Rocket Pharmaceuticals has signed an agreement to sell its rare paediatric disease priority review voucher (PRV) for $180m to advance its gene therapy pipeline.

The PRV was awarded after the US Food and Drug Administration (FDA) granted accelerated approval for the Kresladi (marnetegragene autotemcel) gene therapy.

Kresladi comprises the patient’s own haematopoietic stem cells that are genetically modified to introduce functional copies of the ITGB2 gene.

Rocket Pharmaceuticals expects to use the proceeds to support its cardiovascular gene therapy projects.

These include clinical-stage studies for Danon disease, PKP2-associated arrhythmogenic cardiomyopathy (PKP2-ACM), and BAG3-associated dilated cardiomyopathy (BAG3-DCM).

A PRV is issued by the FDA to sponsors whose products address certain rare paediatric diseases.

PRV holders may use it to seek an accelerated review for another marketing application or sell the voucher to a different sponsor.

In February 2026, the programme was reauthorised, extending the availability of PRVs for qualifying therapies and maintaining incentives for rare paediatric drug development.

Rocket Pharmaceuticals CEO Gaurav Shah said: “The monetisation 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. This strengthens our ability to advance key clinical milestones across our cardiovascular gene therapy pipeline, with all programmes on track.

“We are deeply appreciative of the US Government’s continued recognition of the importance of therapeutic development for rare and often devastating paediatric disorders, which comprises an essential part of Rocket’s mission.”

The sale forms part of the company’s ongoing strategy to manage capital and prioritise investment in selected gene therapy initiatives.