Monday, August 08, 2016 10:59:48 AM
QURE...gene-therapy...Aug. 4th...Upgrade to buy...Target $21 H.C. Wainwright
...add this to the speculation list when it gets oversold...somewhere between $6 and $7...
UniQure NV (Nasdaq: QURE) is the firm that formulated Glybera, the first gene-therapy drug on the market. It’s a treatment for lipoprotein lipase deficiency (LPLD), a rare genetic disorder that causes inflammation of the pancreas – pancreatitis.
Because that disease affects just 1 million people around the world, Glybera is quite pricey — about $1 million per year.
But we’re just getting started here – and researchers are working on gene therapies that will have much lower prices because they will serve much larger patient groups.
UniQure itself has a series of other gene therapies in clinical trials, such as one for hemophilia, a genetic condition that afflicts 400,000 people worldwide. Other targets include Parkinson’s disease; acute intermittent porphyria, which causes severe pain and mental symptoms; and Sanfilippo syndrome, an inherited metabolic condition that causes sufferers to have a hard time processing sugar.
As much as I like uniQure’s science and its product pipeline, it’s a loss-ridden, clinical-stage biotech firm that’s just too volatile for retail investors.
QURE
...add this to the speculation list when it gets oversold...somewhere between $6 and $7...
UniQure NV (Nasdaq: QURE) is the firm that formulated Glybera, the first gene-therapy drug on the market. It’s a treatment for lipoprotein lipase deficiency (LPLD), a rare genetic disorder that causes inflammation of the pancreas – pancreatitis.
Because that disease affects just 1 million people around the world, Glybera is quite pricey — about $1 million per year.
But we’re just getting started here – and researchers are working on gene therapies that will have much lower prices because they will serve much larger patient groups.
UniQure itself has a series of other gene therapies in clinical trials, such as one for hemophilia, a genetic condition that afflicts 400,000 people worldwide. Other targets include Parkinson’s disease; acute intermittent porphyria, which causes severe pain and mental symptoms; and Sanfilippo syndrome, an inherited metabolic condition that causes sufferers to have a hard time processing sugar.
As much as I like uniQure’s science and its product pipeline, it’s a loss-ridden, clinical-stage biotech firm that’s just too volatile for retail investors.
QURE
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