Rocket Pharmaceuticals Inc.

This study tests a gene therapy called RP-A501 in 14 males aged 8 and older with Danon disease, a rare genetic condition that weakens the heart. The therapy uses a harmless virus to deliver a working copy of the LAMP2 gene, aiming to improve heart muscle function and reduce thick…

This early-stage trial tests a single intravenous gene therapy (RP-A701) for adults with dilated cardiomyopathy caused by a specific genetic defect (BAG3). The goal is to see if it is safe and can improve heart function. Only 8 high-risk participants aged 18-65 with moderate hear…

This early-stage trial tests a single intravenous dose of a gene therapy called RP-A601 in 9 adults with a genetic heart condition (PKP2-ACM) that causes dangerous heart rhythms. The goal is to see if the treatment is safe and can restore a missing protein in heart cells to reduc…

This study observes 60 people with Danon disease, a rare genetic heart condition, to see how it changes over time. Participants include males 8 and older and females 8 to 50. The goal is to better understand the disease, not to test a treatment.

This study follows 36 people with a rare genetic heart condition called PKP2-ACM to track how the disease progresses over time. Participants receive standard care while researchers monitor heart rhythms, biomarkers, and quality of life. The goal is to better understand the condit…