CA3183251A1 - Compositions and methods for the treatment of synucleinopathies - Google Patents

Compositions and methods for the treatment of synucleinopathies

Info

Publication number
CA3183251A1
CA3183251A1 CA3183251A CA3183251A CA3183251A1 CA 3183251 A1 CA3183251 A1 CA 3183251A1 CA 3183251 A CA3183251 A CA 3183251A CA 3183251 A CA3183251 A CA 3183251A CA 3183251 A1 CA3183251 A1 CA 3183251A1
Authority
CA
Canada
Prior art keywords
fusion protein
synuclein
sequence
seq
domain
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Pending
Application number
CA3183251A
Other languages
English (en)
French (fr)
Inventor
Akinori HISHIYA
Keizo Koya
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Sola Biosciences LLC
Original Assignee
Sola Biosciences LLC
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Sola Biosciences LLC filed Critical Sola Biosciences LLC
Publication of CA3183251A1 publication Critical patent/CA3183251A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Genetics & Genomics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Biochemistry (AREA)
  • Biophysics (AREA)
  • 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)
  • Plant Pathology (AREA)
  • Obesity (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Diabetes (AREA)
  • Psychiatry (AREA)
  • Psychology (AREA)
  • Hospice & Palliative Care (AREA)
  • Peptides Or Proteins (AREA)
CA3183251A 2020-06-05 2021-06-04 Compositions and methods for the treatment of synucleinopathies Pending CA3183251A1 (en)

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 (1)

Publication Number Publication Date
CA3183251A1 true CA3183251A1 (en) 2021-12-09

Family

ID=76708439

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3183251A Pending CA3183251A1 (en) 2020-06-05 2021-06-04 Compositions and methods for the treatment of synucleinopathies

Country Status (6)

Country Link
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)

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

Family Cites Families (26)

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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
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
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
JP6345688B2 (ja) * 2012-12-05 2018-06-20 ソラ・バイオサイエンシズ・エルエルシー タンパク質発現増強ポリペプチド
US11390899B2 (en) * 2016-09-26 2022-07-19 SOLA Biosciences, LLC Cell-associated secretion-enhancing fusion proteins
US12162929B2 (en) * 2018-02-19 2024-12-10 New York University Alpha-synuclein single domain antibodies
WO2021138582A1 (en) * 2019-12-31 2021-07-08 The Johns Hopkins University Fusion proteins and methods of use thereof

Also Published As

Publication number Publication date
JP2023529371A (ja) 2023-07-10
CN116096737A (zh) 2023-05-09
US20230234997A1 (en) 2023-07-27
WO2021248038A1 (en) 2021-12-09
EP4161953A1 (en) 2023-04-12

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