FDA approved Rocket Pharmaceuticals’ gene therapy Kresladi (marne-cel), delivering MAC-1, and the company said it expects to launch by year-end. Rocket also disclosed that Kresladi earned a rare pediatric disease priority review voucher tied to the approval, underscoring continued FDA activity in rare-disease cell and gene therapies. For biotech investors and developers, the decision is another signal that FDA’s CBER remains open to innovative gene-delivery platforms with clear mechanistic and clinical differentiation. Rocket’s move to commercial-readiness also highlights how quickly sponsors are planning manufacturing and market access after approval. The industry will watch launch execution, uptake, and any follow-on evidence in broader patient populations tied to the MAC-1 approach. Further, the voucher component matters for valuation and pipeline economics by potentially accelerating future review timelines.