Wilson Disease

Prime Medicine Achieves First In Vivo Prime Editing for Wilson Disease

Prime Medicine has treated the first patient using prime editing directly inside the body to address Wilson disease, a rare genetic condition. This marks the first known instance of in vivo prime editing, offering a potential one-time cure by correcting faulty DNA in liver cells to manage copper buildup. The trial aims to replace current complex medication regimens with a permanent genetic fix, with initial results expected next year.