WALTHAM, Mass. — Affinia Therapeutics announced that the U.S. Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application for AFTX-201, a novel gene therapy candidate for the treatment of BAG3-associated dilated cardiomyopathy (DCM). The company plans to initiate a Phase 1/2 clinical trial, known as UPBEAT, in the first half of 2026.
AFTX-201 is an investigational adeno-associated virus (AAV) gene therapy designed to deliver a full-length, functional BAG3 transgene using a proprietary capsid developed by Affinia. The capsid has been engineered to achieve efficient cardiac transduction at significantly lower doses—5 to 10 times lower—than traditional gene therapies that rely on conventional AAV capsids such as AAV9 or AAVrh74. AFTX-201 is intended for administration via a one-time intravenous infusion.
“We are grateful to the FDA for the timely review of our IND application for AFTX-201 for the treatment of BAG3-associated dilated cardiomyopathy and to the patient and clinician community who have provided valuable input and support for our investigational program to-date,” said Hideo Makimura, M.D., Ph.D., Chief Medical Officer at Affinia. “We look forward to initiating the UPBEAT clinical trial at multiple trial sites in the coming weeks and bringing a much-needed treatment option for patients and families affected by this devastating disease.”
The UPBEAT clinical trial is a multicenter, open-label Phase 1/2 study that will assess the safety, tolerability, pharmacodynamics, and early efficacy of AFTX-201 in adult patients with genetically confirmed BAG3 DCM.
“BAG3 DCM is a genetic heart disease with significant medical need despite current standard of care,” said Matthew Wheeler, M.D., Ph.D., a physician scientist in genetic cardiomyopathies and Associate Professor in Cardiovascular Medicine at Stanford Medicine. “A gene therapy approach could make a real difference to patients living with BAG3 DCM.”
In preclinical studies, AFTX-201 demonstrated promising results, including increased BAG3 protein expression in cardiac tissue and full restoration of cardiac function in animal models of disease. These findings support the potential of AFTX-201 to address a major unmet need in genetic cardiomyopathies.
Affinia Therapeutics is advancing a pipeline of first-in-class and best-in-class AAV gene therapies, with a focus on diseases that currently lack effective treatment options. The IND acceptance for AFTX-201 represents a significant step forward in the company’s efforts to bring innovative therapies to patients with severe cardiovascular conditions.
