Gene Therapy for Heart Failure: A Conversation with Eric Adler, MD

In the fall of 2024, the California Institute for Regenerative Medicine (CIRM) helped sponsor the Gene Therapy Initiative symposium in La Jolla, CA. While there, CIRM staff caught up with a few CIRM grantees who are working on gene therapy approaches to treating diseases. One of these grantees was Eric Adler, MD, from the University of California, San Diego (UCSD), who has received more than $8 million in funding from CIRM to work on gene therapy approaches to treat heart failure.

Dr. Adler is a professor of medicine and director of the Strauss Center for Cardiomyopathy at UCSD, and Chief Medical Officer at Lexeo Therapeutics.

This interview is edited slightly for length and clarity.

CIRM: How could gene therapy benefit people with heart failure?
Dr. Adler: Heart failure is often caused by genetic mutations, and I seek to develop genetic medicines to treat these patients. Gene therapy is the most promising hope for patients with heart failure who are otherwise likely to die or require a heart transplant.

CIRM: How do mutations cause heart disease, and what are some symptoms?
Dr. Adler: Heart failure is one of the most common causes of death and hospitalization in the United States. When young people get heart failure, it is frequently caused by errors in their cardiac genes (mutations). When they have these mutations, their heart is often no longer capable of doing its job. Patients sometimes notice feeling short of breath or having fatigue. Others may have their hearts stop without warning.

CIRM: How has CIRM supported this work?
Dr. Adler: CIRM has provided nearly continuous funding for my research on this topic for over 10 years. With the support of CIRM, gene therapies for these patients are being tested as we speak.

CIRM: What has CIRM's impact been on gene therapy and regenerative medicine in the past 20 years?
Dr. Adler: CIRM has led to many breakthroughs in gene and cell therapy that I believe will directly improve the lives of many. It has also supported the careers of physicians and scientists throughout California and has had a significant impact on the fields of biology and medicine.

Written by guest contributor Amy Adams