Understanding RNAi Therapeutic Chemical Engineering to Enhance Extrahepatic Delivery for Improved Therapeutic Outcomes

Chemical engineering of RNAi therapeutics is pivotal for expanding their delivery beyond the liver, to tissues like the CNS, muscle, and eye. Attendees will gain insights into optimizing RNAi constructs for improved stability,

potency, and delivery efficiency, bridging the gap between preclinical promise and clinical success. A must-attend for researchers aiming to unlock the full potential of RNAi in extrahepatic applications by discussing:

  • Incorporating phosphorothioate and 2-modifications to boost siRNA nuclease resistance, enabling prolonged activity in extrahepatic tissues
  • Designing lipid-conjugated siRNAs for enhanced cellular uptake without carriers, simplifying delivery to challenging targets like skeletal muscle or the eye
  • Tailoring hydrophobicity and charge to optimize biodistribution and endosomal escape, maximizing target gene knockdown in non-liver organs