Roundtable: The Delivery Playbook: Integrating Novel Platforms, Chemistries, and Targeting Ligands for Widespread Extrahepatic Impact

The breakthroughs in RNAi delivery are no longer confined to the liver, with a new generation of platforms, from antibody conjugates and DNA nanostructures to novel ligands and chemical modifications, enabling precise targeting of metabolic, muscular, and renal tissues. This roundtable unites pioneers to discuss:

  • Weighing the advantages of modular platforms against bespoke conjugate strategies for specific tissue targets, considering factors like valency, manufacturability, and immunogenicity.
  • Addressing the unique synthesis, characterization, and manufacturing challenges of complex delivery modalities to ensure quality, stability, and scalability for clinical and commercial supply.
  • Strategizing on how to design platforms with inherent adaptability to rapidly retarget new tissues and diseases, maximizing platform value and accelerating development timelines.
  • n selection for optimal risk/reward, ensuring portfolio companies can attract pharma partnerships or IPO success