Rocket Pharmaceuticals, Inc. (the “Company” or “Rocket”) is a fully integrated, commercial-stage biotechnology company advancing genetic medicines for rare and devastating diseases, with a strategic focus on inherited cardiovascular conditions. The Company’s prioritized development portfolio consists of AAV-based gene therapies for genetically defined cardiomyopathies, complemented by KRESLADI™, its first FDA-approved product, for the treatment of pediatric patients with severe LAD-I who have biallelic mutations in the ITGB2 gene and do not have a suitable HLA-matched sibling donor.

Rocket’s in vivo AAV vector-based cardiovascular portfolio includes a late-stage clinical program for Danon disease, a devastating heart failure condition resulting in thickening of the heart, and early-stage clinical programs for PKP2-arrhythmogenic cardiomyopathy (PKP2-ACM), a life-threatening heart failure disease causing ventricular arrhythmias and sudden cardiac death, and BAG3-associated dilated cardiomyopathy (BAG3-DCM), a heart failure condition that causes enlarged ventricles.

Rocket’s ex vivo LV vector-based hematology portfolio includes KRESLADI™, which received accelerated approval from the FDA in March 2026 for the treatment of eligible pediatric patients with severe LAD-I, as well as additional programs for Fanconi Anemia (FA) and Pyruvate Kinase Deficiency (PKD).