Rocket Pharmaceuticals Secures FDA Approval and Funding for Gene Therapy Study

Rocket Pharmaceuticals plans to raise $175 million through a stock sale after the FDA agreed to its proposal for accelerated approval of its gene therapy for Danon disease based on a 12-patient study. The company's stock surged by 42% following the announcement. Danon disease is a rare X-linked disorder that weakens the heart and skeletal muscles, with males typically living until around 20 years old and females until around 35 or 40. Rocket's treatment delivers a functional version of the LAMP2 gene using a viral capsid called an AAV.