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Sep 28, 2015
 |  BioCentury  |  Product Development

Sailing on in Sanfilippo

What positive data for AMT-110 in Sanfilippo B patients means for uniQure

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 also lose motor skills and can experience seizures.

In a Phase I/II trial in four Sanfilippo type B patients, AMT-110 increased levels of active NAGLU measured in the CSF from 0% at baseline to up to 14-17% of normal at three months. This elevation was maintained at 12 months. AMT-110 was safe and well tolerated, with no significant adverse events.

According to principal investigator Marc Tardieu, NAGLU activity of 5-10% of normal is associated with longer lifespans and higher quality of life in Sanfilippo B patients. Tardieu, a pediatric neurologist and professor at the University of Paris-Sud and University Hospital of Bicétre, presented the data on Sept. 19 at the European...

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