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CROSIGALCOGEN OMLIXPARVOVEC
CROSIGALCOGÉN OMLIXPARVOVEC
RECOMBINANT, NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE HU68 (AAVHU68) VECTOR ENCODING CODONOPTIMISED HUMAN GALACTOSYLCERAMIDASE (GALC), UNDER CONTROL OF A CYTOMEGALOVIRUS IMMEDIATE EARLY ENHANCER/CHICKEN BETA-ACTIN PROMOTER (THE CB7 PROMOTER) PLUS A CHIMERIC INTRON, CONSISTING OF A CHICKEN BETA-ACTIN SPLICE DONOR AND A RABBIT BETA-GLOBIN SPLICE ACCEPTOR, TERMINATED WITH A RABBIT BETA-GLOBIN POLYADENYLATION SIGNAL, FLANKED BY ADENO-ASSOCIATED VIRUS 2 (AAV2) INVERTED TERMINAL REPEATS (ITRS).