30 Sep Rocket Pharmaceuticals
Gaurav Shah, M.D., CEO
Oct. 12 | 2:15pm | BlueRock Therapeutics Ballroom
New York, NY
Rocket Pharma is a clinical-stage, multi-platform biotechnology company focused on the development of first, only and best-in-class gene therapies, with direct on-target mechanism of action and clear clinical endpoints, for rare and devastating diseases. Rocket has four clinical-stage ex vivo LVV programs for Fanconi Anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells, Leukocyte Adhesion Deficiency-I, a genetic disorder that causes the immune system to malfunction, Pyruvate Kinase Deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia and Infantile Malignant Osteopetrosis, a genetic disorder characterized by increased bone density and bone mass secondary to impaired bone resorption. Rocket also has a clinical stage in vivo AAV program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure.