BioCentury
ARTICLE | Product Development

Sailing on in Sanfilippo

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

September 28, 2015 7:00 AM UTC

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...