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NIMATPAGEN PARIPARVOVEC
NIMATPAGÉN PARIPARVOVEC
RECOMBINANT, NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 3B (RAAV3B) VECTOR, ENCODING A SHORTENED VERSION OF THE HUMAN COPPER-TRANSPORTING ATPASE 2 (ATP7B) (ALSO KNOWN AS ATPASE COPPER-TRANSPORTING BETA, WILSON DISEASE-ASSOCIATED PROTEIN) WHICH IS DEVOID OF THE FIRST FOUR OF THE SIX METAL BINDING SITES (MBS) (MINI-ATP7B), UNDER CONTROL OF A LIVER SPECIFIC HUMAN ALPHA-1 ANTITRYPSIN (AAT) PROMOTER AND A SYNTHETIC POLYADENYLATION SEQUENCE DERIVED FROM THE POLYADENYLATION SIGNAL OF THE RABBIT BETA-GLOBIN GENE, AND FLANKED BY ADENO-ASSOCIATED VIRUS 2 (AAV2) INVERTED TERMINAL REPEATS (ITRS)