Gene replacement could stop the progression of dilated cardiomyopathy.
Patients suffering from dilated cardiomyopathy due to mutations in the BAG3 gene could have a new curative treatment for the disease.
Renovacor, a preclinical-stage biopharmaceutical company, today announced it will receive $11 million in Series A funding for its recombinant adeno-associated virus-based gene therapy.
“There are currently no precision medicine options for cardiovascular patients with specific genetic mutations — a deficiency that Renovacor hopes to address,” said Magdalene Cook, M.D., president and CEO of Renovacor. “By bringing the first precision therapy for cardiovascular disease to the market, we aim to change the therapeutic paradigm that has existed in this field for more than three decades.”
The company will use the funding to advance its gene replacement therapy by filing an Investigational New Drug application to the U.S. Food and Drug Administration. This will prepare Renovacor to begin human clinical trials in dilated cardiomyopathy patients who have mutations in their BAG3 gene, according to researchers.
Dilated cardiomyopathy, which affects more than 3 million patients in the U.S., Renovacor says, is a condition in which the heart’s ability to pump blood is decreased because the heart’s main pumping chamber is enlarged and weakened.
Some patients develop dilated cardiomyopathy due to mutations in specific genes, including the Bcl2-associated athanogene 3 (BAG3) gene.
Patients who have this mutation are treated with standard care for heart failure. But the five-year survival of a patient with dilated cardiomyopathy is only 50%. And gene replacement therapy has the potential to prevent the progression of the disease.
“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,” said Arthur Feldman, M.D., Ph.D., founder of Renovacor.
Novartis, Venture Fund, Boradview Ventures and BioAdvance co-led the funding, with additional investments from New Leaf Venture Partners and Innogest Capital.
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