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MOTACABTAGÉN LUREVGEDLEUCEL
MOTACABTAGENE LURÉVGEDLEUCEL
ALLOGENEIC T CELLS OBTAINED FROM PERIPHERAL BLOOD BY LEUKAPHERESIS, GENETICALLY MODIFIED BY CRISPR/CAS9 (CLUSTERED REGULARLY INTERSPACED SHORT PALINDROMIC REPEATS/CRISPR-ASSOCIATED PROTEIN 9) MEDIATED GENE EDITING CONSISTING OF TWO GUIDE RNAS (GRNAS) INTRODUCED TRANSIENTLY AS CAS9-GRNA RIBONUCLEOPROTEIN (RNP) COMPLEX FOR THE TARGETED DISRUPTION OF THE T CELL RECEPTOR ALPHA CHAIN CONSTANT (TRAC) AND BETA2 MICROGLOBULIN (B2M) LOCI AND THE INSERTION OF AN ANTI-B CELL MATURATION ANTIGEN (BCMA) CAR TRANSGENE INTO THE TRAC LOCUS VIA AN ADENO-ASSOCIATED VIRUS SEROTYPE 6 (AAV6) VECTOR. THE CAR IS COMPOSED OF A HUMANIZED SINGLE-CHAIN VARIABLE FRAGMENT (SCFV) TARGETING BCMA, FOLLOWED BY A CD8 HINGE AND TRANSMEMBRANE REGION, A 4-1BB (CD137) COSTIMULATORY DOMAIN AND A CD3ZETA SIGNALLING DOMAIN...; PROPOSED INN: LIST 125, RECOMMENDED INN: LIST 87