The therapy targets a complex heart condition, hypertrophic cardiomyopathy, that can lead to the thickening of the heart muscle and a stiffening of the left ventricle. It works by delivering a Myosin binding protein C3 to the heart muscle via a single infusion.
“The hope is that this new gene will restore normal levels of the protein, which regulates the contraction and relaxation of the heart muscle,” the release states. “In preclinical studies, a single dose restored normal levels of the protein which led to disease reversal.”
Cleveland Clinic experts also led a 2022 trial that led to the FDA’s approval of the only other existing treatment for hypertrophic cardiomyopathy, a drug known as mavacamten.
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