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OLENASUFLIGEN RELDUPARVOVEC
OLÉNASUFLIGENE RELDUPARVOVEC
RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS SEROTYPE RH.10 (AAVRH.10) VECTOR, COMPRISED OF A RECOMBINANT GENOME WITH THE AAV2 INVERTED TERMINAL REPEATS (ITR) PACKAGED INTO AN AAVRH.10 CAPSID, EXPRESSING THE HUMAN N-SULFOGLUCOSAMINE SULFOHYDROLASE (HSGSH) CDNA, UNDER THE CONTROL OF THE CMV IMMEDIATE EARLY ENHANCER/CHICKEN BETA ACTIN (CAG) PROMOTER AND A BOVINE HGH POLYA TRANSCRIPTION TERMINATION SITE; PROPOSED INN: LIST 119, PROPOSED LIST 124; REC. LIST 85