July 26th - 28th | Boston, MA
Applied BioMath Presence:
Day and Time: Friday, July 28th | 2:30 pm
Title: Applications of a QSP Model of LNP mRNA Delivery for the Treatment of Crigler-Najjar Syndrome Type 1
- 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.
Presenter: Diana Marcantonio, PhD, Director of Biology
This is a Hanson Wade conference.