CO2023015532A2 - Composiciones de vectores virales y métodos de uso de estas - Google Patents

Composiciones de vectores virales y métodos de uso de estas

Info

Publication number
CO2023015532A2
CO2023015532A2 CONC2023/0015532A CO2023015532A CO2023015532A2 CO 2023015532 A2 CO2023015532 A2 CO 2023015532A2 CO 2023015532 A CO2023015532 A CO 2023015532A CO 2023015532 A2 CO2023015532 A2 CO 2023015532A2
Authority
CO
Colombia
Prior art keywords
compositions
methods
homology arms
viral vectors
present disclosure
Prior art date
Application number
CONC2023/0015532A
Other languages
English (en)
Inventor
Qiang Xiong
B Nelson Chau
Original Assignee
Logicbio Therapeutics Inc
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 Logicbio Therapeutics Inc filed Critical Logicbio Therapeutics Inc
Publication of CO2023015532A2 publication Critical patent/CO2023015532A2/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
    • C12N15/87—Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/90—Stable introduction of foreign DNA into chromosome
    • C12N15/902—Stable introduction of foreign DNA into chromosome using homologous recombination
    • C12N15/907—Stable introduction of foreign DNA into chromosome using homologous recombination in mammalian cells
    • 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
    • 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
    • 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)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • Biotechnology (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Biomedical Technology (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • General Engineering & Computer Science (AREA)
  • Wood Science & Technology (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Medicinal Chemistry (AREA)
  • Cell Biology (AREA)
  • Virology (AREA)
  • Mycology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Peptides Or Proteins (AREA)

Abstract

En la presente se proporcionan composiciones y métodos para una edición génica mejorada con construcciones de vectores AAV. Entre otras cosas, la presente descripción reconoce que los brazos de homología de determinada longitud (por ejemplo, al menos 750 nt o al menos 1000 nt cada uno) pueden presentar una actividad de edición mejorada. En algunas modalidades, la presente descripción reconoce que los brazos de homología de determinada longitud (por ejemplo, al menos 750 nt o al menos 1000 nt cada uno) pueden presentar mejoras adicionales en la actividad de edición cuando los brazos de homología son de diferente longitud.
CONC2023/0015532A 2021-04-30 2023-11-17 Composiciones de vectores virales y métodos de uso de estas CO2023015532A2 (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US202163182738P 2021-04-30 2021-04-30
PCT/US2022/026988 WO2022232545A1 (en) 2021-04-30 2022-04-29 Viral vector compositions and methods of use thereof

Publications (1)

Publication Number Publication Date
CO2023015532A2 true CO2023015532A2 (es) 2023-11-30

Family

ID=83847345

Family Applications (1)

Application Number Title Priority Date Filing Date
CONC2023/0015532A CO2023015532A2 (es) 2021-04-30 2023-11-17 Composiciones de vectores virales y métodos de uso de estas

Country Status (11)

Country Link
EP (1) EP4330417A1 (es)
JP (1) JP2024517743A (es)
KR (1) KR20240004566A (es)
CN (1) CN117321215A (es)
AU (1) AU2022264585A1 (es)
BR (1) BR112023022657A2 (es)
CA (1) CA3216909A1 (es)
CO (1) CO2023015532A2 (es)
MX (1) MX2023012884A (es)
TW (1) TW202309277A (es)
WO (1) WO2022232545A1 (es)

Families Citing this family (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2022182986A1 (en) * 2021-02-26 2022-09-01 Logicbio Therapeutics, Inc. Manufacturing and use of recombinant aav vectors

Family Cites Families (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2341350A1 (en) * 1998-05-15 1999-11-25 Sri International Transgenic animals produced by homologous sequence targeting
KR20160135729A (ko) * 2014-03-21 2016-11-28 더 보드 어브 트러스티스 어브 더 리랜드 스탠포드 주니어 유니버시티 뉴클레아제 없는 게놈 편집
SG11202009446TA (en) * 2018-04-05 2020-10-29 Juno Therapeutics Inc T cells expressing a recombinant receptor, related polynucleotides and methods
KR20210049833A (ko) * 2018-08-10 2021-05-06 로직바이오 테라퓨틱스, 인크. Mma의 치료를 위한 비-붕괴적 유전자 요법

Also Published As

Publication number Publication date
BR112023022657A2 (pt) 2024-01-16
AU2022264585A1 (en) 2023-11-30
CA3216909A1 (en) 2022-11-03
KR20240004566A (ko) 2024-01-11
EP4330417A1 (en) 2024-03-06
CN117321215A (zh) 2023-12-29
WO2022232545A1 (en) 2022-11-03
TW202309277A (zh) 2023-03-01
JP2024517743A (ja) 2024-04-23
MX2023012884A (es) 2023-11-24

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