Medera Doses First Patient in AAV Gene Therapy Trial for DMD Cardiomyopathy

BOSTON and KANSAS CITY, Kan. – July 15, 2025 – Medera Inc. and its Sardocor division, in collaboration with the University of Kansas Medical Center, today announced the successful treatment of the first patient in the MUSIC-DMD Phase 1b clinical trial. This marks a pivotal step for AAV1.SERCA2a, a one-time AAV gene therapy targeting cardiomyopathy secondary to Duchenne muscular dystrophy (DMD-CM).

The patient received the treatment via Medera’s proprietary minimally invasive intracoronary infusion methodology through the left radial artery, tolerated the procedure well, and was discharged overnight.

Medera CEO Ronald Li, PhD, emphasized that addressing DMD-CM, now the leading cause of mortality in DMD patients, is a critical unmet need. AAV1.SERCA2a aims to restore calcium handling in heart muscle cells, potentially reversing disease progression in a condition characterized by calcium overload. Dr. Pradeep Mammen, the study’s principal investigator, highlighted the therapy’s innovative approach to fundamental calcium handling defects. Pat Furlong of PPMD noted its promise for “repairing hearts” in Duchenne, a disease affecting over 300,000 people globally with limited current cardiac treatments.