MX2024011963A - Vectores de virus adenoasociados para la entrega de acido nucleico a las celulas retinianas - Google Patents

Vectores de virus adenoasociados para la entrega de acido nucleico a las celulas retinianas

Info

Publication number
MX2024011963A
MX2024011963A MX2024011963A MX2024011963A MX2024011963A MX 2024011963 A MX2024011963 A MX 2024011963A MX 2024011963 A MX2024011963 A MX 2024011963A MX 2024011963 A MX2024011963 A MX 2024011963A MX 2024011963 A MX2024011963 A MX 2024011963A
Authority
MX
Mexico
Prior art keywords
nucleic acid
vectors
adeno
delivery
associated virus
Prior art date
Application number
MX2024011963A
Other languages
English (en)
Spanish (es)
Inventor
Byrne Leah Caroline Thomas
Molly E Johnson
William Richard Stauffer
Bilge Esin Ozturk
Original Assignee
Univ Pittsburgh Commonwealth Sys Higher Education
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 Univ Pittsburgh Commonwealth Sys Higher Education filed Critical Univ Pittsburgh Commonwealth Sys Higher Education
Publication of MX2024011963A publication Critical patent/MX2024011963A/es

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)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
MX2024011963A 2022-03-30 2024-09-27 Vectores de virus adenoasociados para la entrega de acido nucleico a las celulas retinianas MX2024011963A (es)

Applications Claiming Priority (14)

Application Number Priority Date Filing Date Title
US202263325550P 2022-03-30 2022-03-30
US202263325540P 2022-03-30 2022-03-30
US202263325543P 2022-03-30 2022-03-30
US202263325559P 2022-03-30 2022-03-30
US202263325553P 2022-03-30 2022-03-30
US202263325551P 2022-03-30 2022-03-30
US202263325558P 2022-03-30 2022-03-30
US202263325542P 2022-03-30 2022-03-30
US202263325544P 2022-03-30 2022-03-30
US202263325555P 2022-03-30 2022-03-30
US202263325548P 2022-03-30 2022-03-30
US202263325562P 2022-03-30 2022-03-30
US202263325541P 2022-03-30 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
MX2024011963A true MX2024011963A (es) 2024-11-08

Family

ID=88203541

Family Applications (1)

Application Number Title Priority Date Filing Date
MX2024011963A MX2024011963A (es) 2022-03-30 2024-09-27 Vectores de virus adenoasociados para la entrega de acido nucleico a las celulas retinianas

Country Status (11)

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

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
CN107012171B (zh) * 2011-04-22 2021-07-13 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
CN118416088A (zh) * 2017-03-03 2024-08-02 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
EP3645052A4 (en) * 2017-06-30 2021-07-28 Regents of the University of California ADENO-ASSOCIATED VIRUS VIRIONS WITH ASSOCIATED CAPSIDE VARIANTS AND METHODS OF USE
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
MX2022002652A (es) * 2019-09-09 2022-04-06 Univ Pittsburgh Commonwealth Sys Higher Education Metodos para restaurar la funcion lisosomal de celulas epiteliales de pigmento retiniano mediante activacion de tfeb.

Also Published As

Publication number Publication date
EP4499848A2 (en) 2025-02-05
US20250327092A1 (en) 2025-10-23
KR20250006360A (ko) 2025-01-10
WO2023192463A2 (en) 2023-10-05
TW202405180A (zh) 2024-02-01
AU2023243048A1 (en) 2024-10-03
CN119213136A (zh) 2024-12-27
JP2025510975A (ja) 2025-04-15
IL316014A (en) 2024-11-01
CA3255255A1 (en) 2023-10-05
WO2023192463A3 (en) 2023-12-21
EP4499848A4 (en) 2026-03-25

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