Affinia Therapeutics has received the US Food and Drug Administration (FDA) orphan drug designation for AFTX-201, a treatment in development for BAG3-associated dilated cardiomyopathy (DCM).
The gene therapy is under investigation in adults living with BAG3 DCM, a genetic condition that leads to progressive heart failure and reduced survival despite existing treatments.
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AFTX-201 is an investigational gene therapy delivered through a single intravenous infusion.
The therapy uses a functional BAG3 transgene and is based on the company’s proprietary capsid, which is engineered for targeted delivery to the heart at lower doses than some traditional gene therapies.
The investigational therapy is currently being studied in the Phase I/II UPBEAT clinical trial in the US and Canada.
The trial is recruiting participants aged 18 to 55 years who are diagnosed with BAG3 DCM and have difficulties with daily physical activities due to heart failure.
Affinia Therapeutics chief medical officer Hideo Makimura said: “Receiving orphan drug designation, in addition to the recent fast track designation, from the FDA marks a significant achievement for the AFTX-201 programme and we thank the FDA for their decision.
“AFTX-201 is designed to address the underlying root cause of BAG3 DCM. The UPBEAT clinical trial is actively recruiting at multiple institutions, and we look forward to continue progressing the AFTX-201 clinical programme so that we can bring this potentially transformative treatment to people living with BAG3 DCM.”
Caused by genetic changes that limit BAG3 protein production in heart muscle cells, BAG3-associated DCM is estimated to account for 2.3%-3.6% of DCM cases globally, based on published literature.
The FDA’s orphan drug designation is intended to support the development of treatments for rare diseases in the US.
It may offer development and commercial incentives such as tax credits, exemption from user fees, and potential market exclusivity if the therapy receives approval.
In October 2025, Affinia Therapeutics raised $40m in a Series C funding round, led by New Enterprise Associates.
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