Posted by Michael Wonder on 17 Apr 2026
Medera receives FDA fast track designation for gene therapy targeting Duchenne muscular dystrophy associated cardiomyopathy
16 April 2026 - Medera today announced that the US FDA has granted fast track designation to AAV-SERCA2a, an investigational gene therapy drug designed to treat cardiomyopathy associated with Duchenne muscular dystrophy, building on prior human clinical experience with SERCA2a gene therapy and Medera’s ongoing clinical programs.
AAV-SERCA2a is currently being evaluated in the first in human MUSIC-DMD clinical trial and aims to restore cardiac calcium handling by increasing expression of SERCA2a, a key regulator of heart muscle contraction and relaxation, using Medera's targeted intracoronary gene delivery approach designed to achieve therapeutic cardiac exposure at substantially lower vector doses than conventional systemic gene therapy approaches.
