AAVrh.10

AAVrh.10 is a non-human primate-derived serotype of the adeno-associated virus (AAV) that serves as a highly efficient viral vector platform for gene therapy. Originally isolated from rhesus macaques, AAVrh.10 is distinguished by its robust tropism for the central nervous system (CNS), liver, heart, and skeletal muscle. It is particularly noted for its ability to cross the blood-brain barrier and transduce neurons and glial cells more effectively than many other AAV serotypes, such as AAV2 or AAV9, in certain preclinical models. This vector is utilized across a variety of therapeutic programs to deliver functional genes for the treatment of neurodegenerative and metabolic disorders, including Alzheimer's disease, Batten disease (CLN2), and Friedreich's ataxia. Clinical challenges associated with AAVrh.10 include pre-existing anti-capsid immunity in the human population and the potential for rapid complement activation following systemic administration, which can impact both safety and efficacy.

Other names

rhesus-derived adeno-associated virus serotype 10AAV serotype rh.10