Rocket Pharmaceuticals

Gene Therapies

RP-L201 Clin2 (fast track) leukocyte adhesion deficiency-1 (ODD); RP-A501 Clin1 (fast track) Danon disease (ODD); RP-L301 Clin1 (fast track) pyruvate kinase deficiency (ODD); RP-A601 Clin0 PKP2-ACM (ODD, FT); BAG3 RD cardiovascular

Rocket Pharmaceuticals is a clinical-stage company developing gene therapy treatments for rare childhood disorders. Life-threatening childhood disorders such as Fanconi Anemia, Danon Disease and Leukocyte Adhesion Deficiency are caused by genetic mutations in single genes. Rocket is developing therapies that directly target these mutations in affected cell types using both adeno-associated viral vectors and lentiviral vectors. Rocket’s state-of-the-art laboratories are built for designing, optimizing and advancing candidate therapeutics to clinical stage development.