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Gene Therapy Model Pack

What’s Included?

Applied BioMath Assess™ Gene Therapy Model Pack includes 8 models covering LNP-encapsulated mRNA and siRNA therapies.

  • LNP-encapsulated mRNA expressing an intracellular protein in vitro
    • Two-compartment model of mRNA uptake, protein expression, and protein-mediated enzymatic reaction in vitro
  • LNP-encapsulated mRNA expressing an intracellular protein in vivo
    • Two-compartment model of mRNA delivery, protein expression, and protein-mediated enzymatic reaction in vivo
  • LNP-encapsulated mRNA expressing an extracellular protein in vitro
    • Two-compartment model of mRNA delivery, protein expression, and protein-mediated enzymatic reaction in vitro
  • LNP-encapsulated mRNA expressing an extracellular protein in vivo
    • Two-compartment model of mRNA delivery, protein expression, and protein-mediated enzymatic reaction in vivo
  • LNP- encapsulated siRNA expressing an intracellular protein in vitro
  • LNP-encapsulated siRNA expressing an intracellular protein in vivo 
  • LNP-encapsulated siRNA expressing an extracellular protein in vitro 
  • LNP-encapsulated siRNA expressing an extracellular protein in vivo

Model Resources

Each model in the Gene Therapy Model Pack includes a pharmacology diagram (Figure 1) with model parameters and their nominal values (Figure 2).

Figure 1: Model Diagrams

Figure 1: pharmacology diagram
Created in BioRender.com

Figure 2: Model Parameters

Each model in Applied BioMath Assess™ Gene Therapy Model Pack has a unique set of parameters, a sample of which is shown here, based on the model pharmacology and number of compartments. More details on the full list of parameters are available upon request. In the app, each parameter contains a default value based on expansive literature searches and references.

Figure 2: model parameters and their nominal values

Webinar

Applications of a QSP Model of LNP mRNA Delivery for the Treatment of Crigler-Najjar Syndrome Type 1

Additional Model Packs

18 models covering 9 common pharmacologies.

Models covering both soluble and membrane ligand pharmacologies.

Models for solid tumors, in-vitro experiments, and solid tumors with 2 cell types.

4 models covering in vivo and in vitro models of PROTAC and Molecular Glue Degrader pharmacologies.

Includes 42 models covering monospecific and bispecific ADCs with and without avidity.

Gene Therapy Model Pack

Complete the form to receive your copy of the Gene Therapy Model Pack overview.

Support

On-Demand Support

Have any questions about Applied BioMath Assess or interested in seeing a demo? We have software engineers ready to chat!

Contact us today at abm-support@certara.com.

Documentation

Visit our Applied BioMath Assess documentation portal for product documentation as well as video case studies.