Rocket Pharmaceuticals

About:

Rocket Pharmaceuticals is a biotech company that develops a pipeline of genetic therapies for rare childhood disorders.

Website: http://www.rocketpharma.com/

Twitter/X: RocketPharma

Top Investors: RTW Investments, Frazier Healthcare Partners, Civilization Ventures, Sahsen Ventures, California Institute for Regenerative Medicine

Description:

Rocket Pharmaceuticals is a biotechnology company that is working to develop first-in-class gene therapy treatments for rare and undertreated diseases. The Company's lead program is a lentiviral-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult-to-treat genetic disease that causes bone marrow failure and, in the worst-case scenario, cancer. Additional bone marrow-derived disorders are being studied in preclinical trials, including Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-1 (LAD-1), and Infantile Malignant Osteopetrosis. In addition, the company is working on an AAV-based gene therapy program for an unnamed rare pediatric disease.