)
  • Obesity (AREA)
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  • Virology (AREA)
  • Microbiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Peptides Or Proteins (AREA)
JP2022574541A 2020-06-05 2021-06-04 シヌクレイノパチーの処置のための組成物および方法 Pending JP2023529371A (ja)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US202063035297P 2020-06-05 2020-06-05
US63/035,297 2020-06-05
PCT/US2021/035958 WO2021248038A1 (en) 2020-06-05 2021-06-04 Compositions and methods for the treatment of synucleinopathies

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JP2023529371A true JP2023529371A (ja) 2023-07-10
JP2023529371A5 JP2023529371A5 (https=) 2024-06-10

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US (1) US20230234997A1 (https=)
EP (1) EP4161953A1 (https=)
JP (1) JP2023529371A (https=)
CN (1) CN116096737A (https=)
CA (1) CA3183251A1 (https=)
WO (1) WO2021248038A1 (https=)

Families Citing this family (3)

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Publication number Priority date Publication date Assignee Title
WO2024229777A1 (zh) * 2023-05-11 2024-11-14 深圳先进技术研究院 一种aav载体及其应用
CN116590342B (zh) * 2023-05-11 2026-04-03 普递瑞(上海)医药有限公司 一种aav载体及其应用
CN117402251B (zh) * 2023-12-15 2024-02-23 中国医学科学院基础医学研究所 一种抗小g蛋白rbj的抗体及其应用

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JP2004502781A (ja) * 2000-07-07 2004-01-29 パナシア　ファーマシューティカルズ　インコーポレーテッド 神経組織の損傷を予防するため、およびα−シヌクレイン疾患を処置するための方法
JP2016500371A (ja) * 2012-12-05 2016-01-12 ストラテジア・セラピューティクス・インコーポレイテッド タンパク質発現増強ポリペプチド
WO2019161386A1 (en) * 2018-02-19 2019-08-22 New York University Alpha-synuclein single domain antibodies

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US5804413A (en) 1992-07-31 1998-09-08 University Of Pittsburgh Of The Commonwealth System Of Higher Education Herpes simplex virus strains for gene transfer
GB9415319D0 (en) 1994-07-29 1994-09-21 Medical Res Council HSV viral vector
US5846782A (en) 1995-11-28 1998-12-08 Genvec, Inc. Targeting adenovirus with use of constrained peptide motifs
US6093570A (en) 1995-06-07 2000-07-25 The University Of North Carolina At Chapel Hill Helper virus-free AAV production
US6013516A (en) 1995-10-06 2000-01-11 The Salk Institute For Biological Studies Vector and method of use for nucleic acid delivery to non-dividing cells
AU8605598A (en) 1997-07-31 1999-02-22 University Of Pittsburgh Targeted hsv vectors
US5994136A (en) 1997-12-12 1999-11-30 Cell Genesys, Inc. Method and means for producing high titer, safe, recombinant lentivirus vectors
WO1999061601A2 (en) 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
CN1246532A (zh) * 1998-08-31 2000-03-08 复旦大学 人热休克关联蛋白编码序列、其编码的多肽及制备方法
ATE318923T1 (de) 2000-06-01 2006-03-15 Univ North Carolina Doppelsträngige parvovirus-vektoren
WO2002053703A2 (en) 2001-01-05 2002-07-11 Children's Hospital, Inc. Aav2 vectors and methods
MX359371B (es) 2001-11-13 2018-09-25 Univ Pennsylvania Un metodo para detectar y/o identificar secuencias del virus adeno-asociado y aislamiento de secuencias novedosas identificadas de ese modo.
PT1453547T (pt) 2001-12-17 2016-12-28 Univ Pennsylvania Sequências do vírus adeno-associado (aav) do serotipo 8, vetores contendo as mesmas, e utilizações destas
EP2345731B1 (en) 2003-09-30 2015-10-21 The Trustees of the University of Pennsylvania Adeno-associated virus (AAV) clades, sequences, vectors containing same, and uses thereof
JP5136766B2 (ja) 2004-12-15 2013-02-06 ユニバーシティ オブ ノース カロライナ アット チャペル ヒル キメラベクター
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JP2004502781A (ja) * 2000-07-07 2004-01-29 パナシア　ファーマシューティカルズ　インコーポレーテッド 神経組織の損傷を予防するため、およびα−シヌクレイン疾患を処置するための方法
JP2016500371A (ja) * 2012-12-05 2016-01-12 ストラテジア・セラピューティクス・インコーポレイテッド タンパク質発現増強ポリペプチド
WO2019161386A1 (en) * 2018-02-19 2019-08-22 New York University Alpha-synuclein single domain antibodies

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THE JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 290, no. 12, JPN6025020217, 2015, pages 7426 - 7435, ISSN: 0005753609 *

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CN116096737A (zh) 2023-05-09
EP4161953A1 (en) 2023-04-12
US20230234997A1 (en) 2023-07-27
WO2021248038A1 (en) 2021-12-09
CA3183251A1 (en) 2021-12-09

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