Affinia Therapeutics to Present AFTX-201 Preclinical Data and UPBEAT Trial Progress in BAG3-Associated Dilated Cardiomyopathy at HFSA 2026
Affinia Therapeutics, a clinical-stage gene therapy company developing potentially first-in-class and best-in-class adeno-associated virus (AAV) gene therapies for serious diseases, particularly cardiovascular conditions, has announced that preclinical and translational data for its investigational gene therapy AFTX-201 will be featured at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2026. The presentation will highlight the scientific development of AFTX-201 and the ongoing Phase 1/2 UPBEAT™ clinical trial evaluating the investigational treatment in patients with BAG3-associated dilated cardiomyopathy (DCM).
The announcement represents an important milestone in Affinia Therapeutics’ efforts to develop gene therapies targeting the underlying genetic causes of severe cardiovascular diseases. Through AFTX-201, the company aims to address BAG3-associated DCM, a progressive heart muscle disorder associated with impaired cardiac function and a significant unmet medical need. The investigational therapy is designed to target the genetic basis of the condition, with the goal of providing a potential treatment option for patients who currently lack an approved therapy specifically addressing the underlying disease mechanism.
The HFSA Annual Scientific Meeting provides a platform for cardiovascular specialists, researchers, clinicians and biotechnology companies to exchange scientific findings and discuss emerging approaches to the prevention and treatment of heart failure. Affinia Therapeutics’ participation will include an oral scientific presentation and an industry-sponsored innovation event focused on the role of gene therapy in treating dilated cardiomyopathies.
AFTX-201 to Be Featured in an Oral Scientific Presentation
Laura Richman, D.V.M., Ph.D., DACVP, Chief Development Officer at Affinia Therapeutics, will deliver an oral presentation titled “AFTX-201: A low-dose, investigational AAV gene therapy using a cardiotropic capsid for the treatment of BAG3-associated dilated cardiomyopathy: Journey to the clinic.”
The presentation is scheduled for Saturday, October 10, 2026, at 1:21 p.m. Pacific Time. It is listed under abstract 1639 in Oral Abstract Session 3A, Stage 1, at the HFSA Annual Scientific Meeting 2026.
The presentation will focus on the development of AFTX-201, including the preclinical and translational research supporting its advancement toward clinical evaluation. It will also address the scientific approach behind the investigational gene therapy, particularly the use of a cardiotropic AAV capsid intended to facilitate delivery of the therapeutic genetic material to heart tissue.
AAV-based gene therapy is an area of growing interest in cardiovascular medicine because it offers a potential means of delivering genetic material to cells to address the biological mechanisms responsible for certain inherited disorders. In genetic forms of cardiomyopathy, this approach may provide an opportunity to target the underlying molecular defect rather than relying exclusively on treatments that manage symptoms or complications associated with declining cardiac function.
Affinia’s AFTX-201 program is focused on BAG3-associated DCM, a condition linked to alterations in the BAG3 gene. By developing a therapy directed at the genetic cause of the disease, the company aims to establish a potential treatment approach that could address the progression of cardiac dysfunction.
The oral presentation is expected to discuss the research and development process that supported the program’s progression from preclinical investigation toward clinical testing. Such translational work is important in gene therapy development because it helps researchers evaluate the relationship between the therapeutic design, its biological activity and its potential application in patients.
Addressing the Unmet Need in BAG3-Associated Dilated Cardiomyopathy
BAG3-associated dilated cardiomyopathy is a genetic form of DCM characterized by enlargement of the heart’s ventricular chambers and impaired contraction of the heart muscle. The condition can result in progressive cardiac dysfunction and may develop at an early age, creating substantial challenges for affected individuals and their families.
Dilated cardiomyopathy can interfere with the heart’s ability to pump blood effectively throughout the body. Depending on disease severity and progression, patients may experience declining cardiac function and an increased risk of complications associated with heart failure. Genetic forms of the disease are particularly challenging because the underlying cause is associated with inherited changes that affect the normal function of cardiac cells.
According to Affinia Therapeutics, patients with BAG3-associated DCM face a substantial unmet medical need because the condition can have an early onset and progressive course, while no approved therapeutic option specifically addresses the underlying disease mechanism.
Existing approaches to managing heart failure and cardiomyopathy generally focus on improving cardiac performance, reducing symptoms, managing complications and slowing disease progression. However, these approaches do not necessarily correct the genetic abnormality responsible for an inherited form of cardiomyopathy.
AFTX-201 is being developed with the aim of addressing this underlying genetic cause. The company’s approach is intended to provide a potential disease-targeted treatment option for people living with BAG3-associated DCM.
Commenting on the program, Dr. Richman emphasized the importance of developing a therapy that targets the cause of the disease rather than its downstream effects.
“BAG3 DCM represents a substantial unmet medical need, as patients experience an early onset and progressive cardiac dysfunction with no existing approved therapeutic option that addresses the underlying disease mechanism,” Dr. Richman said.
She also highlighted findings from preclinical research evaluating AFTX-201 in an animal model of BAG3-associated DCM. According to Dr. Richman, these studies demonstrated that a single treatment restored cardiac function and reversed structural abnormalities.
The findings provide a scientific rationale for continued investigation of AFTX-201. Nevertheless, the reported results are from preclinical research and should not be interpreted as evidence that the therapy has demonstrated the same effects in humans. Clinical studies will be necessary to evaluate its safety, tolerability and potential therapeutic benefit in patients.
Investigational Gene Therapy Uses a Cardiotropic AAV Capsid
A central element of the AFTX-201 development strategy is its use of a cardiotropic AAV capsid. AAV capsids are the protein structures surrounding the genetic material carried by an adeno-associated virus vector. Different capsids can influence how effectively a vector reaches and enters particular cell types.
In cardiovascular gene therapy, capsid selection is an important consideration because researchers seek to deliver therapeutic genetic material to heart muscle cells while managing the overall dose and potential risks associated with treatment.
Affinia describes AFTX-201 as a low-dose investigational AAV gene therapy designed for BAG3-associated DCM. Its development strategy incorporates a cardiotropic capsid intended to support delivery to cardiac tissue.
The use of a targeted delivery approach may be important in the development of gene therapies for serious cardiovascular diseases. Efficient delivery to the intended tissue is one of several factors that can influence a candidate therapy’s performance, alongside the genetic payload, vector characteristics, duration of expression, immune responses and overall safety profile.
However, the suitability of a particular AAV vector must be established through rigorous preclinical and clinical evaluation. Researchers must assess whether the intended genetic material reaches the relevant cells, produces the desired biological effect and maintains an acceptable safety profile.
Affinia’s presentation at HFSA 2026 will provide an opportunity to discuss the development strategy and translational evidence supporting AFTX-201. The company’s continued clinical investigation will help determine whether the approach can ultimately provide a meaningful treatment option for patients with BAG3-associated DCM.
Phase 1/2 UPBEAT Clinical Trial Advances
Alongside the scientific presentation, Affinia Therapeutics is advancing its Phase 1/2 UPBEAT™ clinical trial, which is investigating AFTX-201 in patients with BAG3-associated dilated cardiomyopathy.
The trial represents a key step in translating the company’s preclinical research into clinical evaluation. While animal-model studies can provide evidence of biological activity and help establish a rationale for further development, clinical trials are necessary to determine how an investigational therapy performs in people.
The Phase 1/2 design places the program within the early clinical development stage, where researchers evaluate important aspects of treatment safety and tolerability while gathering initial evidence relevant to therapeutic activity. The specific objectives and findings will depend on the study protocol and the data collected during the trial.
Affinia has indicated that the UPBEAT trial is being conducted at multiple institutions in the United States and Canada. The participation of clinical institutions across the two countries is intended to support the evaluation of AFTX-201 in people affected by this genetic form of cardiomyopathy.
Dr. Richman expressed the company’s commitment to advancing the clinical program and working toward a potential treatment option for patients.
“AFTX-201 is designed to address the genetic cause of BAG3 DCM and preclinical studies in an animal model of BAG3 DCM demonstrated a transformative one-time treatment that restored cardiac function and reversed structural abnormalities. We are delighted to advance the UPBEAT clinical trial evaluating AFTX-201 in BAG3 DCM at a number of institutions in the U.S. and Canada so that we can bring a much-needed treatment option to people living with BAG3 DCM,” she said.
The potential for a one-time gene therapy is an important aspect of the program’s development rationale. Unlike conventional treatments that may require ongoing administration, some gene therapy approaches are designed to deliver genetic material intended to produce a sustained therapeutic effect after a single administration.
Whether AFTX-201 can provide durable clinical benefit following one treatment remains to be established through clinical research and long-term follow-up. Its safety, effectiveness, durability and suitability for different patient populations will be important considerations as the program progresses.
Industry Innovation Event to Examine Gene Therapy in Cardiovascular Disease
In addition to the oral scientific presentation, Affinia Therapeutics will sponsor an industry innovation event bringing together internationally recognized leaders in cardiovascular medicine and research.
The dinner presentation, titled “Dilated Cardiomyopathies and Gene Therapy: UPBEAT Clinical Trial in BAG3 DCM with AFTX-201, an Investigational Gene Therapy Using a Low-Dose Cardiotropic Capsid,” is scheduled for Sunday, October 11, 2026, from 7:30 p.m. to 9:00 p.m. Pacific Time.
The event will take place in the Ahwatukee Room on Level 2 of the Sheraton Hotel. According to the company’s announcement, no RSVP is required.
The program will cover emerging insights into the genetic architecture of dilated cardiomyopathy, the potential role of gene therapy in BAG3-associated and other forms of DCM, and the UPBEAT clinical trial evaluating AFTX-201.
The discussion is expected to bring together perspectives from clinical cardiology, human genetics, cardiovascular research and gene therapy development. By addressing both the genetic basis of cardiomyopathy and emerging therapeutic approaches, the event will provide a forum for examining how advances in molecular medicine may influence future treatment strategies.
Leading Cardiovascular Experts to Participate
The industry innovation event will feature Ray E. Hershberger, M.D., Professor of Internal Medicine, Cardiovascular Medicine and Human Genetics at The Ohio State University. Dr. Hershberger is also the founder and principal investigator of the Dilated Cardiomyopathy Research Project.
His involvement brings expertise in cardiovascular medicine and the genetic investigation of dilated cardiomyopathy, areas directly relevant to understanding the disease mechanisms that AFTX-201 is designed to address.
Joshua M. Hare, M.D., FACC, FAHA, will also participate. Dr. Hare is the Louis Lemberg Professor of Medicine and Director of the Interdisciplinary Stem Cell Institute at the University of Miami. He is also an investigator in the UPBEAT clinical trial.
The participation of an investigator involved in the clinical program will provide a clinical research perspective on the ongoing evaluation of AFTX-201 and its potential role in the treatment of BAG3-associated DCM.
Dr. Richman will be joined by Jonathan Plehn, M.D., FACC, Vice President of Clinical Development at Affinia Therapeutics. Together, the participants will contribute perspectives on the genetic foundations of DCM, the development of gene-based treatment strategies and the clinical investigation of AFTX-201.
Their combined expertise is expected to support discussion of the scientific rationale behind gene therapy for inherited cardiomyopathies, the challenges involved in translating preclinical findings into human studies and the evidence needed to establish a new treatment approach.
Advancing Gene Therapy Research for Serious Cardiovascular Conditions
Affinia Therapeutics’ participation in the HFSA Annual Scientific Meeting 2026 highlights its efforts to advance gene therapy research for serious cardiovascular diseases with limited disease-specific treatment options.
The company’s AFTX-201 program combines a low-dose AAV gene therapy strategy with a cardiotropic capsid approach and is supported by preclinical findings in an animal model of BAG3-associated DCM. The ongoing UPBEAT Phase 1/2 clinical trial represents the next stage in determining whether the investigational treatment can translate those findings into potential benefits for patients.
The planned scientific presentation and industry innovation event will also provide opportunities to discuss the broader implications of genetic research for cardiomyopathy treatment. As understanding of inherited cardiovascular diseases continues to evolve, gene therapy approaches may offer new possibilities for addressing conditions in which conventional treatment strategies do not directly target the underlying genetic cause.
For patients with BAG3-associated DCM, the development of a disease-targeted treatment could represent an important advance. However, further clinical evidence will be required to establish whether AFTX-201 can safely and effectively address the condition in humans.
Through the continued development of AFTX-201 and the UPBEAT clinical trial, Affinia Therapeutics is working to evaluate a potential new approach to treating BAG3-associated dilated cardiomyopathy. The program’s progress will depend on the results of ongoing clinical research, including assessments of safety, therapeutic activity and the durability of any observed benefits.
As the company presents its research at HFSA 2026, AFTX-201 remains an investigational therapy, with its ultimate clinical value yet to be determined. The forthcoming discussions will place the program within the broader effort to translate advances in cardiovascular genetics and gene delivery into potential treatments for patients with serious inherited heart conditions.
About AFTX-201 and the UPBEAT™ Clinical Trial
A potential best-in-class genetic medicine given as a one-time intravenous infusion, AFTX-201 is an investigational medicine designed to address the underlying mutation responsible for BAG3-associated dilated cardiomyopathy (DCM). AFTX-201 is designed to deliver a functional BAG3 transgene utilizing Affinia’s proprietary rationally designed capsid, which is intended to enable clinical doses 5-10-fold lower than those associated with conventional capsids such as AAV9 or AAVrh74. Preclinical studies in an animal model of BAG3 DCM demonstrated that AFTX-201 increased BAG3 protein levels in the heart and restored cardiac function and reversed structural abnormalities.
AFTX-201 is being investigated in the UPBEAT™ clinical trial. The UPBEAT clinical trial is evaluating the safety, tolerability, pharmacodynamics, and preliminary efficacy of AFTX-201 in adults diagnosed with BAG3-associated DCM who experience limitations in performing everyday physical activities due to heart failure. The UPBEAT clinical trial is available at a number of institutions across the U.S. and Canada. The trial is eligible to people who have a BAG3 mutation, are 18 – 70 years old, have reduced left ventricular ejection fraction, and have some difficulty performing normal physical activities. Referring physicians and interested participants are encouraged to reach out via clinicaltrials@affiniatx.com.
The U.S. Food and Drug Administration has accepted Affinia’s Investigational New Drug application for AFTX-201 and granted it Fast Track and Orphan Drug designations in 2026. In addition, the European Medicines Agency has granted Orphan Drug designation to Affinia for AFTX-201 and the company has received Clinical Trial Application approval from Health Canada to advance the UPBEAT clinical trial in Canada.
About BAG3 DCM
BAG3-associated 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 people 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.