Session Details

The first-in-human dose for clinical trials for AAV therapeutics are expected to be both safe and have prospect of therapeutic benefit. The choice of first-in-human dose and dose-ranges evaluated in the clinical study designs should be supported by both preclinical studies and/or available clinical data. Regulatory agencies recognize that the data obtained from animal models and in vitro studies may be the only way to estimate a starting human dose and encourage the use of the most appropriate and predictive models. This session will describe dose scaling from preclinical models to the clinic for locally administered CNS and ocular AAV gene therapies as well as systemically administered AAV gene therapies.