CA3255255A1 - Adeno-associated viral vectors for the delivery of nucleic acids to retinal cells - Google Patents

Adeno-associated viral vectors for the delivery of nucleic acids to retinal cells

Info

Publication number
CA3255255A1
CA3255255A1 CA3255255A CA3255255A CA3255255A1 CA 3255255 A1 CA3255255 A1 CA 3255255A1 CA 3255255 A CA3255255 A CA 3255255A CA 3255255 A CA3255255 A CA 3255255A CA 3255255 A1 CA3255255 A1 CA 3255255A1
Authority
CA
Canada
Prior art keywords
polypeptide
seq
amino acid
vector
acid sequence
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
CA3255255A
Other languages
English (en)
French (fr)
Inventor
Leah Caroline THOMAS BYRNE
Molly E. JOHNSON
William Richard Stauffer
Bilge Esin OZTURK
Original Assignee
University of Pittsburgh
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 University of Pittsburgh filed Critical University of Pittsburgh
Publication of CA3255255A1 publication Critical patent/CA3255255A1/en
Pending legal-status Critical Current

Links

Classifications

    • 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
    • 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
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • 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
    • 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
    • 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
    • C12N2750/14145—Special targeting system for viral vectors

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Medicinal Chemistry (AREA)
  • Molecular Biology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Veterinary Medicine (AREA)
  • Biomedical Technology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Virology (AREA)
  • General Engineering & Computer Science (AREA)
  • Epidemiology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Physics & Mathematics (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Proteomics, Peptides & Aminoacids (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)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
CA3255255A 2022-03-30 2023-03-30 Adeno-associated viral vectors for the delivery of nucleic acids to retinal cells Pending CA3255255A1 (en)

Applications Claiming Priority (27)

Application Number Priority Date Filing Date Title
US202263325541P 2022-03-30 2022-03-30
US202263325542P 2022-03-30 2022-03-30
US202263325548P 2022-03-30 2022-03-30
US202263325558P 2022-03-30 2022-03-30
US202263325553P 2022-03-30 2022-03-30
US202263325544P 2022-03-30 2022-03-30
US202263325543P 2022-03-30 2022-03-30
US202263325540P 2022-03-30 2022-03-30
US202263325559P 2022-03-30 2022-03-30
US202263325562P 2022-03-30 2022-03-30
US202263325551P 2022-03-30 2022-03-30
US202263325550P 2022-03-30 2022-03-30
US202263325555P 2022-03-30 2022-03-30
US63/325,543 2022-03-30
US63/325,555 2022-03-30
US63/325,540 2022-03-30
US63/325,544 2022-03-30
US63/325,553 2022-03-30
US63/325,562 2022-03-30
US63/325,550 2022-03-30
US63/325,551 2022-03-30
US63/325,559 2022-03-30
US63/325,541 2022-03-30
US63/325,542 2022-03-30
US63/325,548 2022-03-30
US63/325,558 2022-03-30
PCT/US2023/016871 WO2023192463A2 (en) 2022-03-30 2023-03-30 Adeno-associated virus vectors for nucleic acid delivery to retinal cells

Publications (1)

Publication Number Publication Date
CA3255255A1 true CA3255255A1 (en) 2023-10-05

Family

ID=88203541

Family Applications (1)

Application Number Title Priority Date Filing Date
CA3255255A Pending CA3255255A1 (en) 2022-03-30 2023-03-30 Adeno-associated viral vectors for the delivery of nucleic acids to retinal cells

Country Status (11)

Country Link
US (1) US20250327092A1 (https=)
EP (1) EP4499848A4 (https=)
JP (1) JP2025510975A (https=)
KR (1) KR20250006360A (https=)
CN (1) CN119213136A (https=)
AU (1) AU2023243048A1 (https=)
CA (1) CA3255255A1 (https=)
IL (1) IL316014A (https=)
MX (1) MX2024011963A (https=)
TW (1) TW202405180A (https=)
WO (1) WO2023192463A2 (https=)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025072688A2 (en) * 2023-09-27 2025-04-03 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Adeno-associated virus vectors for nucleic acid delivery to retinal cells, for nucleic acid delivery across retinal regions, or for nucleic acid delivery to retinal ganglion cells and/or retinal pigment epithelium cells

Family Cites Families (6)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN105755044A (zh) * 2011-04-22 2016-07-13 加利福尼亚大学董事会 具有变异衣壳的腺相关病毒病毒体及其使用方法
CN110612353A (zh) * 2017-03-03 2019-12-24 加利福尼亚大学董事会 经由抑制性tRNAs和脱氨酶对突变进行RNA靶向
CA3053154A1 (en) * 2017-06-30 2019-01-03 The Regents Of The University Of California Adeno-associated virus virions with variant capsids and methods of use thereof
AU2019227910B2 (en) * 2018-02-28 2025-07-17 The Regents Of The University Of California A modular system for gene and protein delivery based on AAV
US20220348613A1 (en) * 2019-08-28 2022-11-03 University of Pittsburgh - of the Commonwealath System of Higher Ecucation Adeno-associated viruses and methods and materials for making and using adeno-associated viruses
US20220313722A1 (en) * 2019-09-09 2022-10-06 University Of Pittsburgh - Of The Commonwealth System Of Higher Education Methods of restoring lysosomal function of retinal pigment epithelial cells by activation of tfeb

Also Published As

Publication number Publication date
WO2023192463A3 (en) 2023-12-21
MX2024011963A (es) 2024-11-08
WO2023192463A2 (en) 2023-10-05
TW202405180A (zh) 2024-02-01
KR20250006360A (ko) 2025-01-10
CN119213136A (zh) 2024-12-27
EP4499848A4 (en) 2026-03-25
AU2023243048A1 (en) 2024-10-03
US20250327092A1 (en) 2025-10-23
IL316014A (en) 2024-11-01
JP2025510975A (ja) 2025-04-15
EP4499848A2 (en) 2025-02-05

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