AAVrh.10hC1EI

AAVrh.10hC1EI is an adeno-associated virus (AAV) gene therapy candidate utilizing the rh.10 serotype to deliver a functional copy of the human C1 esterase inhibitor (C1-INH) gene. It is primarily being developed for the treatment of Hereditary Angioedema (HAE), a genetic disorder characterized by recurrent episodes of severe swelling due to deficiency or dysfunction of the C1 esterase inhibitor protein. The vector is designed for systemic administration to achieve sustained hepatic expression and secretion of C1-INH into the circulation, thereby regulating the complement and contact systems. Research presented at ASGCT 2026 highlighted potential safety considerations, specifically identifying rapid and transient complement activation (measured by terminal complement complex levels) following repeat administration in the presence of pre-existing anti-AAVrh.10 antibodies.

Other names

AAVrh.10 expressing human C1 esterase inhibitorAAVrh.10-hC1EI