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A Systems Model of Gene Therapy for Sickle Cell Disease
Register for Your Free Live Webinar Now:

"A Systems Model of Gene Therapy for Sickle Cell Disease"

Tuesday, November 30, 2021 at 2PM ET / 11AM PT
Learn how we developed a systems model of gene therapy for sickle cell disease to predict the impact of treatment variables such as stem cell dose, vector copy number and bone marrow preconditioning on observed clinical readouts of treatment efficacy.

• 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.


Offered Free by: Applied Biomath
See All Resources from: Applied Biomath

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