Cardiomyocyte cell surface receptor for ATC-0187 AAV capsid

The cardiomyocyte cell surface receptor for the ATC-0187 AAV capsid is a novel, proprietary protein identified by Affinia Therapeutics through its ART (Affinia Rational Targeting) platform. This receptor is highly and selectively expressed on the surface of cardiomyocytes, where it serves as the primary binding and entry point for the ATC-0187 capsid, a second-generation cardiotropic adeno-associated virus (AAV) vector. By targeting this specific receptor, the ATC-0187 capsid achieves significantly higher transduction efficiency and more uniform distribution in the heart compared to conventional capsids like AAV9, which rely on less specific interactions with galactose and AAVR. Preclinical studies in non-human primates have demonstrated that this receptor-mediated approach can transduce 90-100% of cardiomyocytes following a single intravenous dose. This high efficiency allows for the delivery of therapeutic genes, such as BAG3 or MYBPC3, at doses 5-10 times lower than standard AAV therapies, thereby reducing the risk of dose-limiting toxicities like liver injury and complement activation. The receptor is a critical component of Affinia's lead gene therapy programs, including AFTX-201 for BAG3-associated dilated cardiomyopathy and ATC-018-MYBPC3 for hypertrophic cardiomyopathy. It represents a significant advancement in the field of cardiotropic gene delivery, enabling the treatment of both rare and prevalent cardiovascular diseases through precise tissue targeting.

Other names

ATC-0187 receptorNovel cardiotropic AAV receptorAffinia cardiotropic receptorCardiomyocyte-specific AAV entry receptor

Mediates selective and efficient receptor-mediated endocytosis and viral entry of the ATC-0187 AAV capsid into cardiomyocytes, facilitating high-level transgene expression at low systemic doses.