Rocket Pharmaceuticals Inc(9IP1)

Description: Rocket Pharmaceuticals, Inc., together with its subsidiaries, operates as a late-stage biotechnology company that focuses on developing gene therapies for rare and devastating diseases. It has three clinical-stage ex vivo lentiviral vector 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; and pyruvate kinase deficiency, a rare red blood cell autosomal recessive disorder that results in chronic non-spherocytic hemolytic anemia. The company also has a clinical stage in vivo adeno-associated virus program for Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart failure; Plakophilin-2 Arrhythmogenic Cardiomyopathy, an inheritable cardiac disorder; and BAG3 Dilated Cardiomyopathy. It has license agreements with Centro de Investigaciones Energéticas, Medioambientales y Tecnológicas, Centro de Investigacion Biomedica En Red, and Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz; UCL Business PLC; The Regents of the University of California; and REGENXBIO, Inc. Rocket Pharmaceuticals, Inc. was founded in 1999 and is headquartered in Cranbury, New Jersey.

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Keywords: Biotechnology Gene Therapy Heart Failure Genetic Disorder Cardiomyopathy Blood Cells Fanconi Anemia Late Stage Biotechnology Anemias Pyruvate Kinase Deficiency Autosomal Recessive Disorder Chronic Non Spherocytic Hemolytic Anemia Danon Disease Fundacion Instituto Hemolysis Jimenez Diaz Multi Organ Lysosomal Associated Disorder

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Home Page: www.rocketpharma.com

9 Cedarbrook Drive
Cranbury, NJ 08512
United States
Phone: 609 659 8001

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Officers

Exchange: F

Country: DE : Germany

Currency: Euro (€)