Maximising Cytosolic Delivery of Oligonucleotides Through Endosomal Escape

  • Evaluating intracellular sequestration of oligonucleotides in endosomes and uptake by glial or immune cells to understand limitations in target neuron bioavailability
  • Exploring ligand and conjugate strategies, including lipid, peptide, and antibody modifications, to improve endosomal escape and cytosolic delivery
  • Linking escape efficiency to dosing, safety, and disease-specific biology to optimise therapeutic index across neuro targets

NEW DATA