Rocket Pharmaceuticals

Rocket Pharmaceuticals is advancing an integrated pipeline of genetic therapies that aim to correct the root cause of complex and rare life-threatening disorders. The company’s mission is to develop first-in-class and best-in-class, curative gene therapies for patients with devastating diseases, guided by their vision of ‘Seeking Gene Therapy Cures’. Rocket Pharmaceuticals’ values include Trust, Generosity, Curiosity, and Elevate, which underpin their strategic approach to gene therapy and have positioned the company to launch a sustainable multi-platform pipeline of treatments. This pipeline includes therapies for Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Danon Disease, Pyruvate Kinase Deficiency (PKD), PKP2 arrhythmogenic cardiomyopathy (ACM), BAG3-associated dilated cardiomyopathy (DCM), and multiple undisclosed diseases. The company’s state-of-the-art manufacturing facility in Cranbury, New Jersey, constructed in 2022, spans approximately 103,720 square feet and is specifically designed for their manufacturing needs. Rocket Pharmaceuticals’ clinical AAV-based programs target Danon Disease and PKP2 arrhythmogenic cardiomyopathy (ACM), with a preclinical program for BAG3-associated dilated cardiomyopathy (DCM). Their LV-based programs include treatments for Fanconi Anemia, Leukocyte Adhesion Deficiency-I, and Pyruvate Kinase Deficiency. The company’s R&D and Corporate Headquarters are located at 9 Cedarbrook Drive, Cranbury, NJ 08512, with an additional office in The Empire State Building, New York, NY. Rocket Pharmaceuticals is a founding member of the BeginNGS™ consortium, partnering with Rady Children’s Institute for Genomic Medicine to drive early diagnosis of rare genetic diseases. They have also partnered with genetic testing leader Invitae to advance the Invitae Unlock™ Cardiomyopathy and Arrhythmia testing program, which aims to accelerate early and accurate diagnosis of genetic cardiovascular disease. This program is the most comprehensive cardiology panel available and seeks to uncover the true cause of a patient’s symptoms, identify the optimal care plan, consider clinical research studies, and provide relief from uncertainty.