Pre-Conference Focus Day

Tuesday June 18, 2024

Novel Delivery Strategies for More Durable & Potent RNA Editing Machinery

RNA editing therapeutics have great potential in treating a brand-new landscape of diseases due to its transient effects, however, it is currently plagued by difficult delivery to target tissues. With the possibility of modifying guide RNA, this session will uncover the unique delivery platforms which can be utilized to enable targeting of specific tissues, considering the capabilities of conjugates for specifically targeting RNA and reviewing opportunities of delivery beyond LNPs to enable repeatable dosing for more long-lasting and safer effects.

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8:00 am Registration & Refreshments

9:15 am Chair’s Opening Remarks

Unlocking the Power of Conjugates for Specific & Efficient Delivery Towards Hard-to-Treat Targets

9:30 am Emerging Strategies to Deliver RNA-Targeted Therapeutics to Extra-Hepatic Tissues

  • Rob Shaffer Co-Founder & Chief Operating Officer, Vrata Therapeutics

10:00 am Roundtable Discussion: Evaluating the Promise of Conjugates to Address Key Delivery Bottlenecks such as Improved Repeatability & Reduced Endosomal Escape

  • Rob Shaffer Co-Founder & Chief Operating Officer, Vrata Therapeutics

11:00 am Morning Break & Networking

Showcasing Delivery Strategies for Successful Delivery to the Liver & Beyond Accelerating RNA Editing Therapeutics into the Clinic

12:00 pm Addressing Viral Delivery Platforms for RNA Editing Machinery to Achieve Less Immunogenic Delivery & Improved Durability

12:30 pm Deep Diving into Human Endogenous Systems to Inspire Novel Delivery Platforms for RNA Editing Using mRNA

1:00 pm Enhanced Delivery Oligonucleotide (EDO) – A Novel Delivery Platform for Superior Oligonucleotide Delivery

Leveraging Novel & Known Delivery Platforms to Inspire New Delivery Strategies for RNA Editors to Cure Genetic Diseases Faster

1:30 pm Lunch & Networking

2:30 pm Roundtable Discussion: Learning from Liver Targeting Strategies to Build on its Success & Improve Delivery of RNA Editing Machinery

Transforming the Treatment of Neurological Diseases by Leveraging Innovative RNA Editing Strategies

3:30 pm Afternoon Break & Networking

4:00 pm Applying the RNAfix Platform to CNS Disorders: Efficient, Specific & Durable RNA Editing >95% in Mouse Brain

  • David Huss Chief Scientific Officer, Shape Therapeutics

4:30 pm Contribution of A-to-I RNA Editing to Neurodegenerative Diseases

  • Dimitra Dafou Associate Professor, Aristotle University of Thessaloniki

5:00 pm Chairs Closing Remarks & End of Focus Day