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JBT-miR2 is a first-in-class, adeno-associated virus (AAV)-based gene therapy designed as a viral-delivered microRNA inhibitor. Its mechanism of action involves reprogramming cardiac muscle cells to divide within the damaged heart, thereby promoting regeneration and repair of heart muscle following permanent injury such as myocardial infarction (heart attack). The therapy works by inhibiting specific microRNAs that restrict cardiomyocyte proliferation, enabling the heart tissue to regenerate after ischemic damage. Preclinical studies have demonstrated increased heart function and reduced scar tissue in animal models treated with JBT-miR2. The drug is being developed primarily for indications including myocardial ischemia and other forms of heart injury, with the goal of preventing progression to heart failure.
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