Promising Gene Therapy for Heart Failure

MARCH 29, 2016
Gale Scott
Could curing heart failure be as simple as administering a one-time outpatient infusion of a gene therapy product?

In a study published in JAMA Cardiology, researchers presented promising results of a Phase II trial showing that gene transfer treatment of patients with heart failure and reduced ejection fraction was safe and successful in over 90% of patients.

In an interview, Jack Reich, PhD, co-founder and CEO of Renova Therapeutics in San Diego, CA, which plans to market the gene product as “RT-100,” said the patients were treated with an infusion of AC6, a protein found in cardiac muscle cells.

In most heart failure patients this protein is down-regulated in the heart, leaving the organ contracting weakly.

In the trial, an inactivated adenovirus vector encoding human adenylyl cyclase type 6 (Ad5.hAC6) was infused directly into the arteries to the heart during cardiac catheterization.

“Forty percent of the heart cells took it up like a sponge,” Reich said.

In the journal article, lead author Kirk Hammond, MD, co-founder of Renova, and colleagues concluded that the two end points of the study “showed significant between-group differences: (1) AC6 gene transfer increased LV peak −dP/dt (P < .03); and (2) AC6 gene transfer increased EF in participants with nonischemic heart failure (P = .02).”

The AC6 gene transfer “safely increased LV function beyond optimal heart failure therapy through a single administration,” the team wrote.

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