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DIRLOCTOCOGEN SAMOPARVOVEC
DIRLOCTOCOGÉNE SAMOPARVOVEC
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS VECTOR WITH A BIO-ENGINEERED CAPSID DERIVED BY DNA SHUFFLING OF 10 AAV CAPSID SEQUENCES (AAV1, 2, 3B, 4, 5, 6 8, 9, AVIAN AND BOVINE AAV) FOLLOWED BY IN VIVO SELECTION IN FAH-/-/RAG2-/-/IL2RG-/- (FRG) MICE PARTIALLY REPOPULATED WITH HUMAN HEPATOCYTES. THE VECTOR HAS AAV2 GENOMIC INVERTED TERMINAL REPEATS (ITR) AND ENCODES A HUMAN B-DOMAIN-DELETED BLOOD COAGULATION FACTOR VIII (HF8, FVIII) UNDER THE CONTROL OF A MODIFIED TRANSTHYRETIN (TTRM) PROMOTER. NOTE: THE NUCLEOTIDE SEQUENCE OF THE VECTOR IS A MODIFICATION OF THE ONE OF THE VECTOR OF ROVOCTOCOGENE DURPARVOVEC (PROP. INN LIST 120, REC. INN LIST 82); REC. INN LIST 83