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.
Download the Event Guide for Full Talk Details
8:15 am Check-In & Light Breakfast
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
10:00 am Roundtable Discussion: Evaluating the Promise of Conjugates to Address Key Delivery Bottlenecks such as Enabling Re-Dosing & Increasing Endosomal Escape
Synopsis
- What are some advantages and disadvantages of using conjugated delivery platforms over other methods such as viral delivery and lipid nanoparticles?
- How can ligands be utilized and optimized to increase the chance of endosomal escape to improve payload delivery?
- What would an ideal conjugated delivery platform look like, from PK to CMC? What hurdles do we need to overcome to get there?