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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JPWO2021248038A5 (https=
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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  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
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  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
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  • Molecular Biology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Biotechnology (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Toxicology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Hematology (AREA)
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  • Obesity (AREA)
  • Virology (AREA)
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  • Psychiatry (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (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

Publications (3)

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JP2023529371A true JP2023529371A (ja) 2023-07-10
JPWO2021248038A5 JPWO2021248038A5 (https=) 2024-06-10
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=)

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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
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
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

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Family Cites Families (23)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
GB8918616D0 (en) 1989-08-15 1989-09-27 Univ Glasgow Herpes simplex virus type 1 mutant
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
EP1082413B1 (en) 1998-05-28 2008-07-23 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
NZ522840A (en) 2000-06-01 2004-12-24 Univ North Carolina A parvovirus vector that carries a duplexed genome resulting in co-packaging of strands of plus and minus polarity tethered together
AU2002248297A1 (en) 2001-01-05 2002-07-16 Children's Hospital, Inc. Aav2 vectors and methods
NZ532635A (en) 2001-11-13 2007-05-31 Univ Pennsylvania A method of identifying unknown adeno-associated virus (AAV) sequences and a kit for the method
DK2359869T3 (en) 2001-12-17 2019-04-15 Univ Pennsylvania Sequences of adeno-associated virus (AAV) serotype 8, vectors containing these, and uses thereof
CN1856576B (zh) 2003-09-30 2011-05-04 宾夕法尼亚州立大学托管会 腺伴随病毒(aav)进化支、序列、含有这些序列的载体及它们的应用
EP1828390B1 (en) 2004-12-15 2012-06-13 The University Of North Carolina At Chapel Hill Chimeric vectors
US20110201686A1 (en) * 2006-06-23 2011-08-18 Yousef Al-Abed Inhibitors of ASS and Synuclein Aggregation
EP2396343B1 (en) 2009-02-11 2017-05-17 The University of North Carolina At Chapel Hill Modified virus vectors and methods of making and using the same
ES2683695T3 (es) 2010-01-12 2018-09-27 The University Of North Carolina At Chapel Hill Repeticiones terminales invertidas restrictivas para vectores virales
CA2788682C (en) 2010-02-05 2019-03-05 The University Of North Carolina At Chapel Hill Compositions and methods for enhanced parvovirus transduction
WO2014053879A1 (en) 2012-10-04 2014-04-10 Centre National De La Recherche Scientifique Cell penetrating peptides for intracellular delivery of molecules
US11390899B2 (en) * 2016-09-26 2022-07-19 SOLA Biosciences, LLC Cell-associated secretion-enhancing fusion proteins
WO2021138582A1 (en) * 2019-12-31 2021-07-08 The Johns Hopkins University Fusion proteins and methods of use thereof

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
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

Non-Patent Citations (2)

* Cited by examiner, † Cited by third party
Title
SCIENTIFIC REPORTS, vol. 7, no. 1, JPN6025020218, 2017, pages 1 - 13, ISSN: 0005597261 *
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
US20230234997A1 (en) 2023-07-27
WO2021248038A1 (en) 2021-12-09
EP4161953A1 (en) 2023-04-12
CA3183251A1 (en) 2021-12-09

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