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POZETALDOGEN ORMESPARVOVEC
POZETALDOGÉN ORMESPARVOVEC
POZÉTALDOGENE ORMESPARVOVEC
RECOMBINANT, NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 9 (RAAV9) VECTOR ENCODING HUMAN ADENOSINE TRIPHOSPHATE (ATP)-BINDING CASSETTE SUB-FAMILY D MEMBER 1 (ABCD1, ALSO KNOWN AS ADRENOLEUKODYSTROPHY (ALD) PROTEIN) UNDER CONTROL OF A HUMAN CYTOMEGALOVIRUS (CMV) ENHANCER/CHICKEN BETA-ACTIN (CBA) PROMOTER PLUS A BETA-ACTIN EXON, A CHIMERIC CHICKEN BETA-ACTIN/RABBIT BETA-GLOBIN INTRON, AND A RABBIT BETA-GLOBIN EXON 3, TERMINATED WITH SIMIAN VIRUS 40 (SV40) AND BOVINE GROWTH HORMONE (BGH) POLYADENYLATION SIGNALS, AND FLANKED BY ADENO-ASSOCIATED VIRUS 2 (AAV2) INVERTED TERMINAL REPEATS (ITRS)