07:00 , Sep 28, 2015 |  BioCentury  |  Product Development

Sailing on in Sanfilippo

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...
03:01 , Oct 28, 2014 |  BC Extra  |  Clinical News

One-year follow up for uniQure's liver gene therapy

uniQure N.V. (NASDAQ:QURE) reported top-line one-year follow-up data from a completed Phase I study in eight acute intermittent porphyria (AIP) patients receiving varying doses of the company's AMT-021, a recombinant adeno-associated viral (AAV) vector containing...
07:00 , Jul 21, 2014 |  BC Week In Review  |  Clinical News

AMT-021: Interim Phase I data

Interim data from an open-label, dose-ranging, Spanish Phase I trial in 8 patients with severe acute intermittent porphyria showed that a single IV injection of up to 2x10 13 genetic components/kg AMT-021 was well tolerated...
07:00 , Jun 5, 2014 |  BC Innovations  |  Cover Story

Alas, porphyria

For over two decades, treatment of acute porphyrias has relied on hemin infusions that restore heme levels, but the therapy is slow to work, has side effects and is expensive. Now, a team led by...
08:00 , Dec 17, 2012 |  BC Week In Review  |  Clinical News

AMT-021: Phase I started

uniQure began an open-label Spanish Phase I trial to evaluate a single IV injection of AMT-021 in 8 patients with severe acute intermittent porphyria. The trial is being conducted under the AIPGENE consortium, which aims...
08:00 , Feb 20, 2012 |  BC Week In Review  |  Company News

Amsterdam Molecular, uniQure deal

Amsterdam Molecular's board unanimously recommended dissolving the company's assets into newco uniQure in a stock deal. Amsterdam said it is facing a "precarious" financial position with cash expected to last until April and the deal...
01:40 , Feb 18, 2012 |  BC Extra  |  Company News

Amsterdam Molecular to transfer assets, delist

Amsterdam Molecular Therapeutics Holding N.V. (Euronext:AMT) said its board unanimously recommended dissolving the company's assets into newly formed uniQure B.V. (Amsterdam, the Netherlands) in a stock deal. Amsterdam said it is facing a "precarious" financial...
08:00 , Jan 2, 2012 |  BioCentury  |  Finance

Small-fry squashed

Among last year's best-performing large cap biotechs were an acquiree and an acquirer: HCV play Pharmasset Inc. and Valeant Pharmaceuticals International Inc. Other big cap stocks also did well overall, while investors abandoned their small-...
07:00 , Oct 31, 2011 |  BC Week In Review  |  Company News

Amsterdam Molecular neurology, hematology, endocrine/metabolic news

Amsterdam Molecular discontinued development of Glybera after CHMP's second recommendation against approval of the product in the EU for lipoprotein lipase (LPL) deficiency. The company will also reduce headcount by 50% to 45...
00:39 , Oct 26, 2011 |  BC Extra  |  Company News

Amsterdam Molecular discontinues Glybera

Amsterdam Molecular Therapeutics Holding N.V. (Euronext:AMT) discontinued development of Glybera following last week's second CHMP decision against approval of the product in the EU for lipoprotein lipase (LPL) deficiency. The company will also reduce headcount...