A small biotech company is tackling one of medicine’s biggest challenges: using gene therapy to help millions of people with heart disease. Gene therapy has mostly been used for rare diseases caused by a single gene problem. But heart disease is common and caused by many factors. Medera, led by CEO Ronald Li, has just finished recruiting patients for a early-stage trial of its gene therapy, SRD-002, for heart failure. This is a big deal because heart disease is the leading cause of death in the U.S., with over 64 million cases worldwide. Current medicines can slow heart failure, but they can’t stop it. Li and his team have spent years studying heart failure and other heart muscle diseases. They’ve found a key target that controls heart function, which could help a large number of patients. Medera was founded 10 years ago by Li and Dr. Roger Hajjar. They’ve built on the work of earlier researchers. The first gene therapy for heart disease was developed by Celladon, where Hajjar was a co-founder. But Celladon had problems after a failed trial. Another company, UniQure, also worked on heart failure gene therapies but ended its collaboration with a big drug company. Now, AskBio is also working on a gene therapy for heart failure. Li says they’ve learned a lot from each other. Medera is developing two gene therapies for heart failure. Instead of targeting a specific gene, they’re focusing on a pathway that controls heart function. This could help a lot of patients, but it also means they have to think carefully about safety. They’re using a special testing technology to check for safety and early signs of success. Li says gene therapy is a growing field, and they need to find practical ways to make it work. He’s hopeful that they can bring effective therapies to heart disease patients.