DNA and money
Source: Genetic Literacy Project

Renovacor, a preclinical-stage gene therapy company, has announced the successful completion of an $11 million Series A financing co-led by Novartis Venture Fund, Broadview Ventures, and BioAdvance, which were joined by New Leaf Venture Partners and Innogest Capital.

Proceeds from the financing will help advance the company’s first-of-its-kind gene replacement therapy through IND filing, in preparation to initiate clinical trials in dilated cardiomyopathy (DCM) patients with mutations in the BAG3 gene.

“There are currently no precision medicine options for cardiovascular patients with specific genetic mutations—a deficiency that Renovacor hopes to address,” said Magdalene Cook, MD, president and CEO of Renovacor. “By bringing the first precision therapy for a cardiovascular disease to the market, we aim to change the therapeutic paradigm that has existed in this field for more than three decades.”

Renovacor’s lead program is a recombinant adeno-associated virus (AAV)-based gene therapy for patients experiencing DCM due to mutations in BAG3. The study of BAG3 mutations and the company’s gene therapy product are based on a decade of research performed by Arthur Feldman, MD, PhD, professor of medicine (Cardiology) at the Lewis Katz School of Medicine, Temple University, and founder of Renovacor.

DCM affects the heart’s ventricles and atria, the lower and upper chambers of the heart, respectively. The disease frequently starts in the left ventricle, the heart’s main pumping chamber. The heart muscle begins to dilate, stretching and becoming thinner. As the inside of that chamber enlarges, the problem often spreads to the right ventricle and then to the atria. The dilated heart muscle doesn’t contract normally nor properly pump blood. As the heart becomes weaker, heart failure can occur as well as heart valve problems, arrhythmias, and blood clots in the heart. DCM affects over three million patients in the U.S. alone, and that number is growing.

Ischemic heart disease is the cause of most cases of DCM, but recently subpopulations have been identified that develop the condition due to specific gene mutations. One of these is the Bcl2-associated athanogene 3 (BAG3) gene. It’s estimated that approximately 35,000 individuals in the United States have this gene mutation, making this an orphan disease. A similar number of DCM patients with BAG3 mutations exist in Europe. These patients are typically younger and progress to end-stage heart failure sooner than patients with ischemic heart disease. Currently, DCM patients with a BAG3 mutation are treated with standard of care for heart failure and their five-year survival is only 50%.

“Renovacor’s gene therapy is the only gene replacement strategy being carried out in a heart failure population today,” said Feldman. “By replacing a missing gene that is causative of disease, Renovacor’s potentially curative treatment aims to stop the progression of the disease and save the lives of otherwise healthy young adults.”

Renovacor also announced its leadership team and board of directors. Cook and Feldman are joined by Thomas Needham, director of Broadview Ventures; Campbell Murray, MD, managing director, Novartis Venture Fund; and Edward J. Benz, MD, president and CEO Emeritus of the Dana-Farber Cancer Institute, and currently professor of medicine at Harvard Medical School.

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