The promise of viral vector‐based gene therapy (GT) as a transformative paradigm for
treating severely debilitating and life‐threatening diseases is slowly coming to fruition with …

Model‐informed approaches provide a quantitative framework to integrate all available
nonclinical and clinical data, thus furnishing a totality of evidence approach to drug …

Live biotherapeutic products (LBPs) are human microbiome therapies showing promise in
the clinic for a range of diseases and conditions. Describing the kinetics and behavior of …

Introduction Sickle cell disease (SCD) is a monogenic disorder that exerts several
detrimental health effects on those affected, ultimately resulting in significant morbidity and …

In this chapter, we envision the future of Quantitative Systems Pharmacology (QSP) which
integrates closely with emerging data and technologies including advanced analytics, novel …

Endothelial dysfunction and chronic anaemia are key features of sickle cell disease (SCD),
which significantly increases the risk of vaso-occlusive crises, pulmonary hypertension and …

In the last 50 years, pain in Sickle Cell Disease (SCD) has become more widely studied
thanks to advancements in technology and awareness. Clinical studies, population surveys …

The differentiation of mammalian hematopoietic stem cells (HSCs) is complex and
multiscale, providing an opportunity for mathematical modeling and simulation to aid in …