Rocket Pharmaceuticals sells it's priority review voucher for $180M, securing non-dilutive funding to extend runway and advance gene therapy pipeline.
Rocket Pharmaceuticals sold the Priority Review Voucher (PRV) it received following FDA’s
The sale extends the company's cash runway and provides non-dilutive capital to advance its cardiovascular gene therapy pipeline.
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Rare Pediatric Disease
Rocket received the PRV alongside the accelerated approval of Kresladi earlier in March this year, and has now entered a definitive agreement to sell it for $180 million. The PRV program was reauthorized in February 2026, extending its availability as an incentive for rare pediatric disease drug development.1
“We are deeply appreciative of the U.S. government’s continued recognition of the importance of therapeutic development for rare and often devastating pediatric disorders, which comprises an essential part of Rocket’s mission,” said Gaurav Shah, M.D., CEO of Rocket Pharmaceuticals.
The proceeds are non-dilutive, meaning they do not require issuing new shares, and will be directed toward advancing Rocket's cardiovascular gene therapy pipeline.1 Pro forma for the transaction, the company expects its cash runway to extend into the second quarter of 2028.
"The monetization of our PRV, following the FDA approval of Kresladi, provides meaningful non-dilutive capital and extends our cash runway into the second quarter of 2028," said Shah. "This strengthens our ability to advance key clinical milestones across our cardiovascular gene therapy pipeline, with all programs on track."
Programs that will benefit from the proceeds include clinical-stage gene therapies for Danon disease, PKP2-associated arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy.
Kresladi (marnetegragene autotemcel) is an approved gene therapy for severe leukocyte adhesion deficiency-I, an ultra-rare genetic pediatric disease caused by mutations in the ITGB2 gene.1 The condition impairs the ability of white blood cells to adhere to blood vessel walls and migrate to tissues to fight infection.2
Infants with severe LAD-I suffer from recurrent, life-threatening bacterial and fungal infections that respond poorly to antimicrobials and require frequent hospitalizations. The disease affects an estimated one in 100,000 to one in 200,000 live births in the U.S., with roughly two-thirds of cases classified as severe.
The accelerated approval was based on increases in neutrophil CD18 and CD11a surface expression as a surrogate endpoint, with confirmation of clinical benefit contingent on longer-term follow-up data from an ongoing study and post-marketing registry.2
Donald B. Kohn, M.D., principal investigator of the Phase I/II study and distinguished professor at UCLA, said: "The approval of Kresladi represents the culmination of many years of scientific research and clinical collaboration aimed at addressing the underlying cause of this devastating disease."
