Beyond the liver: Optimizing Oligonucleotide delivery for extrahepatic targets in discovery
03 Sept 2026
Drug Delivery
- When the screening model, not the molecule, is the problem - In vitro potency under transfection or free uptake often fails to predict extrahepatic delivery in vivo. How do we choose discovery models that better reflect the actual delivery barrier in non-liver tissues?
- Aligning chemistry and format to the target tissue early - How should teams think about backbone chemistry, conjugates, and modality/format choices such as ASO, siRNA, conjugates, or LNPs when the destination is extrahepatic and the delivery problem is still unresolved?
- Designing discovery for downstream developability - Which discovery-stage decisions most often determine whether an extrahepatic candidate survives development, and how can teams build in de-risking early rather than discovering delivery liabilities later in tox or the clinic?
Industry Expert
