Rational prediction of human dose for gene therapy based on translational dose-response studies

Establishing dose response relationships is foundational in drug development. To improve confidence in a development program, understanding dose response in animals and how that translates to early clinical development studies is critical. Traditional approaches to predict clinical doses that achieve minimal biological activity and maintain wide safety margins may not apply to gene therapy (GT) because of differences in biological mechanisms of drug disposition and pharmacological activity when compared with more established drug modalities.