Rocket Pharmaceuticals recently held its sixth annual Rare Disease Day event, emphasizing the company's advancements in gene therapy for rare and devastating diseases. The event brought together leaders in the biotech community, patients, advocates, and employees to showcase hope and progress. Approximately 400 million people worldwide and 30 million people in the US live with rare diseases, impacting more individuals than cancer and AIDS combined. The event also highlighted the challenges faced by those living with rare diseases, including the difficulty of receiving a timely and accurate diagnosis and the lack of FDA-approved treatments. Patient stories, such as that of a DCM (Dilated Cardiomyopathy) patient named Becky, shed light on the chronic nature of rare diseases and the support found within communities. Rocket's CEO and President shared insights on the potential of gene therapy to cure genetic-based diseases, emphasizing the significance of replacing faulty DNA with correct DNA. The company's long-term vision is to utilize gene therapy to cure single gene defects and polygenic diseases. Rocket Pharmaceuticals is actively working on gene therapies for various rare diseases and aims to unlock the ability to pursue cures for a range of conditions. The event highlighted the importance of genetic testing and early diagnosis, with a focus on making genetic testing more accessible. The commitment of Rocket Pharmaceuticals to the advancement of gene therapy for rare diseases presents hope for millions worldwide.