uniQure N.V. (UQ1)
XSTU 
Summary
UQ1 Chart
uniQure: The Market Graded The Wrong Exam
uniQure (QURE) is rated a Speculative Buy with a $47 price target, implying 115% upside. QURE's Huntington's gene therapy BLA is under FDA accelerated review, with a decision expected around November 1. Three-year data met both FDA endpoints, showing 75% slowing of composite and 60% slowing of Total Functional Capacity.
uniQure Plunges on Additional Data From Huntington's Disease Study
QURE's Huntingtons gene therapy shows sustained functional benefits at 36 months, but mixed 48-month data add uncertainty.
uniQure N.V. (QURE) Discusses Additional Data from Phase I/II AMT-130 Study in Huntington's Disease Highlighting Disease Progression Slowing Transcript
uniQure N.V. (QURE) Discusses Additional Data from Phase I/II AMT-130 Study in Huntington's Disease Highlighting Disease Progression Slowing Transcript
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uniQure N.V. Profile
| Biotechnology Industry | Healthcare Sector | Matthew C. Kapusta CEO | XSTU Exchange | NL0010696654 ISIN |
| NL Country | 221 Employees | - Last Dividend | 31 Jan 2014 Last Split | 20 Jun 2007 IPO Date |
Overview
uniQure N.V. is a pioneering company in the biotechnology sector, focused on the advancement of gene therapies for patients grappling with rare and severe illnesses. Since its inception in 1998, uniQure N.V. has been at the forefront of developing innovative treatments aimed at significantly improving the quality of life for patients around the globe. With its headquarters nestled in Amsterdam, the Netherlands, the company leverages cutting-edge science to tackle some of the most challenging medical conditions.
Products and Services
HEMGENIX: A groundbreaking treatment for hemophilia B, HEMGENIX has successfully completed its Phase III HOPE-B pivotal trial. This gene therapy offers a new lease of life for patients suffering from this rare bleeding disorder, potentially reducing the dependence on traditional hemophilia treatments.
AMT-130: A novel gene therapy approach for Huntington's disease, AMT-130 is currently under Phase I/II clinical study. This treatment represents hope for patients with Huntington's disease, aiming to significantly slow down or halt the progression of this devastating neurodegenerative disorder.
AMT-162: Targeting superoxide dismutase 1-amyotrophic lateral sclerosis (SOD1-ALS), AMT-162 is in preclinical trials. ALS, also known as Lou Gehrig's disease, is a condition that affects nerve cells in the brain and spinal cord, leading to loss of muscle control. AMT-162 is being developed to treat this specific form of ALS.
AMT-260: This candidate is in preclinical trials to address temporal lobe epilepsy, a condition that can cause severe and recurrent seizures. AMT-260 aims to provide a new therapeutic approach that could better manage or potentially cure this form of epilepsy.
AMT-191: In the preclinical trial stage, AMT-191 is geared towards the treatment of Fabry disease, a genetic disorder that impairs the body's ability to break down specific fatty substances. AMT-191 could offer a significant advancement in treating this disease.
AMT-161: Also in preclinical trials, AMT-161 is a therapy aimed at treating amyotrophic lateral sclerosis (ALS) caused by mutations. This gene therapy could provide a targeted approach to combat this devastating version of ALS.
AMT-240: Currently in preclinical trials, AMT-240 is being developed for autosomal dominant Alzheimer's disease. This innovative treatment could potentially slow or halt the progression of Alzheimer's in patients with a family history of the disease.
AMT-210: Dedicated to tackling Parkinson's disease, AMT-210 is in the preclinical trial phase. This therapy aims to offer a new disease-modifying treatment option for patients with Parkinson's, a neurodegenerative disorder characterized by the decline of motor function.
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