Scientific capability / GalNAc Targeting and Hepatic Activation Modeling

Choose the mechanism behind liver-directed exposure.

Receptor-mediated uptake and hepatic activation can both support a liver-directed strategy, but they solve different problems. BioTwin provides bounded models for examining the appropriate mechanism in a defined program.

Current work Bounded targeting and activation models

Compare liver-directed mechanisms All scientific capabilities

Two routes, two sets of measurements

GalNAc targeting investigates receptor-mediated uptake into hepatocytes. Hepatic activation investigates conversion during passage through the liver and the resulting hepatic and systemic exposure. A targeting label alone cannot answer the activation question, and an extraction model cannot establish receptor-mediated uptake.

Match the model to the intervention

The current toolkit contains distinct uptake and hepatic-activation operations. Use the one that fits the molecule, administration route and target compartment. Each requires its own parameters and calibration; evidence for one construct does not transfer automatically to another.

Make the delivery question affect the design

For a supplied GalNAc-containing clearance construct, receptor recruitment belongs alongside target binding and uptake. For an activation strategy, conversion and systemic escape may instead decide the experiment. A program can compare these hypotheses without pretending they are the same delivery mechanism.

Choose the liver-directed mechanism before choosing the model.

Describe the route, construct and target compartment so uptake and activation can be assessed on their own terms.

Compare liver-directed mechanisms