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Gene Therapy Safety Study for Heart Failure Underway

Heartbeat graphic


21 May 2018. An early-stage clinical trial is testing gene therapy to treat malfunctioning heart muscle cells in people with heart failure, where the heart cannot pump enough blood for the body. The study, sponsored by Precigen Therapeutics Inc., a subsidiary of synthetic biology company Intrexon Corp. in Germantown, Maryland, that says the first patient in the trial began receiving gene therapy treatments.

Precigen says the treatments, code-named INXN-4001, are designed to help people with heart failure rebuild their damaged heart muscle cells. Heart failure is a condition where the heart cannot pump enough blood to meet the body’s needs, a condition affecting some 5.7 million people in the U.S., according to Centers for Disease Control and Prevention. Most cases of heart failure affect both the left and right sides of the heart, although in some cases only one side is affected. The leading causes of heart failure are conditions that weaken or damage the heart, including coronary heart disease, high blood pressure, and diabetes.

INXN-4001 is a synthetic DNA molecule containing genes with instructions for producing proteins that help build heart muscles. The genes are delivered as a plasmid, a circular DNA molecule produced separate from chromosomes, and are a characteristic of bacterial DNA. In this case, the plasmid contains genes that carry instructions to cells for producing three proteins needed by damaged cardiomyocytes, cells in heart muscles. The company cells the proteins address multiple malfunctions in damaged heart muscle cells.

The early-stage clinical trial is testing the safety of INXN-4001 in a small group of heart failure patients. The study is enrolling 12 patients at University of Arizona’s heart health center in Tucson who suffer from heart failure. The patients also use a left-ventricle assist device, a surgically implanted mechanical pump that helps the damaged heart pump blood throughout the body. Participants are receiving two doses of INXN-4001, and are being followed for 12 months following their treatments, with the study team looking primarily for occurrences of adverse effects within 6 months of their treatments.

Helen Sabzevari, president of Precigen, says in a company statement that the trial is “also the first use of the retrograde coronary sinus infusion procedure in left ventricular assist device patients.” Retrograde coronary sinus infusion delivers the plasmids with a catheter through the coronary sinus, a primary vein, instead of using benign viruses, often employed for delivery of gene therapies. The study team is also tracking the ability of patients to wean themselves from their left-ventricle assist devices, measured by their ability to walk for 6 minutes, and quality of life. In addition, the trial is testing the feasibility of wearable biosensors to collect data on the patients’ daily activity.

Precigen was founded in 2017 to develop and commercialize synthetic gene and cell therapies initially discovered by Intrexon, and is a wholly-owned subsidiary of Intrexon Corp. The company says it is also developing treatments for cancer and autoimmune disorders.

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