The article proposes a plan to expedite approval of rare pediatric gene therapies using the FDA's expedited approval process. These therapies are ideal for rare diseases, as approximately 80% of them are caused by a single genetic mutation that can be corrected with a single treatment. The FDA supports the use of surrogates, such as protein expression, instead of traditional clinical trials with large numbers of patients. Examples include Rocket Pharmaceuticals' trials for Danon's disease and Regenxbio's trials with Ultragenyx for MPS II and MPS IIIA, where the FDA agreed to surrogate endpoints before the trials began. The new framework allows for approval based on small controlled studies, a biological mechanism and early signals of efficacy, with a requirement for post-approval confirmatory studies. For personalized therapies, the collection of data on the natural course of the disease and the sharing of evidence through "master protocols" are recommended. CBER plans to issue a specific guideline for gene therapies in 2025.[1][3][4][7]