Mechanistic Modeling to support Translation of mRNA Therapies from Discovery to Clinic

Time: 12:20 pm
day: Day 1 Track B AM


  • 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