Rocket Pharmaceuticals

Rocket Pharmaceuticals is a biotechnology company focused on developing innovative gene therapies for rare genetic diseases. Founded in 2015, the company employs both adeno-associated viral (AAV) and lentiviral vector (LV) approaches to create first-in-class treatments. Their mission is to find gene therapy cures for patients facing these challenging conditions, emphasizing that DNA is not a definitive fate. The company has a multi-platform approach to gene therapy, targeting diseases such as Fanconi Anemia, Leukocyte Adhesion Deficiency-I, Pyruvate Kinase Deficiency, and Danon Disease. Rocket Pharmaceuticals is committed to supporting the patient community, recognizing the broader impact of rare diseases on families and caregivers. With a strong leadership team and backing from RTW Investments, Rocket Pharmaceuticals continues to advance its pipeline of investigational therapies.