Pre-Conference Workshop Day
8:50 am Check In & Morning Coffee
9:00 am Workshop A: Resolving the Challenge of Targeting Beyond the Liver: Understanding How the Field is Targeting the CNS, Skin & Eyes to Unlock the Potential of RNAi Therapeutics
Synopsis
Recent advancements have been made in targeting outside of the liver, this includes targeting the CNS, skin and eyes. Therefore, in order to continue the success of RNAi-based therapeutics, it is important to understand the vital parameters to allow for delivery to currently undruggable targets.
This workshop will gather experts to discuss:
• Understanding how to cross the BBB and utilizing this to successfully design a delivery platform
• How is the field approaching the challenges of crossing the BBB?
• What are the new delivery platforms which have been developed to target the skin?
• What are important safety parameters to consider when targeting or treating retinal diseases and related detection methods?
• How to establish siRNA-based target validation and develop therapeutic platforms for retinal diseases?
12:00 pm Lunch Break & Networking
1:00 pm Workshop B: Advancing Drug Conjugates for RNAi Therapies: Optimizing the Design & Chemistry of Conjugates & Their Linkers for More Successful Development of RNAi Therapies
Synopsis
Drug conjugates are being widely explored for their use with siRNAs and microRNAs. However, to successfully build these therapeutics, it is vital to understand the chemistry linking these components. Moreover, conjugated drug products have shown great potential in successful and efficient delivery.
This workshop will gather experts to discuss:
• How to design successful linkers between conjugates and siRNA/microRNA?
• Understanding the linker chemistry to ensure it is commercially manufacturable but still breaks apart at the correct point in the body.
• How are these new conjugate designs passing through the cell membrane?
• How do you ensure successful delivery to the target using conjugates, such as antibodies and peptides?
• Which kinds of conjugates should the field be prioritizing?