Testing a new gene therapy for severe inherited high triglycerides

This trial is testing a new gene therapy called GC304 to treat people with a rare, inherited form of very high triglycerides (a type of fat in the blood) that hasn't responded well to diet and medication. The study focuses on patients who carry genetic mutations that cause this condition and have experienced serious pancreatitis (pancreas inflammation) as a result. Researchers want to understand whether this one-time injection is safe and whether it can help lower triglyceride levels over time.

People with this genetic condition often develop life-threatening pancreatitis attacks even with standard treatments. This medication offers a new approach by working at the genetic level to address the root cause, which could prevent future pancreatitis episodes and reduce serious health complications.