A Systems Model of Gene Therapy for Sickle Cell Disease

• 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 model was validated with published clinical data.

• 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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