(INNS)
ARVENAKOGEN SANPARVOVEK(INNC)
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE 5 (AAV5) VECTOR ENCODING A CODON-OPTIMIZED GAIN OF FUNCTION VARIANT (PADUA VARIANT, R338L) OF HUMAN COAGULATION FACTOR IX (FIX), UNDER THE CONTROL OF A MURINE LIVER-SPECIFIC TRANSTHYRETIN (TTR) PROMOTER AND HUMAN GROWTH HORMONE POLYADENYLATION SIGNAL, FLANKED BY AAV2 INVERTED TERMINAL REPEATS. A HUMAN BETA-GLOBIN INTRON HAS BEEN INSERTED BETWEEN THE PROMOTER AND THE FIX CODING REGION. THE VIRUS VECTOR ALSO CONTAINS MUTATIONS IN THE VIRAL AAV5 CAPSID PROTEIN VP1 (S2A AND T711S)(WHO)
ARVENACOGENUM SANPARVOVECUM(INN)