Enabling Extrahepatic Delivery to Increase the Range of Treatable Diseases
As in vivo therapies expand beyond the liver, enabling extrahepatic delivery is essential to unlock new indications. This workshop explores strategies to reach CNS, bone marrow, lung, kidney and immune cells using approaches highlighted across the field.
This workshop will:
- Compare extrahepatic delivery strategies including localized routes, passive targeting, antibody- or bispecific mediated attachment, and next-generation LNP/viral designs
- Outline how teams evaluate biodistribution, species differences and regulatory expectations for non-hepatic targeting
- Provide a framework to select and validate delivery approaches that broaden the treatable disease landscape
- Share practical considerations to reduce CMC burden while achieving precise extrahepatic tropism