hTFR · BBB delivery

hTFR macromolecule brain delivery

Macromolecules do not readily cross the blood-brain barrier. TfR1-mediated transport can improve delivery, but distribution must be measured directly across formats, routes and doses.

hTFR mice humanize the key human TfR1 binding domain and support IV or SC evaluation of AOCs, bispecifics, ADCs and other macromolecules, with route controls in the same study.

The platform connects dosing, brain-distribution imaging, multi-region qPCR and target engagement, with optional composite disease models.

8+Brain regions
IV · SCPeripheral routes
Full chainDosing → distribution → qPCR → efficacy

Discuss a model study

Model strategy

Connect TfR1-targeted antibody and protein delivery with brain-region distribution and target engagement.

Match the model and study conditions to candidate mechanism, intervention stage and target tissue.

hTFR humanized mouse

Distribution and effect after IV / SC dosing

hTFR × 5xFAD

Delivery effect linked to AD efficacy

Evaluation endpoints

01Distribution across cortex, hippocampus and thalamus
02Peripheral-tissue comparison
03qPCR target-gene quantification
04ELISA protein-effect assay

Representative data

Brain distribution map | Fluorescence in situ hybridization distribution of AOC / siRNA in hTFR mice. After peripheral administration of AOC-IV and AOC-SC, fluorescence in situ hybridization in four brain regions (AS, CTX, HIP, TH) of hTFR humanized mice showed broad AOC/siRNA distribution, with an effect comparable to the siRNA-IT intrathecal positive control, while the PBS-IV negative control showed no signal. — 01
Brain distribution map | Fluorescence in situ hybridization distribution of AOC / siRNA in hTFR mice
After peripheral administration of AOC-IV and AOC-SC, fluorescence in situ hybridization in four brain regions (AS, CTX, HIP, TH) of hTFR humanized mice showed broad AOC/siRNA distribution, with an effect comparable to the siRNA-IT intrathecal positive control, while the PBS-IV negative control showed no signal.

hTFR mice · Target gene knockdown. After intravenous AOC administration in hTFR humanized mice, relative target gene mRNA expression (RT-qPCR, D14) in the cortex, hippocampus, thalamus, brainstem, spinal cord and cerebellum as well as heart, liver and quadriceps was significantly reduced compared with the WT Vehicle and hTFR Vehicle controls. — 02
hTFR mice · Target gene knockdown
After intravenous AOC administration in hTFR humanized mice, relative target gene mRNA expression (RT-qPCR, D14) in the cortex, hippocampus, thalamus, brainstem, spinal cord and cerebellum as well as heart, liver and quadriceps was significantly reduced compared with the WT Vehicle and hTFR Vehicle controls.

hTFR×5xFAD · APP target gene knockdown. RT-qPCR (cortex, hippocampus, spinal cord, liver) and ELISA (cortex, hippocampus, DRG, liver sAPPα/sAPPβ) results for APP target gene knockdown in hTFR×5xFAD double-transgenic AD model mice; the AOC 20 mpk and AOC10 mpk dosing groups showed significant reductions at D14 compared with the Vehicle control. — 03
hTFR×5xFAD · APP target gene knockdown
RT-qPCR (cortex, hippocampus, spinal cord, liver) and ELISA (cortex, hippocampus, DRG, liver sAPPα/sAPPβ) results for APP target gene knockdown in hTFR×5xFAD double-transgenic AD model mice; the AOC 20 mpk and AOC10 mpk dosing groups showed significant reductions at D14 compared with the Vehicle control.

Project discussion & study design

Share candidate type, target, intended route and existing results so we can define primary endpoints, groups and study cadence.

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Technical contact

Discuss your target, model or delivery question and learn about study plans, project scope and pricing.

+86 133 7528 3993 yijiusier@lewos.cn