This week, Ionis Pharmaceuticals issued a press release stating that the Phase 3 trial of the huntingtin-lowering therapy formerly known as IONIS-HTTRx has formally begun. This trial is in the hands of a larger company called Roche/Genentech, and the drug is now called RG6042. Roche/Genentech issued a letter to the global HD community which we have shared via HDSA’s website and social media. In addition to the trial updates this letter provides an overview of the ongoing HD studies and where to learn more about them. We’re excited that this study is moving forward and look forward to a smooth recruitment.


HD Buzz Story on uniQure Announcement & Gene Therapy

Last week we got news that a Boston-based company called uniQure received FDA clearance to move forward with a small clinical trial of its gene therapy drug for HD. HD Buzz published an article this week with a deeper dive into the science behind this drug and what we can expect moving forward. They do an excellent job describing gene therapy – and it’s a great thing to read up on, because there will be more of that coming down the pipeline for HD in the near future.