Casgevy is the first CRISPR-based drug to make its way through the approval process, all but curing Sickle Cell Disease and it’s paving the way for similar drugs targeting other diseases. Is Huntington’s disease next?
Data from GENERATION HD1, the Phase 3 clinical trial testing the huntingtin-lowering drug tominersen, have just been published in a scientific journal. The trial ended a while back, so why is this an important milestone, and what’s next?
The HD pipeline is rich and varied. Let's talk about some out-of-the-box approaches for developing drugs for HD that don't involve huntingtin lowering.
The FDA has approved valbenazine, also known as INGREZZA, as a treatment for the movement symptoms of Huntington’s disease
Novartis have announced that they are ending development of the drug branaplam in Huntington’s disease. Here, we review this latest news and its impact on the HD community.
Scientists at IBM and the CHDI Foundation have used artificial intelligence to analyse datasets from Huntington’s disease observational trials to model progression of the disease. They hope their findings will help improve clinical trial design.
Branaplam was originally designed to treat spinal muscular atrophy, but a new paper outlines how it could hold promise for treating Huntington’s. This oral drug lowers huntingtin protein and will now be tested in a study called VIBRANT-HD.
Researchers with PTC Therapeutics recently published exciting new findings - a promising new huntingtin lowering drug that can be taken as a pill. Will this change how we move forward with huntingtin lowering?
Voyager Therapeutics is shifting towards a new technology to deliver gene therapy and away from a planned HD clinical trial. But this could lead to less invasive drugs in the long run, and many other companies are working on HD gene therapies.