Affinia Therapeutics Receives European Medicines Agency Orphan Drug Designation for AFTX-201 in BAG3-Associated Dilated Cardiomyopathy (DCM)

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WALTHAM, Mass.--(BUSINESS WIRE)--Feb 18, 2026--

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 the European Medicines Agency (EMA) has granted Orphan Drug designation to Affinia for AFTX-201. AFTX-201 is a potential first-in-class and best-in-class investigational genetic medicine for the treatment of BAG3-associated dilated cardiomyopathy (DCM).

“BAG3 DCM is an inherited heart disease with a high mortality rate that results in loss of heart function at a young age,” said Fernando Dominguez, M.D., Ph.D., a cardiologist at the Heart Failure and Inherited Cardiac Diseases Unit, Department of Cardiology, Hospital Universitario Puerta de Hierro Majadahonda, Madrid, Spain. “Despite current standard of care, there are no disease-modifying treatments available and nearly 25% of patients require a heart transplant. A one-time therapy that addresses the genetic root cause of BAG3 DCM could be transformative and make a real difference for those living with BAG3 DCM.”

“The EMA Orphan Drug designation indicates the importance of developing new treatment options for rare diseases such as BAG3 DCM,” said Hideo Makimura, M.D., Ph.D., Chief Medical Officer at Affinia. “This designation, along with the recent FDA acceptance of our IND application for AFTX-201, reinforces our belief that our investigational medicine has the potential to be the best-in-class treatment option for those affected by this devastating disease.”

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 therapy products using conventional capsids such as AAV9 or AAVrh74. AFTX-201 is intended to be given as a simple one-time intravenous administration. Preclinical studies in an animal disease model demonstrated that AFTX-201 increased BAG3 protein levels in the heart and completely restored cardiac function.

The EMA grants Orphan Drug designation to encourage the development of medicines intended for the treatment of life-threatening or chronically debilitating rare conditions affecting fewer than 5 in 10,000 individuals in the European Union. This designation provides several incentives, including protocol assistance, 10-year market exclusivity upon marketing authorization, fee reductions, and other financial incentives.

About BAG3 DCM

BAG3 dilated cardiomyopathy (DCM) is a serious, inherited heart condition with a high mortality and a significant unmet medical need. The disease affects more than 70,000 patients in the Canada, E.U., U.S., and U.K. 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 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.

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KEYWORD: EUROPE UNITED STATES NORTH AMERICA MASSACHUSETTS

INDUSTRY KEYWORD: RESEARCH FDA GENETICS CLINICAL TRIALS CARDIOLOGY BIOTECHNOLOGY HEALTH PHARMACEUTICAL SCIENCE

SOURCE: Affinia Therapeutics

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PUB: 02/18/2026 07:45 AM/DISC: 02/18/2026 07:47 AM

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