HK1207109A1 - Methods and compositions for treating amyloid deposits - Google Patents

Methods and compositions for treating amyloid deposits Download PDF

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Publication number
HK1207109A1
HK1207109A1 HK15107526.1A HK15107526A HK1207109A1 HK 1207109 A1 HK1207109 A1 HK 1207109A1 HK 15107526 A HK15107526 A HK 15107526A HK 1207109 A1 HK1207109 A1 HK 1207109A1
Authority
HK
Hong Kong
Prior art keywords
mammal
compositions
methods
csf
amyloid deposits
Prior art date
Application number
HK15107526.1A
Other languages
English (en)
Chinese (zh)
Inventor
Beverly L. Davidson
Bradley T. Hyman
Original Assignee
The General Hospital Corporation
University Of Iowa Research Foundation
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 The General Hospital Corporation, University Of Iowa Research Foundation filed Critical The General Hospital Corporation
Publication of HK1207109A1 publication Critical patent/HK1207109A1/xx

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    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/775Apolipopeptides
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00Medicinal preparations containing peptides
    • A61K38/16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/162Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00Drugs for disorders of the nervous system
    • A61P25/28Drugs 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
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86Viral vectors
    • C12N15/861Adenoviral vectors
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N7/00Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2750/00MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA ssDNA viruses
    • C12N2750/00011Details
    • C12N2750/14011Parvoviridae
    • C12N2750/14111Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143Use 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)
  • 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)
  • Medicinal Chemistry (AREA)
  • Zoology (AREA)
  • Biomedical Technology (AREA)
  • Wood Science & Technology (AREA)
  • Biochemistry (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Molecular Biology (AREA)
  • Immunology (AREA)
  • Biophysics (AREA)
  • General Engineering & Computer Science (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • Toxicology (AREA)
  • Hospice & Palliative Care (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • General Chemical & Material Sciences (AREA)
  • Psychiatry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
HK15107526.1A 2012-05-18 2013-03-14 Methods and compositions for treating amyloid deposits HK1207109A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201261648801P 2012-05-18 2012-05-18
US61/648,801 2012-05-18
PCT/US2013/031725 WO2013172964A1 (en) 2012-05-18 2013-03-14 Methods and compositions for treating amyloid deposits

Publications (1)

Publication Number Publication Date
HK1207109A1 true HK1207109A1 (en) 2016-01-22

Family

ID=49584137

Family Applications (1)

Application Number Title Priority Date Filing Date
HK15107526.1A HK1207109A1 (en) 2012-05-18 2013-03-14 Methods and compositions for treating amyloid deposits

Country Status (12)

Country Link
US (1) US20150183850A1 (enExample)
EP (1) EP2850195B1 (enExample)
JP (2) JP6469000B2 (enExample)
CN (2) CN104540952A (enExample)
AU (1) AU2013263346B2 (enExample)
BR (1) BR112014028666B1 (enExample)
CA (1) CA2873890C (enExample)
ES (1) ES2786078T3 (enExample)
HK (1) HK1207109A1 (enExample)
IN (1) IN2014KN02672A (enExample)
RU (1) RU2673484C2 (enExample)
WO (1) WO2013172964A1 (enExample)

Families Citing this family (16)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR102584655B1 (ko) 2014-11-14 2023-10-06 보이저 테라퓨틱스, 인크. 조절성 폴리뉴클레오티드
WO2016196507A1 (en) 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
EP3307391A1 (en) * 2015-06-12 2018-04-18 INSERM (Institut National de la Santé et de la Recherche Médicale) Methods and pharmaceutical composition for the treatment of alzheimer's disease
EP3444344A4 (en) * 2016-04-14 2020-02-19 TAO Health Life Pharma Co., Ltd. AMYLOSPHEROID TYPE STRUCTURE (ASPD) AND PHARMACEUTICAL COMPOSITION
RU2758488C2 (ru) 2016-05-18 2021-10-28 Вояджер Терапьютикс, Инк. Модулирующие полинуклеотиды
WO2018027037A2 (en) * 2016-08-03 2018-02-08 University Of South Florida Reelin compositions for treatment of neurological disorders
AU2017318717B2 (en) * 2016-09-02 2024-10-24 Spark Therapeutics, Inc. Methods and vectors for treating CNS disorders
EP3309550A1 (en) * 2016-10-12 2018-04-18 sphingotec GmbH Method for the detection of apolipoprotein e4
BR112020006661A2 (pt) 2017-10-03 2020-10-13 Prevail Therapeutics, Inc. terapias de genes para distúrbios lipossomais
CN112501208A (zh) 2017-10-03 2021-03-16 普利维尔治疗公司 用于溶酶体障碍的基因疗法
JP7616995B2 (ja) * 2018-11-28 2025-01-17 プリベイル セラピューティクス,インコーポレーテッド 神経変性疾患のための遺伝子治療
CN114761569A (zh) * 2019-10-16 2022-07-15 康奈尔大学 阿尔茨海默氏病的基因疗法
JP2023520002A (ja) * 2020-03-31 2023-05-15 ▲ヌオ▼貝▲タイ▼医薬科技(上海)有限公司 細胞内タンパク質ミスフォールディング関連疾患およびリソソーム蓄積症におけるpi4k阻害物質の応用
CA3203006A1 (en) * 2020-11-25 2022-06-02 Prevail Therapeutics, Inc. Gene therapies for neurodegenerative disease
US20250297279A1 (en) * 2021-12-15 2025-09-25 Genans Biotechnology Co., Ltd Recombinant aav vectors and use thereof
CN116063405B (zh) * 2022-09-27 2025-08-29 广州译码基因科技有限公司 可提高AAV病毒包装能力的衣壳蛋白突变体MutC及其应用

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5589154A (en) * 1994-11-22 1996-12-31 Rutgers, The State University Of New Jersey Methods for the prevention or treatment of vascular hemorrhaging and Alzheimer's disease
US6436996B1 (en) * 1997-09-30 2002-08-20 Duke University Modulation of nitric oxide production
US6468524B1 (en) * 2000-03-22 2002-10-22 The United States Of America, As Represented By The Secretary Of The Department Of Health And Human Services AAV4 vector and uses thereof
US6855314B1 (en) 2000-03-22 2005-02-15 The United States Of America As Represented By The Department Of Health And Human Services AAV5 vector for transducing brain cells and lung cells
GB0322645D0 (en) * 2003-09-26 2003-10-29 Melacure Therapeutics Ab Use of antisecretory factor peptides
CA2591544A1 (en) * 2004-12-15 2006-06-22 The University Of North Carolina At Chapel Hill Chimeric vectors
MX2007013734A (es) * 2005-05-02 2008-03-14 Genzyme Corp Terapia genica para trastornos de la medula espinal.
KR101470472B1 (ko) * 2006-09-14 2014-12-09 메드제닉스 메디칼 이스라엘 리미티드 장기 지속형 약물 제형
HUE027278T2 (en) * 2007-05-16 2016-11-28 Brigham & Womens Hospital Inc Treatment of synucleinopathies
JP2013533847A (ja) * 2010-04-23 2013-08-29 ユニバーシティ オブ マサチューセッツ コレステロール関連障害のaavベースの治療

Also Published As

Publication number Publication date
JP2015520161A (ja) 2015-07-16
EP2850195A4 (en) 2015-12-09
CA2873890A1 (en) 2013-11-21
IN2014KN02672A (enExample) 2015-05-08
ES2786078T3 (es) 2020-10-08
JP6469000B2 (ja) 2019-02-13
RU2014151218A (ru) 2016-07-10
JP2019031564A (ja) 2019-02-28
WO2013172964A1 (en) 2013-11-21
AU2013263346A1 (en) 2014-12-04
EP2850195A1 (en) 2015-03-25
CA2873890C (en) 2022-12-06
BR112014028666A2 (pt) 2017-06-27
CN112574964A (zh) 2021-03-30
EP2850195B1 (en) 2020-01-22
US20150183850A1 (en) 2015-07-02
AU2013263346B2 (en) 2018-08-23
CN104540952A (zh) 2015-04-22
BR112014028666B1 (pt) 2021-12-07
RU2673484C2 (ru) 2018-11-27

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