MBD-002

MBD-002 is an investigational gene therapy being developed by Medera Biopharmaceutical for the treatment of Barth Syndrome. Barth Syndrome is a rare, X-linked genetic disorder caused by mutations in the *TAZ* gene, which encodes the mitochondrial enzyme tafazzin. This enzyme is critical for the remodeling of cardiolipin, a phospholipid essential for mitochondrial structure and energy production. Deficiency in tafazzin leads to mitochondrial dysfunction, resulting in cardiomyopathy, skeletal muscle weakness, and neutropenia. MBD-002 is designed to deliver a functional copy of the *TAZ* gene to restore tafazzin activity and normalize mitochondrial function. The therapy is currently in the IND-enabling stage and utilizes Medera's proprietary delivery technologies, which include specialized intracoronary administration methods to target cardiac tissue effectively.