KR20220152192A - 알츠하이머병 및 기타 신경 퇴행성 질환의 치료를 위한 레트로머의 안정화 - Google Patents

알츠하이머병 및 기타 신경 퇴행성 질환의 치료를 위한 레트로머의 안정화 Download PDF

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KR20220152192A
KR20220152192A KR1020227022902A KR20227022902A KR20220152192A KR 20220152192 A KR20220152192 A KR 20220152192A KR 1020227022902 A KR1020227022902 A KR 1020227022902A KR 20227022902 A KR20227022902 A KR 20227022902A KR 20220152192 A KR20220152192 A KR 20220152192A
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South Korea
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retromeric
transgene
vps35
core protein
vps26b
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Korean (ko)
Inventor
스캇 에이. 스몰
그레고리 페츠코
야서 에이치. 커레시
사무엘 디. 왁솔
알렉산드리아 포브스
레베카 콕스
Original Assignee
더 트러스티스 오브 콜롬비아 유니버시티 인 더 시티 오브 뉴욕
코넬 유니버시티
메이라지티엑스 유케이 Ii 리미티드
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Publication of KR20220152192A publication Critical patent/KR20220152192A/ko
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • 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
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    • C07K—PEPTIDES
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    • 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
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    • 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
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    • 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
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    • 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
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    • 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)
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  • General Engineering & Computer Science (AREA)
  • Animal Behavior & Ethology (AREA)
  • Wood Science & Technology (AREA)
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  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Toxicology (AREA)
  • Microbiology (AREA)
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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Virology (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • 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)
KR1020227022902A 2019-12-05 2020-12-07 알츠하이머병 및 기타 신경 퇴행성 질환의 치료를 위한 레트로머의 안정화 Pending KR20220152192A (ko)

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

Publications (1)

Publication Number Publication Date
KR20220152192A true KR20220152192A (ko) 2022-11-15

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KR1020227022902A Pending KR20220152192A (ko) 2019-12-05 2020-12-07 알츠하이머병 및 기타 신경 퇴행성 질환의 치료를 위한 레트로머의 안정화

Country Status (12)

Country Link
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=)

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WO2023159238A1 (en) * 2022-02-21 2023-08-24 The Scripps Research Institute Mef2 transcriptional activators to treat neurologic conditions

Family Cites Families (13)

* 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
US20080214482A1 (en) * 2005-11-15 2008-09-04 Scott Small Retromer-based assays and methods for treating alzheimer's disease
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
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
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
WO2017191274A2 (en) * 2016-05-04 2017-11-09 Curevac Ag Rna encoding a therapeutic protein
WO2018112446A2 (en) * 2016-12-18 2018-06-21 Selonterra, Inc. Use of apoe4 motif-mediated genes for diagnosis and treatment of alzheimer's disease
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

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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
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
CN115297891A (zh) 2022-11-04
JP2023505271A (ja) 2023-02-08

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