Newswise — LOS ANGELES (Dec. 11, 2025) — The California Institute for Regenerative Medicine (CIRM) has awarded Cedars-Sinai researchers more than $20 million to study potential treatments for heart failure and an inherited type of heart disease.
CIRM, a taxpayer-funded state agency, supports the development of treatments for an array of diseases and conditions.
“The people of California expect us to deliver on the promise of cell and gene therapy using taxpayer dollars, a trust we take seriously,” said Eduardo Marbán, MD, PhD, executive director of the Smidt Heart Institute at Cedars-Sinai. “With these grants, we will work toward bringing novel therapies to patients with heart disease.”
Hope for Heart Failure
One grant of more than $10 million will allow Cedars-Sinai investigators to spend the next five years studying a treatment for a common form of heart failure called heart failure with preserved ejection fraction.
More than 6 million adults in the U.S. experience heart failure, which occurs when the heart doesn’t pump enough blood to meet the body’s needs. In heart failure with preserved ejection fraction, the most common type of heart failure, the heart is too stiff to properly fill with blood. It leads to death in more than half of people who develop the condition; no medications exist to stop the disease or extend a patient’s life.
David Lefer, PhD, director of Translational Research in the Department of Cardiac Surgery in the Smidt Heart Institute, will lead preclinical studies to investigate a new RNA drug, TY1, combined with the weight loss drug semaglutide as a new, more effective way to treat heart failure with preserved ejection fraction. Investigators aim to launch clinical trials within a few years.
“Obesity is a major risk factor for heart failure with preserved ejection fraction,” Lefer said. “We hope that creating a treatment that couples the benefits of a GLP-1 drug, and the immunomodulatory benefits of the RNA exomer TY1, might reduce the risk of death from heart failure.”
New Research Into a Cause of Sudden Cardiac Death
A second CIRM grant will provide more than $10 million over the course of five years for Cedars-Sinai investigators to study arrhythmogenic cardiomyopathy (ACM), a rare, inherited heart disease. ACM is the leading cause of sudden cardiac death in athletes and young people, and no medications exist to halt its progression. The grant will fund preclinical and safety studies of a new drug that may stop the disease and even reverse the heart damage it causes.
“Many people have no idea they have ACM until they experience cardiac arrest or heart failure,” said Alessandra Ciullo, PhD, a project scientist in the Smidt Heart Institute. “We’d like to offer those patients a treatment to reverse the disease and prolong lives.”
CIRM grants have supported several studies in the Smidt Heart Institute, including research into a potential new treatment for ventricular tachycardia and a cell therapy for pulmonary arterial hypertension.
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