AFTX-201

AFTX-201 is an investigational gene therapy designed to treat BAG3-associated dilated cardiomyopathy (BAG3 DCM), a severe monogenic heart disease. The therapy uses a novel, second-generation adeno-associated virus (AAV) capsid (ATC-0187) engineered for efficient and selective cardiac transduction at low doses. It delivers a fully human, full-length BAG3 transgene to restore normal levels of the BAG3 protein in heart cells, addressing the genetic root cause of the disease. Preclinical studies have shown that a single intravenous dose of AFTX-201 restores cardiac function and structure in animal models with superior efficacy and safety compared to conventional AAV9-based constructs. The therapy is intended as a one-time treatment and has demonstrated robust expression in cardiomyocytes with minimal off-target effects. There are currently no approved therapies targeting the underlying mechanism of BAG3 DCM; many patients progress rapidly to heart failure requiring transplant[1][2][4].

Other names

AAV BAG3-DCM