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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

  • Ranjan Batra Vice President Research and Development, Locanabio

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

  • Dong Wei Chief Executive Officer, EdiGene

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

  • Zhen Li Chief Executive Officer, ADARx

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

  • Paloma Giangrande Vice President, Platform Discovery Sciences, Biology, Wave Life Sciences

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

  • Dinah Weismann Chief Executive Officer & Chief Scientific Officer, Alcediag

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

4:40 pm Panel Discussion: The Future Possibilities of RNA Editing

  • Paul Bolno, MD President and Chief Executive Officer, Wave Life Sciences
  • Dinah Weismann Chief Executive Officer & Chief Scientific Officer, Alcediag
  • Alan Herbert Founder and Head of Discovery, InsideOutBio
  • Nina Papavasilou Professor, Division Head, DKFZ German Cancer Research Centre

5:10 pm Chair’s Closing Remarks

5:15 pm End of Conference Day 2