摘要：

• Crigler-Najjar syndrome type 1 (CN1) is an autosomal recessive disease caused by a marked decrease in uridine-diphosphate-glucuronosyltransferase (UGT1A1) enzyme activity with limited treatment options
• In this talk, we will discuss the application of quantitative systems pharmacology (QSP) modeling to the design and development of an LNP-delivered mRNA gene therapy for the treatment of CN1
• We will cover two case studies, how the “LNP-encapsulated mRNA targeting an intracellular protein in vivo” model in Applied BioMath Assess ™ was used to explore key design features for an LNP-delivered mRNA gene therapy, and how a similar QSP model was developed and calibrated to data from a Gunn rat animal model and used for preclinical-to-clinical translation and FIH dose projections

讲师： Diana Marcantonio, PhD, Senior Director, QSP