Explore the Agenda
8:00 am Check In & Light Breakfast
Workshop A
9:00 am Developing Robust, Reproducible & Sensitive Analytical & Functional Assays to Accurately Measure Editing Efficiency, Off-Target Activity & Impact of Editing on Disease
Understanding the level of achieved editing efficiency and the functional effect of editing is vital to measure efficacy and understand the true impact of RNA editing therapies. Reliable, reproducible analytical assays are essential in this process and can indicate off-target editing, leading to the development of safer, more efficacious RNA editing medicines.
This workshop will cover:
• Developing robust, reproducible analytical assays that mimic human biological mechanisms to accurately measure levels of editing efficiency
• Utilising functional assays to understand the effect of editing on protein function and disease to elucidate efficacy
• Analysing the transcriptome and genome to understand off-target effects and inform biomarker identification and future therapeutic development
12:00 pm Lunch & Networking
Workshop B
1:00 pm Navigating RNA Editing Regulatory Challenges & Optimizing Translation of RNA Editing Candidates to Accelerate IND Approval, Streamline Entry into the Clinic & Achieve Commercial Success for RNA Editors
Developing clinically successful therapies with strong efficacy and tolerability relies on robust preclinical development and regulatory compliance in order to maximize patient safety and ultimately drive market success. Navigating the regulatory landscape for RNA editing can be challenging, with unforeseen hurdles to overcome on the path to commercialization.
This workshop will address:
• Understanding the attributes necessary for clinical translation of RNA editing candidates
• Navigating the regulatory path and upholding standards during preclinical and clinical development
• Identifying key hurdles on the path to commercialization and strategizing solutions to accelerate success in the clinic and on the market