Our Clinical Program: AFTX-201
AFTX-201 is an investigational gene therapy being evaluated for the treatment of BAG3-associated dilated cardiomyopathy (DCM) in the UPBEAT© clinical trial in the U.S.
BAG3-associated dilated cardiomyopathy (DCM)
BAG3 DCM is a devastating monogenic heart disease affecting more than 70,000 people in the Canada, U.S., Europe, and U.K. regions. The BAG3 gene, or Bcl2-associated athanogene 3, encodes for a protein that is critical to the normal structure and function of muscle cells, particularly those in the heart. People affected by BAG3 DCM have a defect in the BAG3 gene resulting in an insufficient amount of functioning BAG3 protein thereby causing heart failure that typically begins in young adulthood and progresses rapidly. Whereas there are treatments for general heart failure, these do not address the underlying genetic cause of BAG3 DCM and unfortunately, almost 25% of patients require a heart transplant.
AFTX-201, an investigational gene therapy for BAG3-associated dilated cardiomyopathy
Using our engineered heart capsid, ATC-187, we’ve rationally designed an investigational gene therapy, AFTX-201, for BAG3-associated dilated cardiomyopathy (BAG3 DCM). AFTX-201 is intended to target the tissues most affected by the disease, allowing a dose that is 5-10 times lower than that required by conventional capsids such as AAV9 or AAVrh74 and potentially improving safety and effectiveness.
We’ve presented our findings at multiple scientific conferences. To see this breakthrough science, please click here.