Affinia Therapeutics to Present Breakthrough Data on AFTX-201, Novel BBB-Penetrant AAV Capsids, and Proprietary Manufacturing Advancements at ASGCT 2025
Affinia Therapeutics, a pioneering gene therapy company focused on developing potentially curative treatments for severe cardiovascular and neurological diseases, has announced that it will present new preclinical data across multiple innovative programs at the upcoming 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT). The conference will take place from May 13–17, 2025, in New Orleans, Louisiana, and will also be accessible virtually.
The company’s presentations will feature new results from AFTX-201, its lead gene therapy candidate for BAG3 dilated cardiomyopathy (DCM), as well as insights into its next-generation, blood-brain barrier (BBB)-penetrant adeno-associated virus (AAV) capsids designed for neurological indications. Additionally, Affinia will showcase advances in its proprietary high-yield AAV manufacturing platform. These findings will be shared through multiple oral and poster sessions, underscoring Affinia’s growing leadership in the field of AAV gene therapy.
AFTX-201: A Potential Best-in-Class Therapy for BAG3 Dilated Cardiomyopathy
AFTX-201 represents a promising new approach to treating BAG3-associated dilated cardiomyopathy—a progressive and life-threatening monogenic heart condition caused by mutations in the BAG3 gene. This mutation results in a deficiency of the BAG3 protein, which plays a crucial role in maintaining the structural integrity and function of heart muscle cells.
The disease currently affects an estimated 70,000 patients across the United States, Europe, and the United Kingdom. Individuals with BAG3 DCM often experience early-onset heart failure, and despite existing treatments, nearly 25% eventually require a heart transplant. No disease-modifying therapies are currently approved, and patients rely on symptomatic management with standard heart failure medications.
Affinia’s AFTX-201 candidate is designed to address the root cause of BAG3 DCM by delivering a full-length, fully human BAG3 gene using the company’s proprietary cardiotropic AAV capsid. Delivered via a one-time intravenous (IV) infusion, AFTX-201 is being developed as a potentially best-in-class therapy that could halt or reverse the course of disease by restoring healthy levels of BAG3 protein in cardiac tissue.
In preclinical studies, AFTX-201 has demonstrated the ability to not only correct BAG3 protein levels in the heart but also restore cardiac function completely. These results are especially promising when compared to the same gene payload delivered using conventional AAV9 capsids, which failed to achieve comparable therapeutic effects at equivalent doses. The superior efficacy of AFTX-201 is attributed to Affinia’s rationally designed AAV capsids that offer enhanced cardiac tropism and improved tissue penetration.
The program is currently in investigational new drug (IND)-enabling studies, with Affinia having successfully completed a pre-IND meeting with the U.S. Food and Drug Administration (FDA). The company plans to submit its IND application in the fourth quarter of 2025 and intends to move rapidly toward initiating clinical trials shortly thereafter.
Transformative AAV Capsid Engineering for Neurological Applications
In addition to its cardiovascular pipeline, Affinia is advancing a portfolio of novel AAV capsids engineered for superior performance in central nervous system (CNS) delivery. These capsids have been rationally designed to penetrate the blood-brain barrier efficiently—a longstanding challenge in the field of gene therapy for neurological disorders.
The new capsids demonstrate more consistent and uniform distribution across CNS tissues compared to conventional AAV9 vectors. Moreover, they achieve effective transduction at doses more than 10 times lower than those required with standard capsids, which could significantly improve safety margins and therapeutic index. These lower required doses may help avoid toxicities commonly associated with AAV therapies, such as hepatotoxicity and dorsal root ganglia (DRG) injury.
By detargeting non-relevant tissues like the liver and DRG and enhancing biodistribution to CNS regions of interest, Affinia’s capsids offer a significant leap forward in the development of AAV-based treatments for neurodegenerative and neurodevelopmental diseases. The ability to deliver therapeutic genes precisely and safely across the BBB could unlock new possibilities for addressing conditions such as Parkinson’s disease, Huntington’s disease, and certain forms of pediatric leukodystrophies.
High-Yield Manufacturing: Enabling Scalable, Cost-Effective Gene Therapy Production
Beyond its therapeutic programs, Affinia is also innovating in the area of gene therapy manufacturing—an essential component for clinical and commercial success. The company has developed a proprietary plasmid design system that significantly increases AAV vector production yields. This high-yield platform has demonstrated consistent, multi-fold improvements across both novel and traditional capsids and various payload types.
Manufacturing remains a major bottleneck in the gene therapy sector, with high costs and scalability challenges limiting broader access to these advanced treatments. Affinia’s innovations are designed to make gene therapy more accessible by improving the efficiency and consistency of production, potentially reducing cost-of-goods and facilitating faster scale-up for both clinical trials and future commercialization.
Rick Modi, Chief Executive Officer of Affinia Therapeutics, expressed his enthusiasm for the upcoming presentations at ASGCT, stating:
“We look forward to unveiling new data on AFTX-201, our lead program for BAG3 dilated cardiomyopathy, alongside exciting advances in our blood-brain barrier-penetrant capsids and proprietary manufacturing platform. These developments reflect our commitment to translating cutting-edge science into transformative therapies that can profoundly improve the lives of patients with serious, often untreatable conditions.”
He added:
“Our near-term priority is to progress AFTX-201 into clinical development. We’re particularly focused on measuring clinical endpoints that have shown early therapeutic signals—sometimes within one to three months—in other cardiovascular indications. This could allow us to rapidly demonstrate proof-of-concept and pave the way for accelerated development timelines.”
Positioning for Long-Term Impact in Gene Therapy
As Affinia continues to advance its pipeline and platform technologies, its integrated approach combining novel vector design, rigorous preclinical validation, and scalable manufacturing positions the company to play a leading role in the next generation of gene therapy innovation. The data presented at ASGCT 2025 will highlight the company’s progress in developing precision-engineered therapies for diseases with high unmet need and limited treatment options.
By targeting both the cardiovascular and neurological systems, Affinia is addressing some of the most difficult challenges in genetic medicine. The company’s emphasis on lowering therapeutic doses, improving tissue targeting, and simplifying administration through IV delivery reflects a thoughtful, patient-centered design philosophy aimed at maximizing both safety and efficacy.
As gene therapy moves into its next phase of maturity, companies like Affinia are pushing the boundaries of what’s possible bringing the field closer to delivering on the promise of durable, one-time treatments for complex genetic diseases.
