Ionis Pharmaceuticals, Inc. (IONS) announced today that Roche has exercised its option to license IONIS-HTTRx following the completion of a Phase 1/2a randomized, placebo-controlled, dose escalation study of IONIS-HTTRx in people with Huntington's disease (HD). Roche will now be responsible for all development and commercial activities. IONIS-HTTRx is the first therapy in clinical development designed to target the underlying cause of HD by reducing the production of the toxic mutant huntingtin protein (mHTT). In conjunction with the decision to license IONIS-HTTRx, Ionis earned a $45 million license fee from Roche.
A DNA double helix is seen in an undated artist's illustration released by the National Human Genome Research Institute to Reuters on May 15, 2012. REUTERS/National Human Genome Research Institute/Handout.
New research reveals how a dysfunction in the brain's so-called self-digestion system leads to cell death in neurodegenerative disorders. The newly discovered mechanism may lead to new therapies for conditions such as Parkinson's and Alzheimer's disease.
Dutch biotech UniQure has won orphan drug designation (ODD) for its investigational treatment for Huntington’s Disease, a rare, inherited neurodegenerative disorder that leads to loss of muscle coordination, behavioural abnormalities and cognitive decline.
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