Neurology & neurodegeneration PBPKQSPTMDD

Whole-body AAV–anti-TargetX rat brain QSP

Reference paper ↗

Mechanistic rat QSP coupling whole-body AAV biodistribution, intracellular transduction, transgene-derived anti-TargetX mAb, FcRn-aware protein PBPK, TargetX binding, and detailed brain/CSF transport across 725 explicit scalar states.

Therapeutic
AAV9/AAV5-encoded anti-TargetX monoclonal antibody
Modality
Gene-delivered antibody
Target
TargetX (anonymized paper target)
Disease
Preclinical CNS antibody delivery (not disease-specific)
Model type
PBPK, QSP, TMDD

Complete model workspace

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Parameters
325
States
725
Equations
725
Derived outputs
27

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Exogenous TargetX disposition

Unit-normalized six-hour exogenous TargetX bolus disposition. The source does not disclose the administered amount, so only the population-fit curve shape and reported 1.4 h half-life are compared.

Starting result

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Starting configuration

Time: 0–6 hour; step 0.1

InputDefaultAvailable range
AAV degradation rate1.007 1/h0.5035–1.511 1/h
Antibody permeability–surface area coefficient2.62e-7 L/h1.31e-7–3.93e-7 L/h

Expected readouts

Free target x in plasma (pM)

Adjustable model parameters

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How the model represents the biology

Mechanistic rat QSP linking IV, intracisterna-magna, or intrastriatal AAV delivery to whole-body vector disposition, transgene-derived anti-TargetX antibody, FcRn-aware protein distribution, and free TargetX in plasma, brain ISF, and CSF.

How do AAV disposition, transduction, and transgene-derived antibody connect to TargetX binding across the full rat-brain QSP model?Route-specific AAV enters whole-body and brain disposition, undergoes receptor-mediated cellular uptake and nuclear delivery, and drives delayed transgene expression. Secreted antibody distributes across plasma, tissues, brain, and cerebrospinal fluid, where it binds local free TargetX reversibly. Direct intravenous antibody and TargetX calibration inputs bypass transduction.BIOLOGY OVERVIEWHow do AAV disposition, transduction, and transgene-derived antibody connect toTargetX binding across the full rat-brain QSP model?AAV, mAb, or TargetXinputRoute-specific scenariobranchesAAV dispositionWhole body, brain and CSFCell uptake andnuclear vectorReceptor uptake to nuclearburdenDelayed transgeneexpressionThree-stage expression andsecretionAnti-TargetX mAbdispositionPlasma, tissues, brain and CSFFree TargetX poolsLocal target synthesis andturnoverLocal mAb–TargetXcomplexesReversible compartmentalbindingPRIMARY READOUTSPlasma, brain and CSF antibodyPlasma, brain and CSF anti…Free TargetXFree TargetXmAb–TargetX complexmAb–TargetX complexNuclear vector and expressionNuclear vector and express…

Solid arrows show modeled movement or change; two-headed arrows show reversible exchange; dashed arrows show modulation without material transfer.

Model scope: This overview intentionally does not reproduce the validated 725-state topology. Blood-cell AAV modules do not produce transgene-derived antibody; direct IV mAb and TargetX are calibration branches; TargetX is anonymized; and disease efficacy or toxicity is not represented.

Modeled relationships (10)
  • AAV, mAb, or TargetX input → AAV disposition: IV / ICM / IST AAV (flow)
  • AAV disposition → Cell uptake and nuclear vector: uptake and nuclear delivery (flow)
  • Cell uptake and nuclear vector → Delayed transgene expression: three expression delays (production)
  • Delayed transgene expression → Anti-TargetX mAb disposition: mAb secretion (production)
  • AAV, mAb, or TargetX input → Anti-TargetX mAb disposition: direct IV mAb calibration (flow)
  • AAV, mAb, or TargetX input → Free TargetX pools: direct IV target calibration (flow)
  • Anti-TargetX mAb disposition → Local mAb–TargetX complexes: local kon binding (production)
  • Free TargetX pools → Local mAb–TargetX complexes: binding consumes target (flow)
  • Local mAb–TargetX complexes → Anti-TargetX mAb disposition: koff returns mAb (flow)
  • Local mAb–TargetX complexes → Free TargetX pools: koff returns TargetX (flow)

Modeled relationships: AAV, mAb, or TargetX input to AAV disposition: IV / ICM / IST AAV (flow); AAV disposition to Cell uptake and nuclear vector: uptake and nuclear delivery (flow); Cell uptake and nuclear vector to Delayed transgene expression: three expression delays (production); Delayed transgene expression to Anti-TargetX mAb disposition: mAb secretion (production); AAV, mAb, or TargetX input to Anti-TargetX mAb disposition: direct IV mAb calibration (flow); AAV, mAb, or TargetX input to Free TargetX pools: direct IV target calibration (flow); Anti-TargetX mAb disposition to Local mAb–TargetX complexes: local kon binding (production); Free TargetX pools to Local mAb–TargetX complexes: binding consumes target (flow); Local mAb–TargetX complexes to Anti-TargetX mAb disposition: koff returns mAb (flow); Local mAb–TargetX complexes to Free TargetX pools: koff returns TargetX (flow).

Primary readouts: Plasma, brain and CSF antibody; Free TargetX; mAb–TargetX complex; Nuclear vector and expression.

Found a scientific issue? Email helpdesk@unibiointelligence.com with model ID liu_2026_aav_mab_targetx_rat_brain_full_qsp.

Research use only — not for patient-specific prediction or dosing advice.

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