uniQure is delivering on the promise of gene therapy -- single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary and partnered gene therapies to treat patients with hemophilia, Huntingtonâs disease and other severe genetic diseases. We are advancing a focused pipeline of innovative gene therapies. We are currently conducting a pivotal phase 3 trial in our lead indication, hemophilia B, and have established preclinical proof-of-concept in Huntingtonâs disease. Our pipeline of adeno-associated virus (AAV)-based gene therapies has been developed using an innovative technology platform, supported by industry-leading proprietary commercial-grade manufacturing capabilities. Source
No articles found.
Catasys has contracted with leading national, regional and local health insurance ...
Catasys has contracted with leading national, r...
Acorda Therapeutics is a biotechnology company focused on developing therapies tha...
Acorda Therapeutics is a biotechnology company ...
At Biogen, our mission is clear: we are pioneers in neuroscience. Biogen discovers...
At Biogen, our mission is clear: we are pioneer...
Rubius Therapeutics developed our proprietary RED PLATFORMÂŽ to generate red blood...
Rubius Therapeutics developed our proprietary R...
Neurocrine Biosciences (Nasdaq: NBIX) is a neuroscience-focused, biopharmaceutical...
Neurocrine Biosciences (Nasdaq: NBIX) is a neur...
Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developi...
Eloxx Pharmaceuticals, Inc. is a clinical-stage...
bluebird bio is pioneering gene therapy with purpose. From our Cambridge, Mass., h...
bluebird bio is pioneering gene therapy with pu...
Adverum is a clinical-stage gene therapy company targeting unmet medical need in o...
Adverum is a clinical-stage gene therapy compan...
Join the National Investor Network and get the latest information with your interests in mind.