The FDA has announced a comprehensive plan to streamline and expedite the approval process for gene therapies aimed at treating ultra-rare diseases. This new framework proposes various pathways to facilitate quicker access to personalized treatments, emphasizing the need for innovation and responsiveness to patient needs. The initiative includes guidance on testing bespoke therapies, which are expected to increase the number of applications for rare disease treatments. The FDA is seeking public comments on these new approaches, which aim to enhance outcomes for patients suffering from conditions with limited treatment options.