New horizons of model informed drug development in rare diseases drug development

A Mitra, N Tania, MA Ahmed, N Rayad… - Clinical …, 2024 - Wiley Online Library
Model‐informed approaches provide a quantitative framework to integrate all available
nonclinical and clinical data, thus furnishing a totality of evidence approach to drug …

PBPK Modeling: Empowering Drug Development and Precision Dosing in China

D Yang, J Li, WY Mak, A Zheng, X Zhu… - CPT …, 2025 - Wiley Online Library
Physiologically based pharmacokinetic (PBPK) modeling, a cornerstone of model‐informed
drug development and model‐informed precision dosing, simulates drug disposition in the …

Translational pharmacokinetic/pharmacodynamic model for mRNA‐0184, an investigational therapeutic for the treatment of heart failure

N Kaushal, H Attarwala, MJ Iqbal… - Clinical and …, 2024 - Wiley Online Library
Heart failure (HF) is a complex, progressive disorder that is associated with substantial
morbidity and mortality on a global scale. Relaxin‐2 is a naturally occurring hormone that …

A quantitative systems pharmacology (QSP) platform for preclinical to clinical translation of in-vivo CRISPR-Cas therapy

DA Desai, S Schmidt, R Cristofoletti - Frontiers in Pharmacology, 2024 - frontiersin.org
Background: In-vivo CRISPR Cas genome editing is a complex therapy involving lipid
nanoparticle (LNP), messenger RNA (mRNA), and single guide RNA (sgRNA). This novel …

Translational PK–PD model for in vivo CAR‐T‐cell therapy delivered using CAR mRNA‐loaded polymeric nanoparticle vector

SJ Kim, GM Mugundu, AP Singh - Clinical and Translational …, 2024 - Wiley Online Library
Autologous chimeric antigen receptor (CAR) T‐cell therapy has demonstrated remarkable
response rates, yet its widespread implementation is hindered by logistical, financial, and …

Future Directions for Quantitative Systems Pharmacology

B Schoeberl, CJ Musante, S Ramanujan - 2025 - Springer
In this chapter, we envision the future of Quantitative Systems Pharmacology (QSP) which
integrates closely with emerging data and technologies including advanced analytics, novel …