CN115297891A - 用于治疗阿尔茨海默病和其他神经退行性病症的逆转运复合体的稳定化 - Google Patents

用于治疗阿尔茨海默病和其他神经退行性病症的逆转运复合体的稳定化 Download PDF

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Publication number
CN115297891A
CN115297891A CN202080093935.2A CN202080093935A CN115297891A CN 115297891 A CN115297891 A CN 115297891A CN 202080093935 A CN202080093935 A CN 202080093935A CN 115297891 A CN115297891 A CN 115297891A
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Prior art keywords
transgene
vps35
composition
antitransport
core protein
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Pending
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CN202080093935.2A
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English (en)
Chinese (zh)
Inventor
S·A·斯莫尔
G·佩茨科
Y·H·库雷什
S·D·瓦克萨尔
A·福布斯
R·考克斯
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Meragutex Uk No 2 Co ltd
Columbia University in the City of New York
Cornell University
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Meragutex Uk No 2 Co ltd
Columbia University in the City of New York
Cornell University
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Application filed by Meragutex Uk No 2 Co ltd, Columbia University in the City of New York, Cornell University filed Critical Meragutex Uk No 2 Co ltd
Priority to CN202510180683.1A priority Critical patent/CN120114618A/zh
Publication of CN115297891A publication Critical patent/CN115297891A/zh
Pending legal-status Critical Current

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    • 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
    • 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
    • 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
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—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
    • A61P25/28—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
    • 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
    • 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
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/008—Vector systems having a special element relevant for transcription cell type or tissue specific enhancer/promoter combination
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/40—Vector systems having a special element relevant for transcription being an insulator
    • 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
    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Zoology (AREA)
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  • Pharmacology & Pharmacy (AREA)
  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
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  • Epidemiology (AREA)
  • Hospice & Palliative Care (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Immunology (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CN202080093935.2A 2019-12-05 2020-12-07 用于治疗阿尔茨海默病和其他神经退行性病症的逆转运复合体的稳定化 Pending CN115297891A (zh)

Priority Applications (1)

Application Number Priority Date Filing Date Title
CN202510180683.1A CN120114618A (zh) 2019-12-05 2020-12-07 用于治疗阿尔茨海默病和其他神经退行性病症的逆转运复合体的稳定化

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201962943999P 2019-12-05 2019-12-05
US62/943,999 2019-12-05
US202063074578P 2020-09-04 2020-09-04
US63/074,578 2020-09-04
PCT/US2020/063627 WO2021113824A1 (en) 2019-12-05 2020-12-07 Stabilization of retromer for the treatment of alzheimer's disease and other neurodegenerative disorders

Related Child Applications (1)

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CN202510180683.1A Division CN120114618A (zh) 2019-12-05 2020-12-07 用于治疗阿尔茨海默病和其他神经退行性病症的逆转运复合体的稳定化

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Publication Number Publication Date
CN115297891A true CN115297891A (zh) 2022-11-04

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CN202080093935.2A Pending CN115297891A (zh) 2019-12-05 2020-12-07 用于治疗阿尔茨海默病和其他神经退行性病症的逆转运复合体的稳定化
CN202510180683.1A Pending CN120114618A (zh) 2019-12-05 2020-12-07 用于治疗阿尔茨海默病和其他神经退行性病症的逆转运复合体的稳定化

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US (1) US20230021959A1 (https=)
EP (1) EP4069315A4 (https=)
JP (2) JP2023505271A (https=)
KR (1) KR20220152192A (https=)
CN (2) CN115297891A (https=)
AU (1) AU2020395327A1 (https=)
BR (1) BR112022011073A2 (https=)
CA (1) CA3160785A1 (https=)
IL (1) IL293599A (https=)
MX (1) MX2022006895A (https=)
PH (1) PH12022551373A1 (https=)
WO (1) WO2021113824A1 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2023159238A1 (en) * 2022-02-21 2023-08-24 The Scripps Research Institute Mef2 transcriptional activators to treat neurologic conditions

Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080214482A1 (en) * 2005-11-15 2008-09-04 Scott Small Retromer-based assays and methods for treating alzheimer's disease
US20160250182A1 (en) * 2013-01-11 2016-09-01 The Trustees Of Columbia University In The City Of Rab7l1 interacts with lrrk2 to modify intraneuronal protein sorting and parkinson's disease risk
US20190241633A1 (en) * 2016-05-04 2019-08-08 Curevac Ag Rna encoding a therapeutic protein
US20190338363A1 (en) * 2016-12-18 2019-11-07 Selonterra, Inc. Use of apoe4 motif-mediated genes for diagnosis and treatment of alzheimer's disease

Family Cites Families (9)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
EP1938104A2 (en) * 2005-10-17 2008-07-02 Institute for Systems Biology Tissue-and serum-derived glycoproteins and methods of their use
WO2009108274A2 (en) * 2008-02-26 2009-09-03 The University Of North Carolina At Chapel Hill Methods and compositions for adeno-associated virus (aav) with hi loop mutations
EP2607900A1 (de) * 2011-12-22 2013-06-26 Protagen AG Markersequenzen für Brustkrebs und deren Verwendung
MX367100B (es) * 2012-02-17 2019-08-05 The Children´S Hospital Of Philadelphia Composiciones del vector aav y metodos para su transferencia genetica a celulas, organos y tejidos.
WO2014110481A2 (en) * 2013-01-11 2014-07-17 The Trustees Of Columbia University In The City Of New York Rab7l1 interacts with lrrk2 to modify intraneuronal protein sorting and parkinson' s disease risk
GB201409519D0 (en) * 2014-05-29 2014-07-16 Univ Leicester Senescent cell biomarkers
AU2016324317A1 (en) * 2015-09-17 2018-03-08 Coda Biotherapeutics, Inc. Compositions and methods for treating neurological disorders
BR112020006661A2 (pt) * 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
US20190345516A1 (en) * 2018-03-12 2019-11-14 Musc Foundation For Research Development Methods for treating eye disease

Patent Citations (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080214482A1 (en) * 2005-11-15 2008-09-04 Scott Small Retromer-based assays and methods for treating alzheimer's disease
US20160250182A1 (en) * 2013-01-11 2016-09-01 The Trustees Of Columbia University In The City Of Rab7l1 interacts with lrrk2 to modify intraneuronal protein sorting and parkinson's disease risk
US20190241633A1 (en) * 2016-05-04 2019-08-08 Curevac Ag Rna encoding a therapeutic protein
US20190338363A1 (en) * 2016-12-18 2019-11-07 Selonterra, Inc. Use of apoe4 motif-mediated genes for diagnosis and treatment of alzheimer's disease

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
MEGHA MOHAN: "Role of the VPS35 D620N mutation in Parkinson\'s disease", PARKINSONISM AND RELATED DISORDERS, vol. 36, 31 March 2017 (2017-03-31), pages 11 *

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Publication number Publication date
CA3160785A1 (en) 2021-06-10
AU2020395327A1 (en) 2022-07-21
BR112022011073A2 (pt) 2022-09-20
MX2022006895A (es) 2022-11-09
CN120114618A (zh) 2025-06-10
WO2021113824A1 (en) 2021-06-10
EP4069315A1 (en) 2022-10-12
KR20220152192A (ko) 2022-11-15
US20230021959A1 (en) 2023-01-26
JP2025149978A (ja) 2025-10-08
PH12022551373A1 (en) 2023-05-08
IL293599A (en) 2022-08-01
EP4069315A4 (en) 2024-03-20
JP2023505271A (ja) 2023-02-08

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