8:00 am Check-In & Light Breakfast
8:50 am Chair’s Opening Remarks
Uncovering RNA Editing’s Position in the Market to Improve Investment for more Effective Commercial Growth
9:00 am Reviewing RNA Editing Market Landscape
Synopsis
- Highlighting new companies and modalities within the RNA editing space
- Showcasing the evolving and growing landscape of RNA editing companies and technologies
- How is RNA editing currently viewed by the market in comparison to other RNA technologies and gene editing modalities?
9:15 am Fireside Chat: Discussing Investment with Investors to Better Understand their Views for Advancement of RNA Editing Therapies
Synopsis
- What motivation do venture capitalists need to invest in RNA editing therapeutics?
- Are venture capitalists ready to invest or are they waiting for a point of inflection?
- What are the risks investors are concerned about which is making them withhold?
- What data packages are they looking for?
10:15 am Morning Break & Speed Networking
Uncovering RNA Editing’s Capability for Evolving a Wider Genetic Disease Landscape to Enhance the Treatable Patient Population via RNA Editing Drugs
11:15 am Unlocking the Unique Opportunities of RNA Editing
Synopsis
- Building a precise RNA editing platform by leveraging endogenous ADAR
- GALNAC mediated ASO delivery introduces gain of function mutation
- Our platform unlocks broad therapeutic potential by addressing undruggable targets
11:45 am Roundtable Discussion: Standing Out from the Crowd: Understanding RNA Editing’s Unique Potential for Successful Treatment of More Diseases
Synopsis
- Uncovering the potential of RNA editing over other more permanent gene editing techniques, for instance to treat acute pain, viral infections, obesity and more.
- What are the key parameter differences in RNA editing vs DNA editing, e.g. guide RNA length, sequences and chemistry?
- What is the full range of indications and mutations which can be targeted by RNA editing therapeutics?
12:45 pm Lunch & Networking
Accomplishing Improved RNA Editing by Harnessing Innovative Screening Methods to Detect Effective Guide RNAs
2:00 pm Generative Machine Learning Models to Engineer ADAR dsRNA Substrates for Efficient & Selective A-to-I RNA Editing
Synopsis
- The therapeutic potential of recruiting endogenous ADAR for RNA editing is challenged 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 have developed a bit diffusion model that elucidates the complex rules that drive ADAR editing and generates de novo gRNAs that not only achieve optimized editing profiles but also sample from a diverse gRNA sequence landscape
- Our bit diffusion AI model outperforms other models and rationale designs across thousands of therapeutic targets. The capacity to generate novel gRNA designs de novo for any target adenosine shortens discovery timelines for designing exquisitely efficient and specific therapeutic candidates for thousands of genetic diseases
2:30 pm Developing Screening Strategies to Identify the Most Effective LEAPER arRNA for More Efficacious RNA Editing Therapies
Synopsis
- LEAPER leverages the endogenous ADAR proteins to enable RNA editing. LEAPER 2.0, incorporates specific modifications to significantly boost on-target editing efficiency
- Through comprehensive saturation mutation scanning, crucial mutations that amplify editing efficiency and provided valuable insights for refining RNA design strategies were identified. These results are instrumental in advancing AI-driven approaches for RNA design optimization.
- Employing AAV to deliver LEAPER 2.0, we accomplished potent and long-lasting RNA editing across various target organs in non-human primates, demonstrating its potential for broad therapeutic application.
3:00 pm Afternoon Break & Scientific Poster Session
Taking Advantage of RNA Trans-Splicing for More Efficient Editing of Alternative Targets to Widen the Disease Landscape
4:00 pm Multi-kilobase RNA edits with Splice Editors
Synopsis
- Splice Editors increase trans-splicing efficiency by leveraging CRISPR-Cas systems
- Splice Editor enabled multi-kilobase edits unlock several key indications and pathogenic variants
- Splice Editing is a promising new modality for RNA editing
4:30 pm Advantages of RNA Trans-splicing for Editing Inherited & Acquired Mutations that Cause Disease
Synopsis
- Describe the principles behind and approaches used for the creation of RNA transsplicing molecules (RTMs)
- Review of cell culture and in vivo animal studies that demonstrate RTM editing of premessenger RNA and reversal of a wide variety of disease endpoints
- How editing by RNA trans-splicing widens the therapeutic potential over other forms of RNA or DNA editing