(SLE)
AVALOTKAGEN ONTAPARVOVEK(INNC)
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/8 (RAAV8) VECTOR, ENCODING CODON-OPTIMIZED, WILD-TYPE HUMAN ORNITHINE TRANSCARBAMYLASE (OTC) UNDER THE CONTROL OF A HYBRID HUMAN THYROXINE-BINDING GLOBULIN (TBG) / HUMAN ALPHA 1- MICROGLOBULIN/BIKUNIN PRECURSOR (AMBP) PROMOTER/ENHANCER. THE VECTOR GENOME IS A HEAD-TO-HEAD DIMER, WITH THE VECTOR GENOME CASSETTE LOCATED 5' OF THE MUTATED INTERNAL INVERTED TERMINAL REPEAT (ITR) IN A REVERSE ORIENTATION AND THE VECTOR GENOME CASSETTE LOCATED 3' OF THE MUTATED INTERNAL ITR IN A FORWARD ORIENTATION; REC. INN LIST 84(WHO)
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/8 (RAAV REP2-CAP8) VECTOR, ENCODING CODONOPTIMIZED, WILD-TYPE HUMAN ORNITHINE TRANSCARBAMYLASE (OTC) UNDER THE CONTROL OF A HYBRID HUMAN THYROXINE-BINDING GLOBULIN (TBG) / HUMAN ALPHA 1-MICROGLOBULIN/BIKUNIN PRECURSOR (AMBP) PROMOTER/ENHANCER. THE VECTOR GENOME IS A HEAD-TOHEAD DIMER, WITH THE VECTOR GENOME CASSETTE LOCATED 5' OF THE MUTATED INTERNAL INVERTED TERMINAL REPEAT (ITR) IN A REVERSE ORIENTATION AND THE VECTOR GENOME CASSETTE LOCATED 3' OF THE MUTATED INTERNAL ITR IN A FORWARD ORIENTATION. GENE THERAPY (ORNITHINE TRANSCARBAMYLASE (OTC) DEFICIENCY)(WHOX)
AVALOTCAGENUM ONTAPARVOVECUM(INNM)