SRD-003

SRD-003 is an investigational gene therapy designed to treat Duchenne muscular dystrophy-associated cardiomyopathy (DMD-CM). It utilizes an adeno-associated virus (AAV)-based vector system to deliver the SERCA2a gene directly into cardiac ventricular muscle cells via intracoronary infusion. The goal is to restore or enhance sarcoplasmic reticulum calcium ATPase 2a (SERCA2a) function, thereby improving calcium trafficking in heart muscle cells and potentially reversing or delaying progressive cardiomyopathy in DMD patients. This approach targets a fundamental defect in DMD-related heart failure that is not addressed by conventional small molecule therapies. SRD-003 has received Orphan Drug Designation for this indication and is currently being evaluated in a Phase 1/2 clinical trial[4][5][6][7][8].

Other names

SERCA2a gene therapySERCA-2a gene therapySERCA 2a gene therapyAAV1/SERCA2arAAV1-SERCA2arAAV-1-SERCA2arAAV 1-SERCA2a