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8:00 am
Coffee & Registration

8:30 am Chair’s Opening Remarks

Synopsis

  • Reflecting on why RNA editing is reaching an inflection point
  • Highlighting the new RNA editing technologies that have advanced significantly in the past few years
  • Applying the therapeutic potential of RNA editing to our work

Improving Fundamental Mechanisms of RNA-Editing In Order to Prepare for Therapeutic Application

8:40 am Fundamentals of RNA Editing in the Central Nervous System

  • Michael Breen Assistant Professor, Icahn School of Medicine at Mount Sinai

Synopsis

  • Understanding RNA editing dynamics across brain regions, cell types,
    development, and disease
  • Dissecting brain cell type- and isoform-specific RNA editing and the influence on
    secondary structures
  • Discussing common genetic rick loci for neurological disorders that alter RNA
    editing levels in the brain as therapeutic candidates

9:10 am Quantitative Analysis of the Endogenous Editome to Guide Base- Editing Development

Synopsis

  • Deciphering why editing quantification is essential for studying RNA editing, often limited by coverage and alignment problems
  • Discussing several common pitfalls and several strategies appropriate for coding and non-coding regions
  • Unravelling methods developed and lessons learned from the endogenous editome that may facilitate better site-directed editing design

9:40 am RNAfix: Therapeutic RNA Editing with Engineered Guide RNAs and Endogenous ADAR

  • Adrian Briggs Head of Platform Technologies, Shape Therapeutics

Synopsis

  • Understanding endogenous ADAR enzyme can be harnessed to edit adenosines in RNA and treat disease. A massively parallel screen of guide RNAs (gRNAs) identifies designs that enable highly efficient and specific editing of clinically
    relevant targets
  • Divulging that machine learning analysis of deep gRNA screening datasets allows computational generation of gRNAs with improved performance relative to any designs in the original screen
  • Discussing identified gRNAs can be further optimized and genetically delivered with AAV to enable therapeutic levels of editing in multiple cell types

10:10 am
Morning Break and Structured Networking

11:10 am Platform Optimization to Enable Therapeutic Development of ADARMediated RNA Editing

Synopsis

  • Discussing Korro Bio’s ADAR platform
  • Highlighting Guide Optimization
  • Showcasing the ADAR Pipeline

11:40 am Panel Discussion: Chemical Modifications of RNA Editing

12:10 pm
Lunch & Networking

Mitigating Against Off-Target Editing To Optimize RNA Editing for Therapeutic Application in a Range of Diseases

1:10 pm Leaper 2.0: Next Generation of Leveraging Endogenous ADAR for Programmable Editing on RNA

  • Wensheng Wei Scientific Founder, EdiGene, Professor with tenure and Director, Peking University Genome Editing Research Center

Synopsis

  • Exploring how LEAPER edits RNA by recruiting endogenous ADAR proteins through double stranded RNA structure formed between arRNA and the specific sequence in the target RNA
  • Showcasing how in LEAPER 2.0, specific elements have been engineered to significantly improve the on-target editing efficiency
  • Understanding how additional designs have been developed to reduce the bystander off-target editing events

1:40 pm Global Quantification Exposes Abundant Low-Level Off-Target Activity by Base Editors

Synopsis

  • Discovering how much of the off-target activity of the deaminases are non-specific
  • Discussing a new class of off-target events that are invincible to the established methods for detection of genomic variations and were thus far overlooked
  • Divulging a computational tool to quantify global off-target activity, which can be used to optimise future base editors

2:10 pm
Afternoon Break & Networking

2:45 pm ITN-European Union ROPES Network

Synopsis

  • Understanding that comprehensive epitranscriptomic analyses will provide the missing link between genomic variability and cellular phenotype, contributing to elucidating the cause of specific diseases and developing novel therapies
  • THe ROPES (ROles of ePitranscriptomic in diseasES) network specifically aims at training a group of competitive Early Stage Researchers (ESRs) which will join a network of academic research groups and biotech companies focused on advancing the knowledge on the role of RNA modifications on different diseases and models, and in promoting the development of innovative therapies

3:15 pm Interactive Discussion Groups

Synopsis

This session facilitates in-depth discussions among participants in an informal environment. After splitting into groups, participants will discuss key challenges and opportunities around their chosen topic

  • Chemical Modifications of RNA
  • Delivery Mechanisms
  • Developing a Validation Library in RNA-Editing

4:00 pm Chair’s Closing Remarks

4:05 pm End of Day One