Clinical-stage biotechnology company developing gene therapies for rare childhood diseases.
Rocket Pharmaceuticals, Inc. is a dynamic biotechnology firm and its subsidiaries are dedicated to advancing gene therapies for rare and severe diseases. Specializing in multiple platforms, the company focuses on developing innovative treatments that target genetic defects responsible for debilitating conditions. Currently, Rocket Pharma boasts three clinical-stage ex vivo lentiviral vector programs. These programs are designed for diseases such as fanconi anemia, which affects bone marrow and hampers blood cell production, leukocyte adhesion deficiency-I, a disorder impairing the immune system, and pyruvate kinase deficiency, a rare red blood cell disorder causing chronic anemia.
Additionally, Rocket Pharmaceuticals, Inc. is pioneering an in vivo adeno-associated virus program targeting Danon disease. This condition, characterized by multi-organ lysosomal-associated dysfunction, often results in premature death due to heart failure. The company's approach integrates cutting-edge genetic therapies with a commitment to clinical excellence, aiming to provide transformative solutions for patients with these challenging diseases.
Rocket Pharma's innovative research is underpinned by strategic partnerships and license agreements with leading institutions and organizations. These collaborations include agreements with Fred Hutchinson Cancer Research Center, Centro de Investigaciones Energeticas, Medioambientales y Tecnológicas (CIEMAT), Centro de Investigacion Biomedica En Red, Fundacion Instituto de investigacion Sanitaria Fundacion Jimenez Diaz, UCL Business PLC, The Regents of the University of California, and REGENXBIO, Inc. Headquartered in Cranbury, New Jersey, Rocket Pharmaceuticals, Inc. remains at the forefront of biotechnological advancements in gene therapy.
Committed to pushing the boundaries of medical science, Rocket Pharmaceuticals, Inc. combines scientific expertise with a passion for addressing unmet medical needs. The company's comprehensive approach not only aims to improve patient outcomes but also underscores its dedication to pioneering therapies that have the potential to redefine the treatment landscape for rare genetic disorders.