Rocket Sells PRV for $180 Million

Rare Daily Staff

Rocket Pharmaceuticals said it signed a definitive agreement to sell for $180 million the Rare Pediatric Disease Priority Review Voucher it received with its recent U.S. Food and Drug Administration accelerated approval of its gene therapy Kresladi.

The company said the sale will extend its cash runway into the second quarter of 2028 and help fund its cardiovascular gene therapy pipeline. Previously, Rocket had guided that its existing cash would fund operations into late 2026.

Rocket plans to use the proceeds to advance its prioritized cardiovascular gene therapy pipeline, which includes clinical‑stage programs in Danon disease, PKP2‑associated arrhythmogenic cardiomyopathy and BAG3‑associated dilated cardiomyopathy.

“This strengthens our ability to advance key clinical milestones across our cardiovascular gene therapy pipeline, with all programs on track,” said Gaurav Shah, CEO of Rocket.

The FDA granted accelerated approval to Kresladi at the end of March to treat severe leukocyte adhesion deficiency type I (LAD‑I), an ultra‑rare, life‑threatening genetic disease that appears in infancy.

Children with the severe form of LAD‑I lack sufficient levels of a protein called CD18, which works with partner molecules known as CD11 integrins to help infection‑fighting white blood cells adhere to blood vessel walls and move into tissues. Without that adhesion step, white blood cells remain largely trapped in the bloodstream, leaving babies vulnerable to recurrent bacterial and fungal infections, poor wound healing and a high risk of death in early childhood without effective treatment.

Kresladi is a one‑time treatment made from a child’s own blood‑forming stem cells that have been modified in a lab to add a working copy of the faulty ITGB2 gene. Doctors collect the patient’s stem cells, insert the healthy gene using a disabled lentiviral vector, then infuse the altered cells back after chemotherapy that clears space in the bone marrow. The goal is for the modified cells to engraft and rebuild the immune system so white blood cells can reach sites of infection and help heal wounds.

Congress reauthorized the Rare Pediatric Disease Priority Review Voucher program in February 2026, extending the availability of vouchers for qualifying therapies and reinforcing its use as an incentive for industry investment in ultra‑rare pediatric indications.

Photo: Gaurav Shah, CEO of Rocket Pharmaceuticals.