Explore the Agenda
8:00 am Check In & Light Breakfast
8:50 am Chair’s Opening Remarks
Illuminating the Investment & Partnering Landscape to Guide Future Directions for Research, Growth & Collaboration to Power the Future of RNA Editing
9:00 am Investment Panel: Attracting Investment & Securing Preclinical Funding for Early-Stage RNA Editing Therapeutic Candidates
• How is RNA editing viewed by investors and what are the directions for growth?
• How to successfully gain funding for preclinical candidates by demonstrating the right data
• What are the hurdles and pitfalls to starting an RNA Editing therapeutics company and what are the timelines, collaborations, and levels of investment required
10:00 am CEOs’ Fireside Chat: Lessons from RNA Editing’s CEOs on Driving Clinical & Commercial Development to Accelerate RNA Editors to Patients
• Delving into the work of RNA editing’s leading CEOs to understand their perspectives on the field, ongoing journey to success, and directions for future development
• Learnings to apply to your own RNA editing pipeline and business strategy to accelerate translation and achieve commercial viability
11:00 am Morning Break & Networking
Advancing RNA Editing Therapeutic Development with Technological Advances & Strategic Collaborations to Evolve the Scope of Editing to Common Disease
12:00 pm Uncovering Novel Therapeutic Targets for RNA Editing through Human Genetics & AI
• Leveraging human genetic data and AI to identify and prioritize genetic targets for RNA editing
• Investigating the target landscape in common diseases with significant unmet therapeutic needs
• Identifying opportunities beyond correcting disease-causing mutations
12:30 pm Round Table Discussion: Employing Machine Learning & Computational Biology to Validate Novel Therapeutic Targets, Design Robust Guide RNA & Predict Functional Effect of Editing, Driving the Development of Safe, Selective Editors
• Utilizing machine learning and computational biology to predict the functional effect of editing to assist preclinical development and safeguard patient safety
• Designing high-quality, durable, and ultra-specific guide RNA using machine learning tools to improve editing magnitude and safety
• Employing computational tools to apply RNA editing to common diseases by targeting multiple mutations and identifying novel therapeutic targets
1:30 pm Lunch Break & Networking
Expanding the Scope of RNA Editing by Analyzing Human Genetics Data & Identifying Differentiated Novel Targets to Successfully Treat a Wider Range of Indications
2:30 pm Panel Discussion: Uncovering Differentiated Targets, Exploring Editing in a Wider Range of Diseases & Identifying Patients Who Could Benefit from RNA Editing
• Identifying differentiated target sequences using human genomics data and AI
• Exploring editing ceilings and limitations of various targets
• Elucidating treatable diseases and identifying patients with the suitable genotype who could benefit from treatment
3:15 pm Defining ADAR1 RNA Editing as a Causal Mechanism of Coronary Artery Disease
• ADAR1 RNA editing is a critical regulatory mechanism of innate immune activation through preventing endogenous double-strand RNA sensing
• Human genetics implicates dsRNA sensing and the inadequate ADAR1 RNA editing as a causal mechanism of coronary artery disease
• Through comprehensive studies utilizing human genetics, primary in vitro culture, mouse models of disease, and single-cell RNA sequencing, we have defined the mechanism by which ADAR1 RNA editing and dsRNA sensing by MDA5 mediates atherosclerotic disease risk
3:45 pm Pioneering RNA Modification Beyond Rare Diseases by Exploring Novel Editing Technologies & Targeting Multiple Mutations to Remove the Barriers to Treating Common Diseases
- Rethinking editing machinery to edit beyond point mutations and specifically modify multiple target sites simultaneously
- Preclinical data to demonstrate the potential of RNA editing outside of rare disease
- Strategically investigating common disease targets to attract investment and pharma partnerships