Medera And University Of Kansas Medical Center Announce Treatment Of First Patient In MUSIC-DMD Phase 1b Clinical Trial For Duchenne Muscular Dystrophy

Medera Inc. ("Medera"), a clinical-stage biopharmaceutical company focused on targeting cardiovascular diseases by developing next-generation therapeutics, and its clinical development division Sardocor, together with the University of Kansas Medical Center, today announced the successful treatment of the first patient in the MUSIC-DMD Phase 1b clinical trial. The trial is investigating AAV1.SERCA2a, a one-time gene therapy treatment for cardiomyopathy secondary to Duchenne muscular dystrophy (DMD-CM).

The patient was successfully treated via left radial artery access using Medera's proprietary minimally invasive intracoronary infusion methodology. The patient tolerated the procedure well and was discharged following an overnight hospital stay for observation.

The MUSIC-DMD Phase 1b trial is an open-label, controlled study evaluating the safety and efficacy of AAV1.SERCA2a, an adeno-associated virus serotype 1 (AAV1) gene therapy that delivers the SERCA2a gene directly to heart muscle cells, in adult males with DMD-associated cardiomyopathy. The study will enroll up to 12 participants across low-dose, high-dose, and control groups. DMD-associated cardiomyopathy is characterized by calcium overload in heart muscle cells, leading to progressive fibrosis and heart failure. AAV1.SERCA2a aims to restore calcium handling in DMD-CM patients by increasing SERCA2a protein production, potentially reversing the underlying disease process.