modelBetibeglogeneAutotemcel

Diagram of BetibeglogeneAutotemcel

Extends from Pharmacolibrary.Drugs.ATC.B.B06AX02.

Information

name:BetibeglogeneAutotemcel
ATC code:B06AX02
route:intravenous
compartments:0
dosage:1mg
volume of distribution:1L
clearance:0
other parameters in model implementation

Betibeglogene autotemcel (also known as Zynteglo) is a gene therapy used for the treatment of beta-thalassemia in patients who require regular red blood cell transfusions. It delivers a functional copy of the beta-globin (HBB) gene into the patient's hematopoietic stem cells to enable normal hemoglobin production. The drug was approved for medical use but production and commercialization have been discontinued in some regions.

Pharmacokinetics

There are no published reports of pharmacokinetic parameters for betibeglogene autotemcel in the literature, as gene therapies involving autologous cell infusions are not described by traditional pharmacokinetic models (e.g., absorption, distribution, clearance) that apply to small molecules or biologics.

References

    Parameters

    TypeNameDefaultDescription
    Modelica.Units.SI.Massweight (from PK_1C)75patient weight (kg)
    Modelica.Units.SI.SpecificVolumeVdPerKg (from PK_1C)0.9Volume of distribution (L/kg)
    Modelica.Units.SI.MassFractionF (from PK_1C)0.8bioavailiability (0-1)
    Pharmacolibrary.Types.ClearanceCl (from PK_1C)20clearance
    Modelica.Units.SI.TimeadminTime (from PK_1C)60first administration time (s)
    Modelica.Units.SI.TimeadminDuration (from PK_1C)600administration duration (s)
    Modelica.Units.SI.TimeadminPeriod (from PK_1C)8*60*60period of administration (default 8 hours)(s)
    Pharmacolibrary.Types.MassadminMass (from PK_1C)1000administration mass (mg)
    IntegeradminCount (from PK_1C)8number of dose administered (1)
    Pharmacolibrary.Types.VolumeVd (from PK_1C)VdPerKg*weightVolume of distribution (m3)
    Pharmacolibrary.Types.MassConcentrationCmin (from PK_1C)0.004minimal therapeutic range
    Pharmacolibrary.Types.MassConcentrationCmax (from PK_1C)0.008minimal therapeutic range
    Pharmacolibrary.Types.MassConcentrationCtox_peak (from PK_1C)0.012toxicity peak level
    Pharmacolibrary.Types.MassConcentrationCtox_trough (from PK_1C)0.006toxicity trough level

    Connectors

    TypeNameDefaultDescription
    Types.ConcentrationOutputC_central (from PK_1C)
    Interfaces.ConcentrationPort_bcentralCPort (from PK_1C)

    Components

    TypeNameDefaultDescription
    Pharmacokinetic.NoPerfusedTissueCompartmentcentral (from PK_1C)
    Pharmacokinetic.ClearanceDrivenEliminationelim (from PK_1C)
    Sources.PeriodicDoseperiodicDose (from PK_1C)
    Modelica.Units.SI.Timet1_2 (from PK_1C)elimination half-life

    Revisions

    • 06/2025 Tomas Kulhanek, generated model from data extracted from PUBMED, DrugBank and LLM(GPT4.1)