ETV:NAGLU

**ETV:NAGLU** is an investigational brain-penetrant enzyme replacement therapy being developed by Denali for mucopolysaccharidosis type IIIB, also known as Sanfilippo syndrome type B. It is designed as a TransportVehicle-enabled recombinant alpha-N-acetylglucosaminidase program intended to address the underlying lysosomal enzyme deficiency caused by loss of NAGLU activity, thereby reducing pathological heparan sulfate accumulation. Based on Denali's ETV platform strategy, the program is intended to improve central nervous system delivery relative to conventional enzyme replacement approaches by leveraging receptor-mediated transport across the blood-brain barrier. Publicly available information indicates the program has been disclosed by Denali as part of its enzyme transport vehicle pipeline for MPS IIIB and appears to remain at the preclinical stage.