AU2013263346B2 - Methods and compositions for treating amyloid deposits - Google Patents

Methods and compositions for treating amyloid deposits Download PDF

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Publication number
AU2013263346B2
AU2013263346B2 AU2013263346A AU2013263346A AU2013263346B2 AU 2013263346 B2 AU2013263346 B2 AU 2013263346B2 AU 2013263346 A AU2013263346 A AU 2013263346A AU 2013263346 A AU2013263346 A AU 2013263346A AU 2013263346 B2 AU2013263346 B2 AU 2013263346B2
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Australia
Prior art keywords
apoe
nucleic acid
pct
aav
vector
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AU2013263346A
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AU2013263346A1 (en
Inventor
Beverly L. Davidson
Bradley T. Hyman
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University of Iowa Research Foundation UIRF
General Hospital Corp
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University of Iowa Research Foundation UIRF
General Hospital Corp
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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
    • 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
    • C07K14/775—Apolipopeptides
    • 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
    • A61K38/162—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from virus
    • 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
    • C12N15/861—Adenoviral 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • 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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  • 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)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Biotechnology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Immunology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Neurology (AREA)
  • Neurosurgery (AREA)
  • Toxicology (AREA)
  • Epidemiology (AREA)
  • Microbiology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Hospice & Palliative Care (AREA)
  • Psychiatry (AREA)
  • Chemical Kinetics & Catalysis (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)
AU2013263346A 2012-05-18 2013-03-14 Methods and compositions for treating amyloid deposits Active AU2013263346B2 (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 (2)

Publication Number Publication Date
AU2013263346A1 AU2013263346A1 (en) 2014-12-04
AU2013263346B2 true AU2013263346B2 (en) 2018-08-23

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ID=49584137

Family Applications (1)

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AU2013263346A Active AU2013263346B2 (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) CN112574964A (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)

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CA2975583A1 (en) 2014-11-14 2016-05-19 Voyager Therapeutics, Inc. Modulatory polynucleotides
WO2016196507A1 (en) 2015-05-29 2016-12-08 University Of Iowa Research Foundation Methods of delivery of transgenes for treating brain diseases
US20180161395A1 (en) * 2015-06-12 2018-06-14 Inserm (Institut National De La Sante Et De La Recherche Medicale) Methods and pharmaceutical composition for the treatment of alzheimer's disease
JP7161401B2 (ja) * 2016-04-14 2022-10-26 公益財団法人神戸医療産業都市推進機構 アミロスフェロイド（ａｓｐｄ）様構造体及び医薬組成物
IL262784B2 (en) 2016-05-18 2023-10-01 Voyager Therapeutics Inc modulatory polynucleotides
NZ750291A (en) * 2016-08-03 2021-07-30 Univ South Florida Reelin compositions for treatment of neurological disorders
CN110121356A (zh) * 2016-09-02 2019-08-13 星火治疗股份有限公司 治疗cns疾病的方法和载体
EP3309550A1 (en) * 2016-10-12 2018-04-18 sphingotec GmbH Method for the detection of apolipoprotein e4
JP7361037B2 (ja) 2017-10-03 2023-10-13 プリベイル セラピューティクス，インコーポレーテッド ライソゾーム病の遺伝子治療
AU2018346102B2 (en) 2017-10-03 2023-05-11 Prevail Therapeutics, Inc. Gene therapies for lysosomal disorders
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
MX2022004524A (es) * 2019-10-16 2022-07-21 Univ Cornell Tratamiento génico para la enfermedad de alzheimer.
KR20230020946A (ko) * 2020-03-31 2023-02-13 누오-베타 파마슈티컬 테크놀로지 (상하이) 씨오., 엘티디. 중수소화 옥소페닐아르신 화합물 및 이의 용도
CN116670291A (zh) * 2020-11-25 2023-08-29 普利维尔治疗公司 用于神经退行性疾病的基因疗法
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

Citations (1)

* Cited by examiner, † Cited by third party
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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

Family Cites Families (9)

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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
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
NZ555830A (en) * 2004-12-15 2009-01-31 Univ North Carolina Chimeric vectors
DK1879623T3 (da) * 2005-05-02 2012-12-17 Genzyme Corp Genterapi til rygmarvssygdomme
WO2008033375A2 (en) * 2006-09-14 2008-03-20 Medgenics Ltd. Long lasting drug formulations
HUE027278T2 (en) * 2007-05-16 2016-11-28 Brigham & Womens Hospital Inc Treatment of synucleinopathies
CA2833912C (en) * 2010-04-23 2021-09-21 University Of Massachusetts Aav-based treatment of cholesterol-related disorders

Patent Citations (1)

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

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
FENG XUAN ET AL, "Adeno-associated viral vector-mediated ApoE expression in Alzheimer's disease mice: low CNS immune response, long-term expression, and astrocyte specificity.", FRONTIERS IN BIOSCIENCE, 2004, pages 1540 - 1546 *

Also Published As

Publication number Publication date
EP2850195A4 (en) 2015-12-09
JP6469000B2 (ja) 2019-02-13
EP2850195A1 (en) 2015-03-25
CN112574964A (zh) 2021-03-30
RU2673484C2 (ru) 2018-11-27
CA2873890C (en) 2022-12-06
HK1207109A1 (en) 2016-01-22
IN2014KN02672A (enExample) 2015-05-08
JP2019031564A (ja) 2019-02-28
RU2014151218A (ru) 2016-07-10
BR112014028666B1 (pt) 2021-12-07
CA2873890A1 (en) 2013-11-21
EP2850195B1 (en) 2020-01-22
ES2786078T3 (es) 2020-10-08
US20150183850A1 (en) 2015-07-02
CN104540952A (zh) 2015-04-22
WO2013172964A1 (en) 2013-11-21
JP2015520161A (ja) 2015-07-16
BR112014028666A2 (pt) 2017-06-27
AU2013263346A1 (en) 2014-12-04

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