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1. WO2022136551 - REPROGRAMMING IMMUNE CELLS BY TARGETED INTEGRATION OF ZETA-DEFICIENT CHIMERIC ANTIGEN RECEPTOR TRANSGENES

Publication Number WO/2022/136551
Publication Date 30.06.2022
International Application No. PCT/EP2021/087306
International Filing Date 22.12.2021
IPC
C07K 14/725 2006.1
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
725T-cell receptors
C12N 9/22 2006.1
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
A61K 35/17 2015.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
KPREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
35Medicinal preparations containing materials or reaction products thereof with undetermined constitution
12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
14Blood; Artificial blood
17Lymphocytes; B-cells; T-cells; Natural killer cells; Interferon-activated or cytokine-activated lymphocytes
A61P 35/00 2006.1
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35Antineoplastic agents
C12N 5/0783 2010.1
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
07Animal cells or tissues
071Vertebrate cells or tissues, e.g. human cells or tissues
078Cells from blood or from the immune system
0783T cells; NK cells; Progenitors of T or NK cells
CPC
A61P 35/00
AHUMAN NECESSITIES
61MEDICAL OR VETERINARY SCIENCE; HYGIENE
PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
35Antineoplastic agents
C07K 14/7051
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
14Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
435from animals; from humans
705Receptors; Cell surface antigens; Cell surface determinants
70503Immunoglobulin superfamily
7051T-cell receptor (TcR)-CD3 complex
C07K 2317/622
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2317Immunoglobulins specific features
60characterized by non-natural combinations of immunoglobulin fragments
62comprising only variable region components
622Single chain antibody (scFv)
C07K 2319/00
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
C07K 2319/03
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
01containing a localisation/targetting motif
03containing a transmembrane segment
C07K 2319/33
CCHEMISTRY; METALLURGY
07ORGANIC CHEMISTRY
KPEPTIDES
2319Fusion polypeptide
33fusions for targeting to specific cell types, e.g. tissue specific targeting, targeting of a bacterial subspecies
Applicants
  • CHARITÉ - UNIVERSITÄTSMEDIZIN BERLIN [DE]/[DE]
Inventors
  • KATH, Jonas Christian
  • REINKE, Petra
  • SCHMÜCK-HENNERESSE, Michael
  • VOLK, Hans-Dieter
  • WAGNER, Dimitrios Laurin
Agents
  • HERTIN UND PARTNER RECHTS- UND PATENTANWÄLTE
Priority Data
20216649.222.12.2020EP
Publication Language English (en)
Filing Language English (EN)
Designated States
Title
(EN) REPROGRAMMING IMMUNE CELLS BY TARGETED INTEGRATION OF ZETA-DEFICIENT CHIMERIC ANTIGEN RECEPTOR TRANSGENES
(FR) REPROGRAMMATION DE CELLULES IMMUNITAIRES PAR INTÉGRATION CIBLÉE DE TRANSGÈNES DE RÉCEPTEURS ANTIGÉNIQUES CHIMÉRIQUES DÉFICIENTS EN ZÊTA
Abstract
(EN) The invention relates to a nucleic acid construct for targeting and integrating a CD3 zeta-deficient chimeric antigen receptor (CAR) fragment into an endogenous CD3 zeta/CD247 gene of a host genome. The invention further relates to a genetically modified human cell expressing an exogenous nucleic acid sequence encoding a CD3 zeta deficient CAR fragment, integrated in- frame into the endogenous CD3 zeta/CD247 gene for gene fusion, to form a functional CAR comprising an exogenous CAR fragment fused with an endogenous CD3 zeta domain.
(FR) L'invention concerne une construction d'acide nucléique pour le ciblage et l'intégration d'un fragment de récepteur antigénique chimérique (CAR) déficient en CD3 zêta dans un gène CD3 zêta/CD247 endogène d'un génome hôte. L'invention concerne en outre une cellule humaine génétiquement modifiée exprimant une séquence d'acide nucléique exogène codant pour un fragment CAR déficient en CD3 zêta, intégré dans la structure dans le gène CD3 zêta/CD247 endogène pour fusion génique, afin de former un CAR fonctionnel comprenant un fragment CAR exogène fusionné avec un domaine zêta CD3 endogène.
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