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ARTICLE | Clinical News

uniQure to pursue pivotal trial of Sanfilippo B therapy

September 22, 2015 3:58 AM UTC

uniQure N.V. (NASDAQ:QURE) plans to advance AMT-110 into a pivotal trial after partner Institut Pasteur presented data from a four-patient Phase I/II trial of the gene therapy to treat Sanfilippo B. Results were presented at the European Society of Gene and Cell Therapy and Finnish Society of Gene Therapy Collaborative Congress on Sept. 19.

Patients received a single dose of AMT-110, an adeno-associated virus 5 (AAV5) vector carrying the N-acetylglucosaminidase alpha ( NAGLU) gene. uniQure said AMT-110 increased NAGLU activity in the cerebrospinal fluid to 14-17% of normal at three months from 0% at baseline in all four patients. NAGLU activity was maintained at 12 months. Principal investigator Marc Tardieu said in a statement that NAGLU activity "in the range of 5-10% of normal" is associated with longer lifespans and higher quality of life in Sanfilippo B patients. ...