Latest news with #BAG3DCM
Associated Press
5 days ago
- Business
- Associated Press
Affinia Therapeutics and DCM Foundation Announce Partnership to Increase Awareness About BAG3 Dilated Cardiomyopathy and Critical Need for Genetic Testing to Help Save Lives
WALTHAM, Mass. & DUBLIN, Ohio--(BUSINESS WIRE)--Jun 3, 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, and the DCM Foundation together with the Genetic Cardiomyopathy Awareness Consortium (GCAC), today announced they have joined forces to raise awareness about BAG3 dilated cardiomyopathy (DCM), with the goal of promoting early diagnosis and the critical need for genetic testing. 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 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. According to a study published in Circulation, the journal of the American Heart Association, close to 50% of cardiomyopathy has some type of genetic basis, such as BAG3 DCM, yet only a fraction of diagnosed cardiomyopathy patients get genetic testing. This new-found partnership between Affinia and the DCM Foundation and GCAC aims to educate about BAG3 DCM and the critical need for genetic testing, and champions the needs and voices of people living with this devastating heart disease through initiatives including: 'The DCM Foundation and GCAC are very grateful for this partnership with Affinia,' said Greg Ruf, Founder and Executive Director, the DCM Foundation. 'By getting more cardiomyopathy patients tested, we can potentially save and improve lives and help advance cardiomyopathy research and therapies. Through this collaboration, we will collectively unite our strengths and work together in the hope of making a real difference for those living with this devastating disease.' Hideo Makimura, M.D., Ph.D., Chief Medical Officer of Affinia, commented, 'BAG3 DCM is a devastating heart disease with a known genetic cause. Unfortunately, only a fraction of patients affected with BAG3 DCM and other genetic cardiomyopathies are tested, which is putting lives at risk. We are committed to working together with the DCM Foundation and GCAC to increase disease awareness and the role genetics plays in cardiomyopathy, which we believe will ultimately lead to better outcomes for people living with BAG3 DCM.' 'Our partnership with the DCM Foundation and GCAC is an exciting milestone as we advance our lead program, AFTX-201 for BAG3 DCM, toward an Investigational New Drug submission and clinical trial initiation which are aligned with Affinia's purpose to make a lasting positive impact in the lives of people affected by devastating rare and prevalent diseases where the genetic cause is understood,' said Rick Modi, Affinia's Chief Executive Officer. About Affinia Therapeutics Affinia Therapeutics is pioneering a 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 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 About DCM Foundation Founded in 2018, the DCM Foundation's mission is to provide hope and support to DCM patients and families with dilated cardiomyopathy through education, research and advocacy. Our mission is being executed through three foundational pillars: information and education, patient and family support, and understanding the need for genetic testing. In 2023, DCMF created the Genetic Cardiomyopathy Awareness Consortium, comprised of 11 patient group members, to address the extreme lack of knowledge about genetics and genetic testing in the cardiomyopathy patient and medical community. For more information, visit and View source version on CONTACT: Media contacts:For Affinia Kathy Vincent [email protected] 310-403-8951For DCM Foundation Buddy Butler [email protected] 304-615-9884 KEYWORD: MASSACHUSETTS OHIO UNITED STATES NORTH AMERICA INDUSTRY KEYWORD: RESEARCH NEUROLOGY GENETICS CARDIOLOGY PHILANTHROPY BIOTECHNOLOGY PHARMACEUTICAL HEALTH FOUNDATION SCIENCE SOURCE: Affinia Therapeutics Copyright Business Wire 2025. PUB: 06/03/2025 07:45 AM/DISC: 06/03/2025 07:44 AM
Business Wire
5 days ago
- Business
- Business Wire
Affinia Therapeutics and DCM Foundation Announce Partnership to Increase Awareness About BAG3 Dilated Cardiomyopathy and Critical Need for Genetic Testing to Help Save Lives
WALTHAM, Mass. & DUBLIN, Ohio--(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 for devastating cardiovascular and neurological diseases, and the DCM Foundation together with the Genetic Cardiomyopathy Awareness Consortium (GCAC), today announced they have joined forces to raise awareness about BAG3 dilated cardiomyopathy (DCM), with the goal of promoting early diagnosis and the critical need for genetic testing. 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 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. According to a study published in Circulation, the journal of the American Heart Association, close to 50% of cardiomyopathy has some type of genetic basis, such as BAG3 DCM, yet only a fraction of diagnosed cardiomyopathy patients get genetic testing. This new-found partnership between Affinia and the DCM Foundation and GCAC aims to educate about BAG3 DCM and the critical need for genetic testing, and champions the needs and voices of people living with this devastating heart disease through initiatives including: BAG3 Patient Advisory Council to offer insight and feedback into patient needs and include the patient voice from people living with BAG3 DCM to inform research and clinical trial design. BAG3 DCM Webinar to help educate about genetics and cardiovascular disease, and the latest advances in research and development, with a focus on Affinia's pipeline and lead program for BAG3 DCM, AFTX-201. AFTX-201 is a potential best-in-class investigational AAV gene therapy intended to be given as a simple one-time intravenous injection. Genetic testing for BAG3 DCM to improve the diagnosis and management of patients affected with this devastating disease. 'The DCM Foundation and GCAC are very grateful for this partnership with Affinia,' said Greg Ruf, Founder and Executive Director, the DCM Foundation. 'By getting more cardiomyopathy patients tested, we can potentially save and improve lives and help advance cardiomyopathy research and therapies. Through this collaboration, we will collectively unite our strengths and work together in the hope of making a real difference for those living with this devastating disease.' Hideo Makimura, M.D., Ph.D., Chief Medical Officer of Affinia, commented, 'BAG3 DCM is a devastating heart disease with a known genetic cause. Unfortunately, only a fraction of patients affected with BAG3 DCM and other genetic cardiomyopathies are tested, which is putting lives at risk. We are committed to working together with the DCM Foundation and GCAC to increase disease awareness and the role genetics plays in cardiomyopathy, which we believe will ultimately lead to better outcomes for people living with BAG3 DCM.' 'Our partnership with the DCM Foundation and GCAC is an exciting milestone as we advance our lead program, AFTX-201 for BAG3 DCM, toward an Investigational New Drug submission and clinical trial initiation which are aligned with Affinia's purpose to make a lasting positive impact in the lives of people affected by devastating rare and prevalent diseases where the genetic cause is understood,' said Rick Modi, Affinia's Chief Executive Officer. About Affinia Therapeutics Affinia Therapeutics is pioneering a 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 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 About DCM Foundation Founded in 2018, the DCM Foundation's mission is to provide hope and support to DCM patients and families with dilated cardiomyopathy through education, research and advocacy. Our mission is being executed through three foundational pillars: information and education, patient and family support, and understanding the need for genetic testing. In 2023, DCMF created the Genetic Cardiomyopathy Awareness Consortium, comprised of 11 patient group members, to address the extreme lack of knowledge about genetics and genetic testing in the cardiomyopathy patient and medical community. For more information, visit and
Associated Press
28-04-2025
- Business
- Associated Press
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 View source version on CONTACT: Media contact: Kathy Vincent [email protected] 310-403-8951 KEYWORD: UNITED STATES NORTH AMERICA LOUISIANA MASSACHUSETTS INDUSTRY KEYWORD: RESEARCH NEUROLOGY GENETICS CLINICAL TRIALS CARDIOLOGY BIOTECHNOLOGY HEALTH PHARMACEUTICAL SCIENCE SOURCE: Affinia Therapeutics Copyright Business Wire 2025. PUB: 04/28/2025 05:14 PM/DISC: 04/28/2025 05:14 PM
