EGT-201 is an adeno-associated viral vector serotype 9 (AAV9) vector-based gene therapy consisting of the alpha-N-acetyglucosaminidase (NAGLU) transgene. Developed by Esteve Pharmaceuticals, it is being investigated for Sanfilippo syndrome type B.[L45939]

FLT201 is a recombinant adeno-associated viral vector serotype S3 containing codon optimised expression cassette encoding human beta (?)-glucocerebrosidase variant 85 (GCasevar85)

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