Rocket sells priority review voucher for $180M

Rocket Pharmaceuticals has entered into a definitive agreement to sell its rare pediatric disease priority review voucher to an undisclosed buyer for $180 million.

Proceeds from the transaction will support advancement of Rocket’s prioritized cardiovascular gene therapy pipeline, including clinical-stage programs in Danon disease, PKP2-associated arrhythmogenic cardiomyopathy, and BAG3-associated dilated cardiomyopathy.

Rocket was granted the rare pediatric disease PRV in March 2026, following the approval of Kresladi. The autologous hematopoietic stem cell-based gene therapy was approved for the treatment of pediatric patients with severe leukocyte adhesion deficiency-I (LAD-I) due to biallelic variants in ITGB2 without an available human leukocyte antigen-matched sibling donor for allogeneic hematopoietic stem cell transplant.

LAD-I predisposes patients to recurrent and fatal infections and is near-uniformly fatal in childhood without an allogeneic hematopoietic stem cell transplant. Kresladi contains autologous hematopoietic stem cells that have been genetically modified with a lentiviral vector to deliver a functional copy of the ITGB2 gene, which encodes for the beta-2 integrin component CD18, a key protein that facilitates leukocyte adhesion and enables their extravasation from blood vessels to fight infection.