(SLE)
ADLINAKOGEN CIVAPARVOVEK(INNC)
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/6 (RAAV6) VECTOR, WHICH CONTAINS A PROMOTER-LESS HUMAN COAGULATION FACTOR IX (HF9, FACTOR IX OR FIX) TRANSGENE CASSETTE, ENCODING EXONS 2-8 AND SPLICE ACCEPTOR SITE SEQUENCE (SA) FROM HF9 EXON 2, FLANKED BY A SEQUENCE HOMOLOGOUS TO THE ZINC-FINGER NUCLEASE (ZFN) CLEAVAGE SITE OF THE HUMAN ALBUMIN (HALB) INTRON 1; REC. INN LIST 84(WHO)
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/6 (RAAV REP2-CAP6) VECTOR, WHICH CONTAINS A PROMOTERLESS HUMAN COAGULATION FACTOR IX (HF9, FACTOR IX OR FIX) TRANSGENE CASSETTE, ENCODING EXONS 2-8 AND SPLICE ACCEPTOR SITE SEQUENCE (SA) FROM HF9 EXON 2, FLANKED BY A SEQUENCE HOMOLOGOUS TO THE ZINC-FINGER NUCLEASE (ZFN) CLEAVAGE SITE OF THE HUMAN ALBUMIN (HALB) INTRON 1(WHOX)
ADLINACOGENUM CIVAPARVOVECUM(INN)