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DEVAFIDUGEN CIVAPARVOVEC
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/6 (RAAV REP2-CAP6) VECTOR, WHICH CONTAINS A PROMOTER-LESS HUMAN ALPHA-L-IDURONIDASE (HIDUA) TRANSGENE CASSETTE, ENCODING PARTIAL EXON 1 AND FULL EXONS 2-14 WITH THE FIRST 28 AMINO ACIDS REMOVED, 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
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/6 (RAAV6) VECTOR, WHICH CONTAINS A PROMOTER-LESS HUMAN ALPHA-L-IDURONIDASE (HIDUA) TRANSGENE CASSETTE, ENCODING PARTIAL EXON 1 AND FULL EXONS 2-14 WITH THE FIRST 28 AMINO ACIDS REMOVED, AND SPLICE ACCEPTOR SITE SEQUENCE (SA) FROM HIDUA EXON 2, FLANKED BY A SEQUENCE HOMOLOGOUS TO THE ZINCFINGER NUCLEASE (ZFN) CLEAVAGE SITE OF THE HUMAN ALBUMIN (HALB) INTRON 1; REC. INN LIST 84