A Systems Model of Gene Therapy for Sickle Cell Disease

Webinar Details

Recorded on Tuesday, November 30th, 2021

Speaker: Raibatak Das, PhD, Principal Scientist at Applied BioMath

Duration: 1 Hour

Sickle cell disease (SCD) is an inherited genetic disease of the blood with no known cure at this time. Stem cell gene therapy is an emerging experimental therapy for SCD with the potential for lifelong cure but it is an expensive multi-step treatment regimen with several months to over a year before treatment stabilization.

We developed a quantitative systems pharmacology (QSP) model to predict how varying treatment parameters such as stem cell dose and vector copy number will affect post-treatment hemoglobin and red blood cell dynamics after autologous stem cell gene therapy. The QSP approach used here can guide rational therapeutic design of gene therapy for SCD and other genetic disorders.

This webinar is ideal for scientists and decision makers in drug R&D who want to leverage systems pharmacology for drug discovery and development.


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