Check-In Opens & Light Breakfast | 8.00
Workshop A | 9.00 – 12.00
Uncovering the Pre-Clinical to Clinical Roadmap to Successfully Translate Non-Viral Vehicles for Safe & Efficacious RNA Delivery Leading to Improved Patient Outcomes
Synopsis
Being able to translate from the drug concept into an effective drug product can be a huge hurdle due to the difficulty in understanding which preclinical studies and models are robust and relevant for non-viral RNA delivery vehicles. Understanding a successful roadmap for moving through preclinical studies into the clinic is essential to ensure success and better outcomes for patients.
This workshop will address:
- What road map and endpoints are required to successfully transition the vehicle in vitro to in vivo studies?
- How to successfully translate a delivery vehicle from an animal to a human?
- What measures and comparative studies are required to successful translate delivery vehicle vs a drug product?
- How is the field successfully developing representative pre-clinical models for analyzing non-viral delivery vehicles suitable for clinical translatability?
Lunch & Networking Break | 12.00
Workshop B | 91.00 – 4.00
Unlocking Targeted Non-Viral Delivery Methods for RNA-Based Gene Editing Payloads to Minimize Toxic Effects for Improved Therapies
Synopsis
Gene editing payloads contain both the editor and guide RNA, making them much more complex to package. Similarly, there is potential for toxic side effects of permanent off target editing, and such it is vital that drug developers employ cell specific delivery vehicles. Uncover the key principles for successful delivery of RNAbased gene editors to ensure safe and specific editing.
This workshop will address:
- How to sufficiently package the large and complex gene editing machinery?
- How to optimize delivery vehicles for improved cell specificity to minimise the toxic effect of off target gene editing?
- How to successfully scale up non-viral gene editing delivery vehicles with the correct amount of each payload?
- How is endosomal escape affected by the different proportions of cargo component and larger RNAs?