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LARUPARETIGÉN ZOVAPARVOVEC
LARUPARÉTIGENE ZOVAPARVOVEC
RECOMBINANT, NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 2 (AAV2) VECTOR, IN WHICH THE CAPSID CONTAINS THREE TYROSINE (Y) TO PHENYLALANINE (F) MUTATIONS (AAV2TYF), ENCODING CODON-OPTIMISED HUMAN RETINITIS PIGMENTOSA GTPASE REGULATOR (RPGR) ISOFORM 6 (RPGR-ORF15), UNDER CONTROL OF THE HUMAN G PROTEINCOUPLED RECEPTOR KINASE 1 PROMOTER, PRECEDED BY A SYNTHETIC SPLICE DONOR/SPLICE ACCEPTOR, TERMINATED WITH THE SIMIAN VIRUS 40 (SV40) POLYADENYLATION (POLYA) SEQUENCE, AND FLANKED BY AAV2 INVERTED TERMINAL REPEATS (ITRS)