Rocket Pharmaceuticals’ gene therapy Kresladi (marne-cel) for leukocyte adhesion deficiency type I was among FDA’s March approvals and earned a rare pediatric disease priority review voucher. Rocket said the therapy is the first BLA to pass the CBER review this year, and it plans to launch by year-end. The approval adds to the growing pipeline activity around cell and gene therapies that rely on CBER’s review pathways. It also highlights how priority review vouchers can alter development timelines and commercial planning. Operationally, Rocket’s next steps—scale-up, launch readiness, and payer and provider education—will determine how quickly the first approved product moves from regulatory milestone to real-world treatment access.