“...moving beyond the liver means demonstrating that targeted delivery can be clinically successful, and that this success is repeatable.”
Oligonucleotide therapeutics have delivered on their promise in the liver, where a fenestrated endothelium and abundant hepatocyte receptors do much of the work. Most of the disease landscape, however, lies beyond it, and conjugation is becoming the central strategy for getting there.
In the first cross-journal collaboration between Nucleic Acid Insights and Bioconjugate Insights, a panel of leading experts, including Oxana Beskrovnaya (Chief Innovation Officer, Dyne Therapeutics), Sasha Ebrahimi (Scientific Leader, Emerging Drug Delivery Platforms, GSK), Sritama Bose (Associate Director and Head of Chemistry, Orfonyx Bio), and Eduardo Paredes (Chief Technology Officer, Leal Therapeutics), examines how conjugation is extending oligonucleotide delivery into muscle, the central nervous system, the lung, and the kidney. The discussion covers linker design and payload loading, the characterization demands created by hybrid chemistries, the manufacturing requirements that separate a platform from a one-off success, and what would signal that the field has genuinely moved beyond the liver.
