Rocket Pharmaceuticals

Rocket Pharmaceuticals is an emerging biotechnology company dedicated to developing first-class gene therapy treatments for rare untreated diseases. The company's lead program is a lentivirus-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult-to-treat genetic disorder that can lead to bone marrow failure and potential cancers. Preclinical studies for other bone marrow-derived diseases are underway, targeting Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-1 (LAD-1), and Infantile Malignant Osteoporosis. The company is also developing an AAV-based gene therapy program for undisclosed rare pediatric diseases.