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1. WO2020198675 - GENERATION OF CHIMERIC ANTIGEN RECEPTOR (CAR)-PRIMARY NK CELLS FOR CANCER IMMUNOTHERAPY USING A COMBINATION OF CAS9/RNP AND AAV VIRUSES

Publication Number WO/2020/198675
Publication Date 01.10.2020
International Application No. PCT/US2020/025454
International Filing Date 27.03.2020
IPC
A61K 38/46 2006.01
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
38Medicinal preparations containing peptides
16Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
43Enzymes; Proenzymes; Derivatives thereof
46Hydrolases (3)
C12N 5/10 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
C12N 9/16 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
C12N 9/22 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
9Enzymes, e.g. ligases (6.); Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating, or purifying enzymes
14Hydrolases (3.)
16acting on ester bonds (3.1)
22Ribonucleases
C12N 15/11 2006.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
C12N 15/113 2010.01
CCHEMISTRY; METALLURGY
12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09Recombinant DNA-technology
11DNA or RNA fragments; Modified forms thereof
113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
Applicants
  • RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL [US]/[US]
Inventors
  • LEE, Dean, Anthony
  • MEYER, Kathrin, Christine
  • KARAROUDI, Meisam, Naeimi
  • LIKHITE, Shibi
Agents
  • LANIER, J., Gibson
  • CURFMAN, Christopher, L.
Priority Data
62/825,00727.03.2019US
Publication Language English (EN)
Filing Language English (EN)
Designated States
Title
(EN) GENERATION OF CHIMERIC ANTIGEN RECEPTOR (CAR)-PRIMARY NK CELLS FOR CANCER IMMUNOTHERAPY USING A COMBINATION OF CAS9/RNP AND AAV VIRUSES
(FR) GÉNÉRATION DE CELLULES NK PRIMAIRES DE RÉCEPTEUR D'ANTIGÈNE CHIMÈRE (CAR) POUR UNE IMMUNOTHÉRAPIE ANTICANCÉREUSE À L'AIDE D'UNE COMBINAISON DE VIRUS CAS9/RNP ET AAV
Abstract
(EN)
Disclosed are methods for genetically engineering cells using Adeno-associated viral (AAV) delivery of a CRISPR/CAS9 system and ribonucleoproteins for integration. In some aspects, disclosed herein are AAV plasmids for performing said methods
(FR)
La présente invention concerne des procédés pour l'ingénierie génétique de cellules à l'aide de l'administration d'un virus adéno-associé (AAV pour Adeno-Associated Viral) d'un système CRISPR/CAS9 et de ribonucléoprotéines pour une intégration. Selon certains aspects, la présente invention concerne des plasmides de virus AAV permettant de mettre en œuvre lesdits procédés.
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