Rocket Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company dedicated to developing gene therapies for rare inherited diseases. The company leverages a lentiviral vector platform to design and deliver corrective genes to patients suffering from serious genetic disorders. Its research pipeline includes multiple ex vivo autologous gene therapy candidates aimed at addressing conditions such as Fanconi anemia, leukocyte adhesion deficiency-I, pyruvate kinase deficiency and Danon disease, among others.
Rocket’s proprietary platform is built on decades of lentiviral vector research and has been optimized to ensure stable gene transfer, durable expression and a favorable safety profile. The company’s lead programs are advancing through preclinical development and early-phase clinical trials, with a focus on demonstrating proof of concept for long-term therapeutic benefit. In addition to its internal pipeline, Rocket collaborates with academic institutions and patient advocacy groups to support translational research and broaden access to its treatments.
Founded in 2015 and headquartered in New York City, Rocket Pharmaceuticals operates research and manufacturing facilities in the United States and Europe. The company has established partnerships with contract research organizations and clinical sites across North America and the European Union to conduct multicenter trials. This global infrastructure positions Rocket to accelerate patient enrollment and streamline regulatory filings in multiple regions.
Under the leadership of Chairman and Chief Executive Officer Jason L. Cole, M.D., Rocket Pharmaceuticals is guided by a team of experienced executives, scientists and advisors. The company’s board includes experts in gene therapy, hematology and rare disease drug development, reflecting its commitment to advancing innovative treatments. Rocket continues to pursue strategic collaborations and funding opportunities to support the development of its pipeline and bring transformative therapies to patients with high unmet medical needs.
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