AU2020212026B2 - Highly efficient transduction and lateral spread in the retina by a novel AAV virus enhanced by rational design - Google Patents

Highly efficient transduction and lateral spread in the retina by a novel AAV virus enhanced by rational design

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Publication number
AU2020212026B2
AU2020212026B2 AU2020212026A AU2020212026A AU2020212026B2 AU 2020212026 B2 AU2020212026 B2 AU 2020212026B2 AU 2020212026 A AU2020212026 A AU 2020212026A AU 2020212026 A AU2020212026 A AU 2020212026A AU 2020212026 B2 AU2020212026 B2 AU 2020212026B2
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AU
Australia
Prior art keywords
acid sequence
promoter
nucleic acid
retinal
raav
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Application number
AU2020212026A
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English (en)
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AU2020212026A1 (en
Inventor
Sanford L. Boye
Shannon E. Boye
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University of Florida Research Foundation Inc
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University of Florida Research Foundation Inc
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Publication of AU2020212026A1 publication Critical patent/AU2020212026A1/en
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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
    • 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
    • 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
    • A61K48/0075—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 delivery route, e.g. oral, subcutaneous
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • 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
    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0048—Eye, e.g. artificial tears
    • 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

Landscapes

  • 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)
  • General Health & Medical Sciences (AREA)
  • Molecular Biology (AREA)
  • Biomedical Technology (AREA)
  • Medicinal Chemistry (AREA)
  • Biochemistry (AREA)
  • General Engineering & Computer Science (AREA)
  • Biophysics (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • Virology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Epidemiology (AREA)
  • Plant Pathology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Physics & Mathematics (AREA)
  • Microbiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
AU2020212026A 2019-01-23 2020-01-23 Highly efficient transduction and lateral spread in the retina by a novel AAV virus enhanced by rational design Active AU2020212026B2 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201962795695P 2019-01-23 2019-01-23
US62/795,695 2019-01-23
PCT/US2020/014838 WO2020154535A1 (en) 2019-01-23 2020-01-23 Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design

Publications (2)

Publication Number Publication Date
AU2020212026A1 AU2020212026A1 (en) 2021-07-22
AU2020212026B2 true AU2020212026B2 (en) 2025-10-09

Family

ID=71737005

Family Applications (1)

Application Number Title Priority Date Filing Date
AU2020212026A Active AU2020212026B2 (en) 2019-01-23 2020-01-23 Highly efficient transduction and lateral spread in the retina by a novel AAV virus enhanced by rational design

Country Status (7)

Country Link
US (1) US20220133909A1 (enExample)
EP (1) EP3914229A4 (enExample)
JP (1) JP7624727B2 (enExample)
CN (1) CN113347962B (enExample)
AU (1) AU2020212026B2 (enExample)
CA (1) CA3125294A1 (enExample)
WO (1) WO2020154535A1 (enExample)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2024223696A1 (en) * 2023-04-24 2024-10-31 Universiteit Gent Compounds to treat inherited retinal disease

Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2018156654A1 (en) * 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof
US20180355376A1 (en) * 2015-05-12 2018-12-13 The United States Of America,As Represented By The Secretary, Department Of Health And Human Service Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone

Family Cites Families (10)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU7467598A (en) * 1997-04-21 1998-11-13 University Of Florida Materials and methods for treatment of retinal diseases
US20070015238A1 (en) 2002-06-05 2007-01-18 Snyder Richard O Production of pseudotyped recombinant AAV virions
EP3409296A1 (en) * 2005-04-07 2018-12-05 The Trustees of the University of Pennsylvania Method of increasing the function of an aav vector
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
AU2011242527B2 (en) * 2010-04-23 2016-05-19 University Of Florida Research Foundation, Inc. rAAV-guanylate cyclase compositions and methods for treating Leber's congenital amaurosis-1 (LCA1)
EP3113787B1 (en) * 2014-03-04 2019-12-04 University of Florida Research Foundation, Inc. Improved raav vectors and methods for transduction of photoreceptors and rpe cells
LT3137497T (lt) * 2014-05-02 2021-07-26 Genzyme Corporation Aav vektoriai, skirti tinklainės ir cns genų terapijai
US10533187B2 (en) 2015-03-18 2020-01-14 University Of Florida Research Foundation, Incorporated Methods and compositions for restoration of cone function in BCM
US20170348387A1 (en) * 2016-02-29 2017-12-07 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for nphp5 lca-ciliopathy
EP3491008A2 (en) * 2016-07-26 2019-06-05 BioMarin Pharmaceutical Inc. Novel adeno-associated virus capsid proteins

Patent Citations (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20180355376A1 (en) * 2015-05-12 2018-12-13 The United States Of America,As Represented By The Secretary, Department Of Health And Human Service Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
WO2018156654A1 (en) * 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof

Non-Patent Citations (1)

* Cited by examiner, † Cited by third party
Title
Nonnenmcaher, M, et al., 'Intracellular Transport of Recombinant Adeno-Associated Virus Vectors', Gene Therapy, 2012, vol. 19, no. 6, pages 649 - 658 *

Also Published As

Publication number Publication date
JP7624727B2 (ja) 2025-01-31
CN113347962A (zh) 2021-09-03
US20220133909A1 (en) 2022-05-05
WO2020154535A1 (en) 2020-07-30
EP3914229A4 (en) 2022-11-02
CA3125294A1 (en) 2020-07-30
JP2022523050A (ja) 2022-04-21
CN113347962B (zh) 2025-01-28
EP3914229A1 (en) 2021-12-01
AU2020212026A1 (en) 2021-07-22

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