Affinia Therapeutics to Present AFTX-201 for BAG3-Associated Dilated Cardiomyopathy (DCM) and Highlight Proprietary Capsid Engineering and Manufacturing Data at the American Society of Gene & Cell Therapy 2026 Annual Meeting

WALTHAM, Mass.--(BUSINESS WIRE)--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 that it will present new preclinical data on the company’s lead program AFTX-201, as well as highlight the design of the Phase 1/2 UPBEAT^© clinical trial investigating AFTX-201 in patients with BAG3-associated dilated cardiomyopathy (DCM), and Affinia’s proprietary capsid engineering and high-yield manufacturing process, in several oral and poster sessions at the American Society of Gene & Cell Therapy (ASGCT) 2026 Annual Meeting, being held May 11-15, 2026 in Boston, MA and virtually.

AFTX-201 is a potential best-in-class investigational genetic medicine for the treatment of BAG3-associated 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 (IV) administration. AFTX-201 increased BAG3 protein levels in the heart and fully restored cardiac function in an animal model of BAG3 DCM.

The U.S. Food and Drug Administration has accepted Affinia’s Investigational New Drug (IND) application for AFTX-201 and granted it Fast Track designation during the first quarter of 2026. In addition, the European Medicines Agency has granted Orphan Drug designation to Affinia for AFTX-201 and the company recently received Clinical Trial Application approval from Health Canada to advance the UPBEAT clinical trial in Canada.

“We look forward to unveiling new preclinical data on AFTX-201, our lead program for BAG3-associated DCM, and the design of our Phase 1/2 UPBEAT clinical trial as well as advancements in our blood brain barrier-penetrant capsids and our high-yield manufacturing process at this year’s ASGCT Annual Meeting,” said Rick Modi, Affinia’s Chief Executive Officer. “The data being presented showcases our scientific progress and readiness for clinical testing of AFTX-201, affirming Affinia’s commitment to delivering potentially curative genetic therapies for people living with devastating diseases.”

• Two oral presentations will showcase AFTX-201 preclinical data and Affinia’s proprietary manufacturing process for AFTX-201.
• Affinia will also hold four poster presentations. The first poster provides an overview of the design of the UPBEAT Phase 1/2 clinical trial evaluating AFTX-201 in patients with BAG3-associated DCM. The second poster describes the development, optimization, and qualification of a comprehensive product-specific analytical panel for AFTX-201 that also sets the foundation for the development of additional product candidates. The company will also present a poster showcasing new results that further validate the central nervous system (CNS) transduction potential of ATC-134, Affinia’s novel engineered AAV capsid which exhibits widespread CNS expression in more than 90% of neurons after IV dosing in nonhuman primates. Lastly, the company will present preclinical data on an investigational gene therapy, developed through a collaboration with Modalis Therapeutics, which has the potential to be a best‑in‑class treatment for Myotonic Dystrophy Type 1.

The oral and poster presentation details are provided below. Abstracts can be found at https://annualmeeting.asgct.org/. Data from the oral presentations are embargoed until 6:00 am ET on the presentation day.

Oral presentations

Title: Process development, technology transfer, clinical manufacturing, and process characterization of AFTX-201, an investigational new medicine for the treatment of BAG3-associated dilated cardiomyopathy
Presenter: Matt Edwards, Vice President, Technical Operations, Affinia Therapeutics
Session: Scale-up, scale-out, formulation, supply chains, quality, and logistics I
Date/Time: Wednesday, May 13, 2026, 4:30-4:45 pm ET
Location: Westin Seaport Commonwealth Ballroom ABC (Concourse Level)
Abstract Number: 248

Title: AFTX-201: A novel investigational AAV gene therapy for the treatment of BAG3-associated dilated cardiomyopathy
Presenter: Giri Murlidharan, Ph.D., Senior Director of Translational Biology, Affinia Therapeutics
Session: Gene therapy for cardiovascular disease
Date/Time: Thursday, May 14, 2026, 3:45-4:00 pm ET
Location: MCEC Room 206AB (Level 2)
Abstract Number: 381

Poster presentations

Title: Study design of a phase 1/2 multicenter, open-label, dose-exploration and dose-expansion trial of AFTX-201 in adults with BCL-2 associated athanogene 3 (BAG3) mutation-associated dilated cardiomyopathy (UPBEAT trial)
Presenter: Charles Cram, Senior Director of Clinical Operations, Affinia Therapeutics
Date/Time: Wednesday, May 13, 2026, 5:00-6:30 pm ET
Location: Exhibit Hall
Abstract Number: 2363

Title: Development, technology transfer, and qualification of product-specific methods for AFTX-201, an investigational new medicine for the treatment of BAG3-associated dilated cardiomyopathy
Presenter: Shahrzad Parker, Ph.D., Senior Scientist, Affinia Therapeutics
Date/Time: Wednesday, May 13, 2026, 5:00-6:30 pm ET
Location: Exhibit Hall
Abstract Number: 2215

Title: Engineered AAV capsid achieves robust transduction in non-human primate central nervous system after low dose systemic administration
Presenter: Kyle Chamberlain, Ph.D., Senior Scientist, Affinia Therapeutics
Date/Time: Wednesday, May 13, 2026, 5:00-6:30 pm ET
Location: Exhibit Hall
Abstract Number: 2026

Title: Pairing AAV genome and capsid engineering of an epigenetic-editing modality to develop a best-in-class treatment for Myotonic Dystrophy Type 1 (DM1)
Presenter: Keith Connolly, Ph.D., Associate Director, Modalis Therapeutics
Date/Time: Tuesday, May 12, 2026, 5:00-6:30 pm ET
Location: Exhibit Hall
Abstract Number: 1492

About the UPBEAT^© clinical trial

The UPBEAT^© clinical trial is a multicenter, single-arm open-label Phase 1/2 clinical trial designed to evaluate the safety, tolerability, pharmacodynamics, and preliminary efficacy of AFTX-201 in adults with genetically confirmed BAG3-associated dilated cardiomyopathy. The trial includes a dose-exploration phase followed by a dose-expansion phase. All participants will receive a single intravenous infusion of AFTX-201 at a dose that has been deemed safe and efficacious based on preclinical studies. The primary objective of the trial is to evaluate safety and tolerability through 52 weeks following administration. Secondary and exploratory objectives include pharmacodynamic and preliminary efficacy assessments, which will be evaluated as changes from baseline. Study design, dose selection, and monitoring plans are informed by input from patients, clinicians, and regulators and by completed nonclinical proof-of-concept, biodistribution, and safety studies, which demonstrated complete correction of heart ejection fraction to normal (wild type) level and adequate safety margin for the doses being explored in the clinical trial. Protocol-defined stopping rules, centralized safety data review, and oversight by an independent Data Safety Monitoring Board are implemented to ensure the safety of participants. Multiple trial sites across the U.S. and Canada are planned to be available. Travel arrangements can be made for interested participants not living near a trial site. Interested participants who have a BAG3 mutation, are 18 – 55 years old, and not able to perform normal activities are encouraged to reach out via [e-mail].

About BAG3 DCM

BAG3 dilated cardiomyopathy (DCM) is a serious, inherited heart condition with a high mortality rate and a significant unmet medical need. The disease affects more than 70,000 patients in Canada and the E.U., U.K., and U.S. regions. The BAG3, or Bcl2-associated athanogene 3, gene encodes for a protein that is critical to the normal structure and function of heart cells. Patients with BAG3 DCM have a mutation in the BAG3 gene and a deficiency in functional BAG3 protein, resulting in early onset heart failure that progresses rapidly. Despite current standard of care, almost 25% of patients require a heart transplant.

About Affinia Therapeutics

Affinia Therapeutics is a clinical-stage biotech company pioneering a transformational treatment paradigm shift to a new class of rationally designed gene therapies that treat rare and prevalent diseases. Affinia Therapeutics’ pipeline of first-in-class or best-in-class product candidates, initially in cardiovascular diseases, leverages its proprietary next-generation capsids, payloads, or manufacturing approaches and have shown efficacy, safety, and differentiation in relevant animal models. For more information, visit https://www.affiniatx.com.