scAAVrh10.cTnT.S100A1-ARC

scAAVrh10.cTnT.S100A1-ARC is an experimental bicistronic gene therapy designed to treat cardiomyopathy associated with Duchenne muscular dystrophy (DMD). The therapy utilizes a self-complementary adeno-associated virus serotype rh10 (scAAVrh10) vector under the control of a cardiac-specific troponin T (cTnT) promoter to ensure targeted expression in heart tissue. It simultaneously delivers two therapeutic genes: S100A1, which encodes a calcium-binding protein that improves diastolic function and calcium handling, and ARC (apoptosis repressor with caspase recruitment domains), which inhibits programmed cell death to prolong cardiomyocyte survival. Preclinical studies in mouse models have shown that this dual-gene approach improves long-term cardiac outcomes and prevents heart failure, while safety has been demonstrated in canine models via intracoronary delivery.

Other names

S100A1-ARC gene therapyS-100A1-ARC gene therapyS 100A1-ARC gene therapyDual S100A1 and ARC gene therapy