$11 Million to Push Precision Medicine for Rare Heart Disease


Precision medicine is very commonly used to treat different types of cancer. Now, thanks to $11 million in new financing, Magdalene Cook, M.D., CEO of Renovacor wants to use precision medicine to treat a fatal heart disease.  Cook will lead a charge in gene therapy to treat this rare heart disease. 

“We can detect causative genetic mutations for certain subpopulations and develop precision medicines to address those, as opposed to treating all cardiovascular disease with more of a broad brush,” says Cook. 

The aim is to use precision medicine to treat dilated cardiomyopathy (DCM). This is a condition where the heart’s left ventricle becomes enlarged which causes the heart to struggle to pump blood. DCM is found most commonly in patients with heart disease caused by narrowed coronary arteries; however, a smaller number of patients with DCM developed it due to a mutation in the BAG3 gene. This gene controls the contractility of the heart. When there is a mutation in the gene, it reduces the amount of blood that can be pumped. This causes a dilation and the heart becomes enlarged. This gene also allows for the heart to change shape to accommodate new conditions such as increased amounts of exercise. 

A 2018 study revealed that DCM caused by a BAG3 mutation begins earlier and develops faster than DCM caused by coronary artery disease. Not only this, but DCM caused by a BAG3 mutation is more likely to progress to end stage heart failure. In spite of this, all DCM patients receive the same care.

The new treatment replaces the missing BAG3 to possibly halt the rapid progression of DCM. This would stop the disease before it even began. At this point, the $11 million is being used to move this idea into the clinic and to begin looking into the future of this treatment.

[Source: fiercebiotech.com]

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