AU2019325332B2 - Compositions and methods for modulating transduction efficiency of adeno-associated viruses - Google Patents

Compositions and methods for modulating transduction efficiency of adeno-associated viruses

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Publication number
AU2019325332B2
AU2019325332B2 AU2019325332A AU2019325332A AU2019325332B2 AU 2019325332 B2 AU2019325332 B2 AU 2019325332B2 AU 2019325332 A AU2019325332 A AU 2019325332A AU 2019325332 A AU2019325332 A AU 2019325332A AU 2019325332 B2 AU2019325332 B2 AU 2019325332B2
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Australia
Prior art keywords
gpr108
cell
aav
cells
transduction
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AU2019325332A
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AU2019325332A1 (en
Inventor
Amanda DUDEK
Nerea Zabaleta LASARTE
Luk H. VANDENBERGHE
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Schepens Eye Research Institute Inc
Massachusetts Eye and Ear
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Massachusetts Eye and Ear Infirmary
Schepens Eye Research Institute Inc
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    • 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
    • C07K14/705—Receptors; Cell surface antigens; Cell surface determinants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/62—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being a protein, peptide or polyamino acid
    • A61K47/64—Drug-peptide, drug-protein or drug-polyamino acid conjugates, i.e. the modifying agent being a peptide, protein or polyamino acid which is covalently bonded or complexed to a therapeutically active agent
    • 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
    • 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
    • C12N7/00—Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • 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)
  • Organic Chemistry (AREA)
  • Engineering & Computer Science (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Zoology (AREA)
  • General Health & Medical Sciences (AREA)
  • Wood Science & Technology (AREA)
  • Molecular Biology (AREA)
  • Biochemistry (AREA)
  • Virology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Medicinal Chemistry (AREA)
  • Biomedical Technology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Immunology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Cell Biology (AREA)
  • Toxicology (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)
  • Medicinal Preparation (AREA)
AU2019325332A 2018-08-21 2019-08-21 Compositions and methods for modulating transduction efficiency of adeno-associated viruses Active AU2019325332B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862720859P 2018-08-21 2018-08-21
US62/720,859 2018-08-21
PCT/US2019/047546 WO2020041498A1 (en) 2018-08-21 2019-08-21 Compositions and methods for modulating transduction efficiency of adeno-associated viruses

Publications (2)

Publication Number Publication Date
AU2019325332A1 AU2019325332A1 (en) 2021-03-18
AU2019325332B2 true AU2019325332B2 (en) 2025-11-13

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US (1) US12195745B2 (enExample)
EP (1) EP3841109A4 (enExample)
JP (1) JP7451497B2 (enExample)
AU (1) AU2019325332B2 (enExample)
WO (1) WO2020041498A1 (enExample)

Families Citing this family (8)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BR112020022858A2 (pt) 2018-05-11 2021-02-23 Massachusetts Eye And Ear Infirmary tropismo específico hepático de vírus adeno-associado
JP2023523132A (ja) 2020-04-09 2023-06-02 ユニバーシティ オブ シェフィールド 遺伝子療法による治療
WO2022103826A1 (en) * 2020-11-10 2022-05-19 The Children's Hospital Of Philadelphia Compositons and methods for in vivo gene transfer
GB202215198D0 (en) 2022-10-14 2022-11-30 Univ Sheffield Gene therapy treatment
WO2025114699A1 (en) 2023-11-30 2025-06-05 Cytomos Limited Cell selection
WO2025188755A1 (en) 2024-03-04 2025-09-12 Kate Therapeutics, Inc. Adeno-associated virus compositions for the treatment of duchenne muscular dystrophy
WO2025226842A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Expression control by drg-expressed mirnas
WO2025226841A1 (en) 2024-04-24 2025-10-30 Kate Therapeutics, Inc. Gene therapy approach for treating disorders associated with tnnt2

Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005072364A2 (en) * 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector

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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
EP1572893B1 (en) 2001-11-09 2009-01-07 THE GOVERNMENT OF THE UNITED STATES OF AMERICA as represented by THE SECRETARY of the DEPARTMENT OF HEALTH AND HUMAN SERVICES Production of adeno-associated virus in insect cells
NZ536274A (en) * 2002-05-01 2008-04-30 Univ Florida Improved rAAV expression systems for genetic modification of specific capsid proteins
ES2428218T3 (es) * 2005-04-07 2013-11-06 The Trustees Of The University Of Pennsylvania Cápsides de AAV rh48 modificadas, composiciones que las contienen y usos de las mismas
WO2014124282A1 (en) * 2013-02-08 2014-08-14 The Trustees Of The University Of Pennsylvania Enhanced aav-mediated gene transfer for retinal therapies
EP3209311B1 (en) * 2014-10-21 2024-03-06 University of Massachusetts Recombinant aav variants and uses thereof
US10081659B2 (en) * 2015-04-06 2018-09-25 The United States Of America, As Represented By The Secretary, Dept. Of Health And Human Services Adeno-associated vectors for enhanced transduction and reduced immunogenicity
WO2017083423A1 (en) * 2015-11-10 2017-05-18 The Board Of Trustees Of The Leland Stanford Junior University Methods and compositions for modulating aav infection
US11326182B2 (en) * 2016-04-29 2022-05-10 Voyager Therapeutics, Inc. Compositions for the treatment of disease
EP4209501A1 (en) * 2016-05-13 2023-07-12 4D Molecular Therapeutics Inc. Adeno-associated virus variant capsids and methods of use thereof
US11584780B2 (en) 2016-07-26 2023-02-21 Biomarin Pharmaceutical Inc. Adeno-associated virus capsid proteins
SG10201913002QA (en) * 2016-08-15 2020-03-30 Genzyme Corp Methods for detecting aav
US20180182497A1 (en) * 2016-12-22 2018-06-28 DxRx, Inc. Creating engagement with an inner circle social network in substance abuse treatment
US20210292373A1 (en) 2018-07-10 2021-09-23 University Of Florida Research Foundation, Incorporated Aav vp1u chimeras
TW202532428A (zh) * 2019-04-26 2025-08-16 美商愛德維仁生物科技公司 用於玻璃體內遞送之變異體aav蛋白殼

Patent Citations (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005072364A2 (en) * 2004-01-27 2005-08-11 University Of Florida A modified baculovirus expression system for production of pseudotyped raav vector

Also Published As

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JP2021533795A (ja) 2021-12-09
EP3841109A1 (en) 2021-06-30
WO2020041498A1 (en) 2020-02-27
JP7451497B2 (ja) 2024-03-18
US20210317478A1 (en) 2021-10-14
US12195745B2 (en) 2025-01-14
AU2019325332A1 (en) 2021-03-18
EP3841109A4 (en) 2022-09-21

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