uniQure N.V. says Phase I/II data on AMT-110 support both the start of a pivotal trial for Sanfilippo type B syndrome, and the company's plans to use its AAV5 vector in other rare lysosomal storage disorders with a CNS component.
AMT-110 uses an adeno-associated virus 5 (AAV5) vector to deliver the N-acetylglucosaminidase alpha (NAGLU) gene to the CNS via intracerebral injection.
Mutations in NAGLU cause Sanfilippo type B, also called mucopolysaccharidosis IIIB (MPS IIIB). Patients with MPS IIIB are unable to degrade heparan sulfate glycosaminoglycans (HSGAGs), which accumulate in the lysosome. The clinical manifestations of the disease are mainly neurological and include behavior and sleep disorders, communication problems and intellectual deterioration. As it progresses, patients