June 12, 2026 —
Rocket Pharmaceuticals announced the closing of the sale of its Rare Pediatric Disease Priority Review Voucher for gross proceeds of $180 million, providing non-dilutive capital to support the company’s genetic medicine pipeline.
The PRV was granted by the U.S. FDA in March 2026 in connection with the approval of KRESLADI™, Rocket’s gene therapy for severe leukocyte adhesion deficiency-I, or LAD-I, a rare and life-threatening primary immunodeficiency. KRESLADI represents an important commercial milestone for Rocket and supports the company’s transition into a commercial-stage genetic medicine company.
Before the PRV sale, Rocket reported cash, cash equivalents, and investments of $144.4 million as of March 31, 2026. Following the $180 million in proceeds, the company’s pro forma cash, cash equivalents, and investments increased to approximately $322.6 million. Rocket expects these resources to fund operations into the second quarter of 2028.
The financing strengthens Rocket’s balance sheet as it continues to advance its broader pipeline of genetic therapies, including both lentiviral and AAV-based gene therapy programs. While KRESLADI is an ex vivo lentiviral gene therapy for LAD-I, Rocket’s AAV pipeline is primarily focused on cardiovascular diseases with high unmet need.
Rocket’s lead AAV program is RP-A501, an investigational AAV9 gene therapy for Danon disease, a rare and often fatal genetic disorder caused by mutations in the LAMP2 gene. Danon disease is characterized by severe cardiomyopathy and can also involve skeletal muscle weakness and neurological manifestations. RP-A501 is designed to deliver a functional LAMP2B gene and is being evaluated in a pivotal Phase 2 study.
The company is also advancing RP-A601, an AAV-based gene therapy candidate for PKP2-associated arrhythmogenic cardiomyopathy, or PKP2-ACM. PKP2 mutations are linked to arrhythmogenic right ventricular cardiomyopathy, a genetic heart disease that can cause arrhythmias, progressive cardiac dysfunction, and sudden cardiac death. Rocket has been engaging with the FDA on alignment for a potential pivotal Phase 2 trial design while continuing enrollment in the Phase 1 study.
Another AAV cardiovascular program, RP-A701, is being developed for BAG3-associated dilated cardiomyopathy, or BAG3-DCM. BAG3 mutations can lead to heart failure through impaired cardiac muscle function and ventricular dilation. Rocket has received IND clearance for RP-A701, and Phase 1 trial start-up activities have been underway.
Together, these programs reflect Rocket’s strategic focus on AAV-mediated gene therapy for inherited cardiomyopathies, where a single-gene defect contributes to progressive and life-threatening cardiac disease. The company’s cardiovascular pipeline is designed to address the underlying genetic causes of disease by delivering functional genes to affected tissues.
The PRV sale gives Rocket additional financial flexibility as it supports the launch of KRESLADI and advances its AAV programs through key clinical and regulatory milestones. With pro forma funding into the second quarter of 2028, Rocket is positioned to continue developing its rare disease gene therapy pipeline while managing the capital requirements of clinical development, manufacturing, and commercialization.
