8:00 am
Coffee and Registration
8:30 am Chair’s Opening Remarks
Pre-Clinical Models Exploring the Initial Challenges and Methods When Striving to Make RNA Editing a Therapeutic Reality
8:40 am RNA Targeting Gene Therapy for Neuromuscular Disease
Synopsis
- Understanding strategy for targeting RNA vs. DNA to treat neuromuscular diseases
- Comparing RNA targeting systems for therapeutic purposes
- Discussing in vivo considerations for RNA targeting gene therapy
9:10 am RNA Therapeutics with LEAPER 2.0: Initial Proof of Concept in Preclinical Models including Human Primary Cells, Mice and Nonhuman Primates
Synopsis
- Understanding how therapeutically meaningful RNA editing rate was achieved in human primary cells in selected genetic diseases
- Comparing to LEAPER 1.0, LEAPER 2.0 results in a significantly higher level of RNA editing in a mouse model
- Highlighting the potent and durable RNA editing achieved in multiple target organs of non-human primates via AAV delivery of LEAPER 2.0
9:40 am An ADARx Pre-Clinical Model of RNA Editing Therapeutics
Synopsis
- Showcasing and overcoming the initial challenges
- Analysing the recent developments and new technologies
- An exclusive look into ADARx novel pre-clinical model of RNA editing therapeutics
10:10 am
Morning Break
11:00 am Understanding Lessons Learned From AON Clinical Development and Translation to the RNA Base Editing Axiomer Technology
Synopsis
- Understanding the lessons learned from a big pharma company
- Exploring the journey that led to success
Disease Specific Case Studies Using RNA Editing Therapeutically Across Diseases
11:30 am AIMers: Correcting Driver Mutations for the Treatment of Alpha-1 Antitrypsin Deficiency (AATD) and Beyond
Synopsis
- Understand how our RNA editing platform allows for correction of driver mutations of AATD
- Developing RNA Editing AIMers to correct homozygous “ZZ” mutations back to “MZ” heterozygous state
- AIMers lead to durable and functional expression of M-AAT protein, establishing an RNA editing platform to target liver and potentially other organs
12:00 pm
Lunch
1:00 pm RNA Editing for Personalized Diagnostics in Neuropsychiatry
Synopsis
- Exploring how Alcediag develops RNA editing blood biomarkers powered by artificial intelligence in Mental health and neurosciences, inflammatory disease and oncology
- Discovering EDIT-B, EDIT-B is the first blood test based on RNA editing biomarkers to be introduced in psychiatry where diagnoses are only based on clinical evaluations
- Highlighting the efficacy of EDIT-B, it differentiates unipolar and bipolar depressed patients with a performance of over 80%. It will change the game by introducing precision medicine in psychiatry
1:30 pm Flipons, Editing and Cancer
Synopsis
- Understanding how in addition to the recoding of proteins, RNA editing regulates biological responses by modifying RNAs encoded by repeat elements
- Highlighting with ADAR1, the process is targeted by flipons that form Z-RNA in regions of dsRNA, preventing interferon induction
- Discussing tumors that produce excess dsRNAs use ADAR1 to inhibit interferondriven immune responses against them
- Exploring targeting of Z-flipon recognition by ADAR1 represents a novel therapeutic intervention to enhance immune checkpoint blockade
2:00 pm Beacon: The RNA Editing Landscape
2:40 pm
Afternoon Break
3:10 pm ADARs, APOEBECS, and tumours: RNA Editing and Disease Manifestations
Synopsis
- Discussing how RNA editing can clearly alter disease manifestations, but there are bottlenecks with regards to efficiency and specificity
- Exploring RNA editing enzymes (ADARs and APOEBECs) that are prominently expressed in most tumours (vs healthy tissues)
- Can we target these to HLA-presented epitopes toward apparent neoepitope formation (and T-cell clearance?)
3:40 pm RNA Editing in Mesothelioma
Synopsis
- Discussing RNA editing in mesothelioma development
- Highlighting DsRNA in mesothelioma
- Exploring ADARs and response to therapy
4:10 pm A-I RNA Editing in the Cardiovascular System and Disease
Synopsis
- Highlighting how A-I RNA editing is important for cardiac development
- Analysing how dysregulation of ADARs leads to cardiac dysfunction and disease progression
- Understanding cell-type-specific regulation of ADARs in the heart is important for proper cardiac function