Rocket Pharmaceuticals Inc.

This early-stage trial tests a single intravenous dose of a gene therapy (RP-A701) in 8 adults with dilated cardiomyopathy caused by BAG3 gene mutations. The goal is to see if the treatment is safe and can improve heart function. Participants must be 18-65 years old with mild to …

This study tests a gene therapy called RP-A501 in 14 males aged 8 and older with Danon disease, a rare genetic condition that causes heart problems. The treatment uses a harmless virus to deliver a working copy of the LAMP2 gene directly into the body. The goal is to improve hear…

This early-stage trial tests a one-time gene therapy for people with a genetic heart condition that causes dangerous heart rhythms. The therapy delivers a working copy of the PKP2 gene to heart cells. The study involves 9 adults with an implanted defibrillator and frequent extra …

This study follows 60 people with Danon disease, a rare genetic heart condition, to see how it progresses. Participants include males 8 and older and females 8 to 50. Researchers will measure heart size, blood markers, and quality of life to better understand the disease and help…

This study follows people aged 12 and older who have a genetic heart condition called PKP2-ACM. Researchers will monitor heart rhythms, biomarkers, and quality of life over time to better understand how the disease naturally progresses. No new treatments are given; participants c…