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Gene Therapy Breakthrough for Rare Heart Disease
23 Jul
Summary
- FDA grants orphan drug designation for AFTX-201 gene therapy.
- AFTX-201 targets BAG3-associated dilated cardiomyopathy (DCM).
- UPBEAT clinical trial is actively recruiting participants.
Affinia Therapeutics has secured orphan drug designation from the US Food and Drug Administration (FDA) for its investigational gene therapy, AFTX-201. This designation is for the treatment of BAG3-associated dilated cardiomyopathy (DCM), a serious genetic condition affecting the heart muscle.
AFTX-201 is designed to address the root cause of BAG3 DCM, a disease characterized by reduced BAG3 protein production. The gene therapy utilizes a functional BAG3 transgene delivered through a single intravenous infusion, employing a proprietary capsid for enhanced cardiac targeting at reduced doses.
The UPBEAT clinical trial, a Phase I/II study, is currently recruiting participants aged 18 to 55 in the US and Canada. The trial specifically seeks individuals diagnosed with BAG3 DCM who experience limitations in daily physical activities due to their heart condition.
Orphan drug designation is granted by the FDA to encourage the development of treatments for rare diseases. It offers potential incentives such as tax credits and market exclusivity, supporting therapies like AFTX-201 aimed at conditions like BAG3 DCM, which impacts an estimated 2.3%-3.6% of DCM cases globally.
