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EGT-101 is an adeno-associated viral vector serotype 9 (AAV9) vector-based gene therapy consisting of the human sulfamidase (SGSH) transgene. Developed by Esteve Pharmaceuticals, it is being investigated for Sanfilippo syndrome type A.
EGT-101 is an adeno-associated viral vector serotype 9 (AAV9) vector-based gene therapy consisting of the human sulfamidase (SGSH) transgene. Developed by Esteve Pharmaceuticals, it is being investigated for Sanfilippo syndrome type A.
