Tranzactioneaza Rocket Pharmaceuticals, (US.RCKT) - 20.03 USD (%) Tranzactioneaza
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Descriere companie
Rocket Pharmaceuticals, Inc. (https://www.rocketpharma.com/) is a clinical-stage, multi-platform biotechnology company that is focused on the development of gene therapies for rare and devastating diseases. The Company’s clinical-stage ex vivo lentiviral vector (LVV) programs include: Fanconi Anemia (FA), is 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 (LAD-I), is a genetic disorder that causes the immune system to malfunction; Pyruvate Kinase Deficiency (PKD), is a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia, and Infantile Malignant Osteopetrosis (IMO), is a genetic disorder characterized by increased bone density and bone mass secondary to impaired bone resorption.