MOTACABTAGENE LUREVGEDLEUCEL

UNII:	7CH7MR6SN5
Type:	INGREDIENT SUBSTANCE
Formula:
Preferred Substance Name:	MOTACABTAGENE LUREVGEDLEUCEL
GSRS Full Record:	7CH7MR6SN5 i

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 .BETA.2 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 CD3.ZETA. SIGNALLING DOMAIN. EXPRESSION OF THE CAR IS DRIVEN BY THE ELONGATION FACTOR, EF-1.ALPHA., PROMOTER AND IS TERMINATED BY A SYNTHETIC POLY A SEQUENCE. THE BCMA EXPRESSION CASSETTE IS FLANKED BY TWO TRAC HOMOLOGY ARMS GUIDING THE EXPRESSION CASSETTE TO THE TRAC LOCUS. THE T CELLS ARE CULTURED IN THE PRESENCE OF GROWTH MEDIUM CONTAINING INTERLEUKIN 2 (IL-2) AND 7 (IL-7) AND ARE 30% CAR+ T CELLS, 0.4% TCR+ AND 30% B2M+
Motacabtagene Lurevgedleucel
motacabtagene lurevgedleucel [INN]

Note

UNIIs are generated based on scientific identity characteristics using ISO 11238 data elements. UNII availability does not imply any regulatory review or approval. Synonyms and mappings are based on the best public information available at the time of publication. Please report any problems/errors associated with this data to FDA-SRS@fda.hhs.gov.

MOTACABTAGENE LUREVGEDLEUCEL

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