Targeting Underlying Gene Mutation Causing Dilated Cardiomyopathy with Dr. Marc Semigran Renovacor

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3y ago 17:28

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Dr. Marc Semigran is the Chief Medical Officer of Renovacor and shines a light on BAG3-associated cardiomyopathy and the cardiac gene therapy in development to treat this rare genetic disease.

Marc explains, "So, what we are focusing on are patients who have a genetic or familial origin of their dilated cardiomyopathy. Our first target is a gene described as BAG3, which is an important protein within the cardiac muscle cells. And some patients with heart failure have the heart failure because the gene that they have is mutated and is not able to fully express a functional protein."

"We are studying our lead asset, which is REN-001, a modified virus that we believe will specifically target the heart. This will insert into the cardiac muscle cells a good copy of the BAG3 gene, which can then be expressed and allow those cardiac muscle cells to begin to function normally."

#Renovacor #GeneTherapy #DilatedCardiomyopathy #DCM #BAG3 #RareDisease

Renovacor.com

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