Pre-Conference Day

Interactive, case study-led workshop exploring how leading developers are overcoming the biological barriers limiting oligonucleotide delivery beyond the liver. Through realworld development examples and collaborative discussion, attendees will evaluate the trade-offs between emerging targeting platforms and leave with a practical framework for selecting the right delivery strategy for their own programmes.

Workshop highlights

• Work through real-world case studies of extrahepatic and CNS delivery programmes to understand where promising approaches translated successfully and where they broke down between preclinical models and the clinic
• Compare transferrin receptor conjugates, antibody shuttles, ligand-based targeting and non-conjugated approaches to determine which strategy is best suited to different tissues, indications and therapeutic modalities
• Leave with a practical framework to identify whether delivery strategy, targeting ligand or oligonucleotide chemistry represents the greatest development risk in your programme and where to focus optimisation efforts first

Interactive workshop exploring how developers are designing, selecting and optimizing conjugates, ligands and linkers to improve tissue-specific delivery and maximize therapeutic performance. Through expert discussion and practical case studies, attendees will evaluate the critical design decisions that influence target efficiency, manufacturability, safety and long-term platform potential.

Workshop highlights

• Evaluate the latest ligand discovery strategies, comparing established targets such as ASGPR and transferrin receptor with emerging receptor biology to identify new opportunities for tissue-specific delivery
• Explore how linker design influences conjugate performance, understanding the impact of stability, release mechanisms and pharmacokinetics on efficacy, safety and therapeutic index
• Understand the CMC and manufacturing considerations that shape successful conjugate development, including process scalability, raw material selection, technology transfer and the impact of design decisions on manufacturability and clinical supply
• Leave with a practical framework for building a competitive targeting platform, including the key scientific, translational and commercial questions partners and investors will expect your technology to answer before advancing into collaboration or development