Pioneering Gene Therapies for Rare Diseases

Rocket Pharmaceuticals is on a mission to develop first-in-class and best-in-class curative gene therapies for patients with devastating rare diseases. The company has strategically built a sustainable multi-platform pipeline of treatments that directly target the genetic mutation in affected cells. Rocket's integrated pipeline includes potential therapies for Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Danon Disease, Pyruvate Kinase Deficiency, arrhythmogenic cardiomyopathy, and more.

With decades of biotech leadership experience and a passion for helping patients, Rocket has grown rapidly, moving four programs into the clinic in just two years. Rocket strives to elevate the life experience of patients and families by creating a supportive community and putting their needs first.