CA3073937A1 - Recombinant adeno-associated vectors - Google Patents

Recombinant adeno-associated vectors Download PDF

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Publication number
CA3073937A1
CA3073937A1 CA3073937A CA3073937A CA3073937A1 CA 3073937 A1 CA3073937 A1 CA 3073937A1 CA 3073937 A CA3073937 A CA 3073937A CA 3073937 A CA3073937 A CA 3073937A CA 3073937 A1 CA3073937 A1 CA 3073937A1
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CA
Canada
Prior art keywords
vectors
raavrec3
aav
nucleic acid
cell
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
CA3073937A
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English (en)
French (fr)
Inventor
Matthew During
Original Assignee
Ovid Therapeutics Inc
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 Ovid Therapeutics Inc filed Critical Ovid Therapeutics Inc
Publication of CA3073937A1 publication Critical patent/CA3073937A1/en
Pending legal-status Critical Current

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    • 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
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • 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
    • 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
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • 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
    • 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
    • A61K48/0008—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—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 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • 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
    • C12N2750/14121—Viruses as such, e.g. new isolates, mutants or their genomic sequences
    • 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
    • C12N2750/14122—New viral proteins or individual genes, new structural or functional aspects of known viral proteins or genes
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • General Health & Medical Sciences (AREA)
  • Biomedical Technology (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Virology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Plant Pathology (AREA)
  • Physics & Mathematics (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Veterinary Medicine (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicinal Preparation (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Peptides Or Proteins (AREA)
CA3073937A 2017-08-25 2018-08-22 Recombinant adeno-associated vectors Pending CA3073937A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762550458P 2017-08-25 2017-08-25
US62/550,458 2017-08-25
PCT/US2018/047466 WO2019040586A1 (en) 2017-08-25 2018-08-22 RECOMBINANT ADENO-ASSOCIATE VECTORS

Publications (1)

Publication Number Publication Date
CA3073937A1 true CA3073937A1 (en) 2019-02-28

Family

ID=65439655

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3073937A Pending CA3073937A1 (en) 2017-08-25 2018-08-22 Recombinant adeno-associated vectors

Country Status (10)

Country Link
US (2) US20190085358A1 (https=)
EP (1) EP3655041A4 (https=)
JP (1) JP2020533968A (https=)
KR (1) KR20200042935A (https=)
CN (1) CN111163811A (https=)
AU (1) AU2018320849A1 (https=)
CA (1) CA3073937A1 (https=)
IL (1) IL272655A (https=)
MX (1) MX2020002148A (https=)
WO (1) WO2019040586A1 (https=)

Families Citing this family (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022514194A (ja) 2018-11-21 2022-02-10 セルテゴ セラピューティクス インコーポレイテッド 自殺のリスクを低減し、うつ病を迅速に軽減するためのガボキサドール
CA3149164A1 (en) * 2019-09-03 2021-03-11 Suma Krishnan Compositions and methods for the treatment of congenital ichthyoses
MX2022014599A (es) 2020-05-20 2022-12-16 Certego Therapeutics Inc Gaboxadol deuterado en anillo y su uso para el tratamiento de trastornos psiquiatricos.
CA3191543A1 (en) * 2020-09-04 2022-03-10 Lei Cao Novel engineered capsid serotype of recombinant adeno-associated viral vector with enhanced transduction efficiency and widespread distribution in the brain
US20230086710A1 (en) * 2021-09-14 2023-03-23 California Institute Of Technology Aav capsid variants

Family Cites Families (30)

* Cited by examiner, † Cited by third party
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WO1999061601A2 (en) * 1998-05-28 1999-12-02 The Government Of The United States Of America, As Represented By The Secretary, Department Of Health And Human Services Aav5 vector and uses thereof
JP4969002B2 (ja) * 1999-06-08 2012-07-04 ユニバーシテイ・オブ・アイオワ・リサーチ・フアウンデーシヨン ｒＡＡＶ形質導入を増加するための化合物および方法
AU2003259803B2 (en) * 2002-08-12 2007-08-02 The Regents Of The University Of Michigan Diagnosis and treatment of tuberous sclerosis
US7427396B2 (en) * 2004-06-03 2008-09-23 Genzyme Corporation AAV vectors for gene delivery to the lung
US7838657B2 (en) * 2004-12-03 2010-11-23 University Of Massachusetts Spinal muscular atrophy (SMA) treatment via targeting of SMN2 splice site inhibitory sequences
ES2525067T3 (es) * 2005-04-07 2014-12-17 The Trustees Of The University Of Pennsylvania Método de incremento de la función de un vector de AAV
US9415121B2 (en) * 2008-12-19 2016-08-16 Nationwide Children's Hospital Delivery of MECP2 polynucleotide using recombinant AAV9
EP2692868A1 (en) * 2012-08-02 2014-02-05 Universitat Autònoma De Barcelona Adeno-associated viral (AAV) vectors useful for transducing adipose tissue
MX380973B (es) * 2013-05-01 2025-03-12 Genzyme Corp Composiciones y metodos para tratar la atrofia muscular espinal.
IL297919A (en) * 2013-07-22 2023-01-01 Childrens Hospital Philadelphia Variant aav and compositions, methods and uses for gene transfer to cells, organs and tissues
GB201403684D0 (en) * 2014-03-03 2014-04-16 King S College London Vector
WO2015171547A1 (en) * 2014-05-05 2015-11-12 Ovid Therapeutics Inc. Methods of treating cognitive impairment associated with neurodegenerative disorders
WO2015184259A1 (en) * 2014-05-30 2015-12-03 The Board Of Trustees Of The Leland Stanford Junior University Compositions and methods to treat latent viral infections
EP3151866B1 (en) * 2014-06-09 2023-03-08 Voyager Therapeutics, Inc. Chimeric capsids
EP3200830B1 (en) * 2014-10-03 2020-09-09 University of Massachusetts High efficiency library-identified aav vectors
KR102423442B1 (ko) * 2015-12-11 2022-07-20 캘리포니아 인스티튜트 오브 테크놀로지 아데노-관련 바이러스를 지시하기 위한 타겟팅 펩타이드
MX2018007234A (es) * 2015-12-14 2018-11-09 Univ Pennsylvania Vectores virales adenoasociados utiles en el tratamiento de atrofia muscular espinal.
US11364308B2 (en) * 2016-05-13 2022-06-21 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
US11905312B2 (en) * 2017-02-15 2024-02-20 The University Of North Carolina At Chapel Hill Methods and compositions for gene transfer across the vasculature
CN110621318A (zh) * 2017-03-15 2019-12-27 奥维德医疗公司 排他地由设计者药物激活的设计者受体在治疗癫痫发作紊乱中的用途
US20190184034A1 (en) * 2017-12-20 2019-06-20 Ovid Therapeutics Inc. USE OF hM4Di IN THE TREATMENT OF SEIZURE DISORDERS
KR20210040358A (ko) * 2018-06-14 2021-04-13 오비드 테라퓨틱스 인크. 안젤만 증후군의 치료에서 mir-92a 또는 mir-145의 사용
CN113966399A (zh) * 2018-09-26 2022-01-21 加州理工学院 用于靶向基因疗法的腺相关病毒组合物
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CA3116391A1 (en) * 2020-09-14 2022-03-14 President And Fellows Of Harvard College Recombinant adeno associated virus encoding clarin-1 and uses thereof
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Also Published As

Publication number Publication date
CN111163811A (zh) 2020-05-15
WO2019040586A1 (en) 2019-02-28
IL272655A (en) 2020-03-31
KR20200042935A (ko) 2020-04-24
AU2018320849A1 (en) 2020-03-05
US20210371880A1 (en) 2021-12-02
JP2020533968A (ja) 2020-11-26
MX2020002148A (es) 2020-07-20
EP3655041A1 (en) 2020-05-27
EP3655041A4 (en) 2021-04-21
US20190085358A1 (en) 2019-03-21

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