28-04-2025
Affinia Therapeutics to Present New Data on its AFTX-201 Program in BAG3 Dilated Cardiomyopathy, BBB-Penetrant AAV Capsids, and Proprietary Manufacturing Technology at the 28th Annual American Society of Gene & Cell Therapy 2025 Annual Meeting
WALTHAM, Mass.--(BUSINESS WIRE)--Apr 28, 2025--
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 for devastating cardiovascular and neurological diseases, today announced that new preclinical data on AFTX-201, its lead program for BAG3 dilated cardiomyopathy (DCM), as well as its novel AAV capsids for diseases of the central nervous system (CNS), and the company's high-yield manufacturing process, will be presented in several oral and poster sessions at the 28 th Annual American Society of Gene and Cell Therapy (ASGCT) 2025 Annual Meeting, being held May 13-17, 2025 in New Orleans, LA and virtually.
Affinia has rationally designed novel capsids and gene therapies with increased tropism for cardiac muscle, skeletal muscle, or CNS and more uniform tissue distribution than AAV9. This improved biodistribution is attained at doses that are greater than 10-fold lower than those used with conventional capsids such as AAV9 while detargeting the liver and dorsal root ganglia, both potential sites of toxicity. Furthermore, Affinia has developed a proprietary plasmid design system that results in multi-fold improvement in manufacturing yields across a range of novel and conventional capsids and payloads.
AFTX-201, a potential best-in-class investigational gene therapy intended to be given as a simple one-time intravenous (IV) administration, is undergoing investigational new drug (IND)-enabling studies. Affinia has completed a successful pre-IND meeting with the U.S. Food and Drug Administration (FDA) and plans to file an IND in the fourth quarter of 2025. BAG3 DCM is a devastating monogenic heart disease affecting more than 70,000 patients in the U.S., Europe, and U.K. regions alone. The BAG3 gene, or Bcl2-associated athanogene 3, encodes for a protein that is critical to the normal structure and function of heart cells. Patients affected by BAG3 DCM have a mutation in the BAG3 gene and a deficiency in functioning BAG3 protein, resulting in early onset heart failure that progresses rapidly. Despite current standard of care, almost 25% of patients require a heart transplant. AFTX-201 is designed to deliver a fully human, full-length BAG3 transgene using Affinia's novel cardiotropic capsid. Preclinical studies with AFTX-201 have shown correction of BAG-3 protein levels in the heart and complete restoration of cardiac function, whereas such benefits were not achieved with the same gene construct using a conventional capsid at the same dose.
'We look forward to unveiling data on AFTX-201, our lead program for BAG3 DCM, as well as our high-yield manufacturing process and our BBB-penetrant capsids,' said Rick Modi, Affinia's Chief Executive Officer. 'Affinia's purpose is to bring potentially curative treatments to patients affected by devastating diseases. In the near-term, we're focused on advancing our AFTX-201 investigational program into the clinic and evaluating multiple endpoints that have been shown, for other cardiovascular drugs and diseases, to demonstrate efficacy as early as one-to-three months post-dose.'
The oral and poster presentation details are provided below. Abstracts can be found at Updated data, new graphics, and follow up information to be presented are embargoed until 6:00 AM CT on the presentation day for oral abstracts and until 6:00 AM CT on May 13, 2025 for poster abstracts.
Oral presentation
Title: Development of a Flexible High Yielding, High Performing Process for Manufacturing of AFTX-201, a Novel Investigational AAV Gene Therapy for Treatment of BAG3 Dilated Cardiomyopathy
Session: CMC for AAV Vectors
Date/Time: Friday, May 16, 2025, 4:45-5:00 pm CT
Location: Room 288-290
Abstract Number: AMA1147
Presenter: Matt Edwards, MBA, Vice President (VP), Process Science, Affinia
Poster presentations
Title: High-Yield, Pan-Serotype Plasmid System for Manufacturing Adeno-Associated Virus Gene Therapies: Cost and Efficiency Benefits for R&D and Commercial Processes
Date/Time: Tuesday, May 13, 2025, 6:00-7:30 pm CT
Location: Poster Hall 12
Abstract Number: AMA1150
Presenter: Matt Edwards, MBA, VP, Process Science, Affinia
Title: A Novel Investigational AAV Gene Therapy for Treatment of BAG3 Dilated Cardiomyopathy
Date/Time: Wednesday, May 14, 2025, 5:30-7:00 pm CT
Location: Poster Hall 12
Abstract Number: AMA1161
Presenter: Giri Murlidharan, Ph.D., Senior Director, Vector Translational Biology, Affinia
Title: Novel AAV Capsids That Bind Human Transferrin Receptor (TFRC) Demonstrate Widespread and Preferential CNS Tropism in TFRC-KI Mice After Low-dose Systemic Dosing
Date/Time: Thursday, May 15, 2025, 5:30-7:00 pm CT
Location: Poster Hall 12
Abstract Number: AMA877
Presenter: Giri Murlidharan, Ph.D., Senior Director, Vector Translational Biology, Affinia
Title: Engineered AAV Capsids That Target a Novel Human Brain Endothelial Receptor Achieve Robust Transduction in Non-human Primate Central Nervous System After Intravenous Dosing
Date/Time: Thursday, May 15, 2025, 5:30-7:00 pm CT
Location: Poster Hall 12
Abstract Number: AMA1256
Presenter: John Reece-Hoyes, Ph.D., Senior Director, Head of Vector Biology, Affinia
About Affinia Therapeutics
Affinia Therapeutics is pioneering 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 in cardiovascular and neurological 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
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KEYWORD: UNITED STATES NORTH AMERICA LOUISIANA MASSACHUSETTS
INDUSTRY KEYWORD: RESEARCH NEUROLOGY GENETICS CLINICAL TRIALS CARDIOLOGY BIOTECHNOLOGY HEALTH PHARMACEUTICAL SCIENCE
SOURCE: Affinia Therapeutics
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PUB: 04/28/2025 05:14 PM/DISC: 04/28/2025 05:14 PM
