First-in-Human gene therapy trial aims to fix faulty heart genes

NCT ID NCT07137338

Summary

This is a very early study testing the safety of a new gene therapy for adults with a specific inherited form of heart muscle weakness (dilated cardiomyopathy). The therapy, called RP-A701, is given as a single intravenous dose and aims to deliver a working copy of a faulty gene (BAG3) to the heart. Researchers will first check if the treatment is safe in 8 patients and look for early signs that it might improve heart function.

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