Dose Optimization for Gene Therapy Using QSP Modeling

Time: 8:25 am
day: Day Two


Quantitative Systems Pharmacology (QSP) is proving to be an invaluable, especially for novel modalities such as gene therapy and the technology has increasingly been accepted by regulators

A QSP model related the AAV vectors to the affected organ and PK to plasma level to optimize dosing for rare disease gene therapy

The model enabled the simulation of a range of virtual patients, including different ages, ethnicities and body mass