Rocket Pharmaceuticals, Inc, together with its subsidiaries, operates as a multi-platform biotechnology company that focuses on developing gene therapies for rare and devastating pediatric diseases. It has clinical-stage lentiviral vector (LVV) programs under clinical testing to treat fanconi anemia, a genetic defect in the bone marrow that reduces production of blood cells or promotes the production of faulty blood cells; and leukocyte adhesion deficiency-I, a genetic disorder that causes the immune system to malfunction It also has additional pre-clinical stage LVV programs include 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.
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