CA3125294A1 - 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 Download PDF

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Publication number
CA3125294A1
CA3125294A1 CA3125294A CA3125294A CA3125294A1 CA 3125294 A1 CA3125294 A1 CA 3125294A1 CA 3125294 A CA3125294 A CA 3125294A CA 3125294 A CA3125294 A CA 3125294A CA 3125294 A1 CA3125294 A1 CA 3125294A1
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Canada
Prior art keywords
nucleic acid
acid sequence
mammal
raav particle
cells
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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
CA3125294A
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English (en)
French (fr)
Inventor
Shannon E. Boye
Sanford L. Boye
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.)
University of Florida Research Foundation Inc
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University of Florida Research Foundation Inc
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Application filed by University of Florida Research Foundation Inc filed Critical University of Florida Research Foundation Inc
Publication of CA3125294A1 publication Critical patent/CA3125294A1/en
Pending legal-status Critical Current

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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
    • 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
    • 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
    • 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

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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)
  • 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)
  • 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)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
CA3125294A 2019-01-23 2020-01-23 Highly efficient transduction and lateral spread in the retina by a novel aav virus enhanced by rational design Pending CA3125294A1 (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 (1)

Publication Number Publication Date
CA3125294A1 true CA3125294A1 (en) 2020-07-30

Family

ID=71737005

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3125294A Pending CA3125294A1 (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

Family Cites Families (12)

* 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
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
US20120322861A1 (en) 2007-02-23 2012-12-20 Barry John Byrne Compositions and Methods for Treating Diseases
SG10201800541SA (en) * 2010-04-23 2018-03-28 Univ Florida Raav-guanylate cyclase compositions and methods for treating leber's congenital amaurosis-1 (lca1)
US10308957B2 (en) * 2014-03-04 2019-06-04 University Of Florida Research Foundation, Inc. rAAV vectors and methods for transduction of photoreceptors and RPE cells
AU2015252797C1 (en) * 2014-05-02 2021-04-22 Genzyme Corporation AAV vectors for retinal and CNS gene therapy
WO2016149664A1 (en) 2015-03-18 2016-09-22 University Of Florida Research Foundation, Inc. Methods and compositions for restoration of cone function in bcm
EP3294894B8 (en) * 2015-05-12 2019-09-25 The U.S.A. as represented by the Secretary, Department of Health and Human Services Aav isolate and fusion protein comprising nerve growth factor signal peptide and parathyroid hormone
US20170348387A1 (en) * 2016-02-29 2017-12-07 The Trustees Of The University Of Pennsylvania Aav-mediated gene therapy for nphp5 lca-ciliopathy
US11584780B2 (en) * 2016-07-26 2023-02-21 Biomarin Pharmaceutical Inc. Adeno-associated virus capsid proteins
CA3114549A1 (en) * 2017-02-21 2018-08-30 University Of Florida Research Foundation, Incorporated Modified aav capsid proteins and uses thereof

Also Published As

Publication number Publication date
AU2020212026A1 (en) 2021-07-22
CN113347962B (zh) 2025-01-28
JP2022523050A (ja) 2022-04-21
US20220133909A1 (en) 2022-05-05
EP3914229A4 (en) 2022-11-02
EP3914229A1 (en) 2021-12-01
WO2020154535A1 (en) 2020-07-30
JP7624727B2 (ja) 2025-01-31
CN113347962A (zh) 2021-09-03
AU2020212026B2 (en) 2025-10-09

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