Modified Adeno-associated virus capsid VF18 (AAV-VF18)

Modified Adeno-associated virus capsid VF18 (AAV-VF18) is a synthetic, engineered viral protein shell designed for optimized gene delivery to the liver (Vivet Therapeutics, 2023). It was developed using capsid engineering techniques, such as DNA shuffling, to create a chimeric vector with enhanced hepatocyte transduction efficiency compared to naturally occurring serotypes like AAV8 (Patent WO2019012082A1). This capsid is the primary delivery vehicle for VTX-801, an investigational gene therapy for Wilson disease, which carries a functional ATP7B transgene to restore copper metabolism (ClinicalTrials.gov, NCT04537377). By improving liver tropism, AAV-VF18 allows for potentially lower therapeutic doses, which may reduce the risk of systemic immune activation. However, like other AAV vectors, its efficacy can be limited by pre-existing neutralizing antibodies in the patient population. Monitoring of capsid-specific immune responses and liver function is essential during clinical application to ensure safety and durability of the transgene expression.

Other names

AAV-VF18VF18 capsidEngineered Adeno-associated virus capsid VF18Chimeric AAV capsid VF18

Mediates the delivery of a therapeutic transgene to hepatocytes by binding to cell surface receptors, undergoing endocytosis, and transporting the genetic payload to the nucleus for expression.