EGT-201

EGT-201 is an investigational AAV9-based gene therapy being developed for the treatment of Sanfilippo B syndrome (mucopolysaccharidosis IIIB), a lysosomal storage disorder caused by deficiency of the enzyme alpha-N-acetylglucosaminidase (NAGLU). It consists of an adeno-associated virus serotype 9 (AAV9) vector encoding the human NAGLU transgene, designed to restore NAGLU activity in affected patients and thereby reduce pathological accumulation of heparan sulfate and related substrates in the central nervous system and other tissues.[3][7][9][11] EGT-201 was initially developed by researchers at the Universitat Autònoma de Barcelona in collaboration with Esteve and has received orphan drug designation from both the FDA and EMA, with development currently at the preclinical stage.[1][3][7][9][11]