FDA’s March approval of Rocket Pharmaceuticals’ gene therapy Kresladi (marne-cel) marked a notable regulatory milestone, with the drug earning a rare pediatric disease priority review voucher. Rocket said the MAC-1–delivering therapy is expected to launch by year-end, extending the company’s first-product commercialization plans. The approval also signals continued traction for gene therapies navigating FDA’s review workflows—Rocket noted that Kresladi was the first BLA to pass CBER this year, and it cleared in the setting of a high-need rare genetic indication: leukocyte adhesion deficiency type I. For biotech leaders, the combination of a specific CBER review pathway and the voucher add-on underscores how regulatory designations can accelerate downstream adoption for rare-disease assets while shaping development strategies for next launches.