Post by Olga Flamini (Borodovitsyna), MD, PhD

Another Turn in the UniQure Story Several weeks ago, I posted about the UniQure story and the abrupt turn the FDA took in its evaluation of the company's Huntington's disease gene therapy program. Yesterday, the gene therapy field received encouraging news: the FDA reversed its earlier decision requiring UniQure to conduct an additional placebo-controlled clinical trial and will continue evaluating the therapy based on the existing clinical and natural history datasets. This decision feels much more aligned with the guidance documents and public messaging the FDA has been issuing recently, emphasizing the importance of bringing therapies to patients with devastating rare diseases and giving them a chance when few or no treatment options exist. For patients and families affected by Huntington's disease, every year matters. For Huntington and many other rare disease trials, additional placebo-controlled trials can add years to development, and in rapidly progressive diseases, patients don’t have time. It will be interesting to see what broader implications this decision may have for the rare disease and gene therapy fields. For now, it is positive news for patients, developers, and the field overall. It is an exciting time to be working in rare disease drug development.