Rocket Pharmaceuticals develops gene therapies for severe and rare inherited diseases. It uses an AAV (Adeno-Associated Virus) platform to deliver corrected genes directly into patients, with the goal of producing a therapeutic protein in targeted cells of the heart, liver, eye, or central nervous system to modify the disease. The therapy work involves engineering AAV constructs so the corrected gene is expressed in diseased cells. The company aims to create first-in-class or best-in-class treatments and generates revenue through partnerships with larger pharma companies, grants, and potential future sales of approved therapies. Core values include Trust, Generosity, Curiosity, and Elevation, guiding a patient-centric approach that puts families first and supports ongoing scientific advancement. Overall goal: provide life-changing gene therapies for patients with few options and expand treatment options for rare genetic disorders.
Company Size
201-500
Company Stage
IPO
Headquarters
Cranbury Township, New Jersey
Founded
2015
See people who can refer or advise you
Help us improve and share your feedback! Did you find this helpful?