Apr 6, 2026

An investigational medicine that addresses the genetic root cause of BAG3 DCM, AFTX-201 is being evaluated as a treatment for BAG3 DCM in the UPBEAT trial in the U.S. and Canada

AFTX-201 is designed using Affinia’s proprietary capsid engineered for efficient cardiac transduction at doses that are 5-10-fold lower than doses of gene therapies using conventional capsids

WALTHAM, Mass. – April 6, 2026 – Affinia Therapeutics (“Affinia”), an innovative gene therapy company with a pipeline of first-in-class and/or best-in-class adeno-associated virus (AAV) gene therapies initially for devastating cardiovascular diseases, today announced the approval of its clinical trial application (CTA) by Health Canada for AFTX-201, a potential best-in-class investigational genetic medicine for the treatment of BAG3-associated dilated cardiomyopathy (DCM).

AFTX-201 is designed to deliver a fully human, full-length BAG3 transgene using Affinia’s proprietary capsid engineered for efficient cardiac transduction at doses that are 5-10-fold lower than those associated with gene therapies using conventional capsids such as AAV9 or AAVrh74. The safety and efficacy of AFTX-201 as a treatment for people living with BAG3 DCM is being investigated in the UPBEAT© clinical trial. AFTX-201 is given as a simple one-time intravenous administration. Preclinical studies in an animal disease model demonstrated that AFTX-201 increased BAG3 protein levels in the heart and completely restored cardiac function.

“BAG3 DCM is a serious, inherited heart condition with a high mortality and a large unmet medical need. It leads to early onset, progressive heart failure, and premature death and there is no treatment that exists which targets the underlying mechanism of disease. We applaud Affinia for developing promising therapies such as AFTX-201 that address the genetic root cause of BAG3 DCM,” said Rafik Tadros, M.D., Ph.D., Canadian Research Chair in Translational Cardiovascular Genetics, Director of the Cardiovascular Genetics Centre at the Montréal Heart Institute, and the Philippa and Marvin Carsley Chair in Cardiovascular Genetics and Associate Professor at the University of Montréal.

This approval from Health Canada follows recent advances in the clinical development of AFTX-201. Affinia’s Investigational New Drug (IND) application for AFTX-201 has been accepted by the U.S. Food and Drug Administration (FDA) and the FDA granted AFTX-201 Fast Track designation. The European Medicines Agency (EMA) has granted Orphan Drug designation to Affinia for AFTX-201.

“The approval of our CTA in Canada for AFTX-201 is another significant clinical milestone, following the FDA’s recent acceptance of our IND application and granting of the Fast Track designation for AFTX-201,” said Hideo Makimura, M.D., Ph.D., Chief Medical Officer at Affinia. “We are working diligently with multiple trial sites and investigators across the U.S. and Canada to begin patient enrollment and dosing in the next few weeks in the UPBEAT clinical trial so that we can bring this potentially transformative treatment to patients living with BAG3 DCM and make a meaningful impact in their lives.”

Source: Affinia Therapeutics