athanogene 3 (BAG3) gene.

Dilated cardiomyopathy is a condition affecting over 3 million patients in the US and growing steadily. Many patients develop DCM due to ischemic heart disease. Recently subpopulations have been identified that develop DCM due to mutations in specific genes that have been shown to result in the development of DCM. One of these specific genes is the BAG3 gene. The prevalence of disease‑causing BAG3 haploinsufficiency is estimated at approximately 35,000 individuals in the United States, representing an orphan disease by FDA guidelines. It is estimated a similar number of DCM patients with BAG3 mutations exist in Europe. These patients are typically younger and progress to end-stage heart failure sooner than patients with ischemic heart disease. Currently, DCM patients with a BAG3 mutation are treated with standard of care for heart failure. Despite improvements in pharmacotherapy and care, the five‑year survival of a patient with DCM is only 50%. The development of a BAG3 gene replacement therapy for patients with DCM that carry BAG3 mutations could potentially prevent the progression of disease in this otherwise healthy population of young adults.