hTFR · BBB DELIVERY

hTFR macromolecule brain delivery

Use hTFR humanized mice to connect peripheral dosing, brain distribution and target engagement for TfR1-targeted macromolecules.

Macromolecules do not readily cross the blood-brain barrier. TfR1-mediated transport is a major delivery strategy, but brain distribution varies by format, route and dose and should be measured directly. Our hTFR platform connects peripheral dosing, brain imaging and multi-region qPCR quantification.

  • IV · SCPeripheral dosing
  • 8+ brain regionsDistribution & quantification
  • hTFR × 5xFADOptional composite model
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Service positioning

For comparing TfR1-targeted macromolecule delivery and, where needed, linking it to disease-model efficacy.

  • hTFR humanized mouse
  • IV / SC peripheral dosing
  • 8+ brain regions and peripheral tissues
  • Composite models such as hTFR × 5xFAD

When teams usually need it

When in vitro binding is complete and peripheral-to-CNS delivery needs confirmation; when formats or routes must be compared; or when established delivery needs to connect with disease efficacy.

Research questions

01How is the candidate distributed across brain regions after peripheral dosing?

02How do dosing routes change target engagement?

03Can delivery performance connect to efficacy in an indication-specific model?

Study design

Choose an experiment combination around the research question. Steps, groups and timing are confirmed per project.

  1. 01

    Candidate & dosing

    Align format, dose, route and observation time.

  2. 02

    Brain distribution

    Assess cortex, hippocampus, thalamus and peripheral tissues.

  3. 03

    Target engagement

    Measure target-gene or protein changes with qPCR and ELISA.

  4. 04

    Disease-model validation

    Select an indication-specific model for continued efficacy testing.

Models & endpoints

See how models map to endpoints, then open the topic page for model scope and representative evidence.

Representative models
  • hTFR humanized mouse
  • hTFR × 5xFAD
Primary endpoints
  • Distribution across cortex, hippocampus and thalamus
  • Peripheral-tissue comparison
  • qPCR target-gene quantification
  • ELISA protein-effect assay

DELIVERABLES

Data & delivery

  • Brain-region distribution images and quantification
  • Target-gene or protein effects
  • Route and group comparisons
  • Delivery-to-efficacy analysis

Deliverables are confirmed against the actual study scope and experiment combination.

Technical contact

Tell us your candidate type, study goal and development stage. We will help identify the experiment combination for hTFR macromolecule brain delivery.