8:00 am Coffee & Registration

8:50 am Chair’s Opening Remarks

Providing a Future Outlook for the RNA Editing Landscape

9:00 am Mapping the RNA Editing R&D Landscape: A 2023 Forecast

Synopsis

  • Discussing the market trends and challenges of RNA editing therapeutics
  • Projecting the potential of the different RNA editing therapeutics
  • Analyzing the intensifying RNA editing race as biopharma continue to buy in

9:30 am Panel Discussion: Exploring Unique Challenges for the Development of RNA Editing Therapies

  • Steven Seedhouse Managing Director, Biotechnology Equity Research, Raymond James
  • Naveen Krishnan Senior Director of Venture Investment, Bayer
  • Paul Bolno President & Chief Executive Officer, Wave Life Sciences

Synopsis

  • Providing an overview of the current standpoint on RNA editing therapies
  • Understanding how regulators and drug developers can work collaboratively to accelerate novel candidates into the clinic
  • Navigating the commercialization and development challenges facing RNA editing technologies 

10:15 am Scientific Poster Session & Morning Networking Break

Navigating Machine Learning & AI Models to Enable Target Discovery & Efficient Editing

11:15 am Applying Data Science & Machine Learning Approaches to Improve Efficacy of ADAR

Synopsis

  • Learning the principles of ADAR-based RNA editing using data science
  • Divulging that machine learning models enable the prediction of ADAR editing levels and contribute to the improvement of ADAR’s editing efficiency
  • Using machine learning to optimize the structure of chemically modified guide RNAs to model editing efficacy

11:45 am Generative Machine Learning Enables DGuide RNA Design for Precise RNA Editing

  • Ron Hause Vice President & Head of Analytics & Informatics, Shape Therapeutics

Synopsis

  • The therapeutic potential of recruiting endogenous ADAR for RNA editing is hindered by its natural preference to edit adenosines within certain sequence contexts and its proclivity to edit multiple adenosines in a dsRNA substrate
  • Using high-throughput screening and structurally-aware generative AI, we can rapidly design gRNAs that recruit ADAR to efficiently and specifically edit any disease target of interest, regardless of its sequence context

12:15 pm Discovering Targetable mRNA Structures in the Transcriptome

  • Dave Mauger Director of Data Science, Arrakis Therapeutics

Synopsis

  • Global transcriptomic data are critical to understanding mRNA isoform diversity across cells and tissues
  • Integrating global biochemical assays with biophysical data enables the identification of relevant RNA structures
  • Transcriptomic target engagement data measures selectivity and enables target discovery efforts

12:45 pm Networking Lunch Break

Revolutionizing RNA Editing Platforms through Splice Switching Antisense Oligonucleotides (ASOs) for Enhanced Safety & Efficiency

1:45 pm Case Study: Recent Advances in Splice Switching ASOs for CNS Diseases

Synopsis

  • Understanding the potential of RNA editing to slow down or stop the neurodegenerative effects of ALS
  • Novel technology platform to improve safety and potency profile of ASOs

2:15 pm Shuttle Peptides Enable Delivery of Splice Switching ASOs to Airway Epithelial Cells

  • David Guay Vice President of Innovation, Feldan Therapeutics

Synopsis

  • Development and optimization of the shuttle peptides that enable rapid delivery of PMOs to airway cells
  • PMO delivery to mouse airway epithelial cells in vivo

2:45 pm Afternoon Networking Break

Exploring RNA Editing Mediated Exon Evolution & Gene Activation Platforms to Drive Enhanced Expression

3:15 pm Re-Engineering the Transcriptome via RNA Exon Editing: A Novel Therapeutic Approach

  • Daniel Rosan Chief Business & Financial Officer, Ascidian Therapeutics

Synopsis

  • Deep-dive into the approach of editing exons at the RNA level via RNA transplicing; what are the advantages and disadvantages compared to other gene editing approaches
  • Leveraging high-throughput molecular biology in tandem with cutting edge computational biology and structure-based RNA design to increase RNA exon editing efficiencies to therapeutically relevant levels
  • Enabling therapeutic targeting of large genes and genes with high mutational variance while keeping gene expression circuits intact

3:45 pm G-Rich Motifs within Phosphorothioate-Based ASOs Drive Activation of FXN Expression through Indirect Effects

  • Feng Damon Wang Post Doc Associate, Jon Watts Lab (RNA Therapeutics Institute, UMass Chan Medical School

Synopsis

  • Identification of ASOs complementary to two regions within the first intron of FXN pre-mRNA, which could increase FXN mRNA by 2-fold in patient fibroblasts
  • Identification of two guanosine-rich motifs (CCGG and G4) within the phosphorothioate-based ASOs that were required for FXN activation
  • An atlas of gene activation platform technologies

4:15 pm Chair’s Closing Remarks

4:20 pm End of the 4th RNA Editing Summit