*Please note that all agenda timings are Eastern Time. For PDT time,

(please download the full program here.)

8:30 am Online Networking

Synopsis

Grab a quick cup of tea or coffee from the comfort of your own home office and jump straight into your opportunity to connect with new contacts from active companies in the field and exchange digital business cards. Network and form lasting connections through this exclusive virtual speed networking!

8:55 am Chair’s Opening Remarks

Developing RNA Editing Technologies in Preclinical Models

9:00 am Emergence of Novel A-to-I RNA Editing Sites

Synopsis

• How RNA editing sites are born?
• Reviewing how small and large scale genomic alterations lead to the formation and elimination of editing sites
• Understanding linage specific editing

9:30 am LEAPER: A Novel Oligonucleotide-Based RNA Base Editing Therapy Delivered via Lipid Nanoparticles (LNP) or Adenoassociated Virus (AAV)

  • Dong Wei Chief Executive Officer, Edigene

Synopsis

• LEAPER (Leveraging endogenous ADAR for programmable editing of RNA) enables precise and sequence specific A-to-I conversion at a desired mRNA sequence
• Synthetic LEAPER-based oligonucleotides (arRNA) delivered through LNP generated robust editing efficiency in vitro and in IDUA W392X mouse model, a model of MPS I disease
• arRNA expressed by AAV generated robust editing efficiency in vitro and in IDUA W392X mouse model

10:00 am Online Networking & Scientific Poster Session

Synopsis

Grab a quick cup of tea or coffee and take a look at the virtual poster hall to learn more about the up and coming advancements in 2020. You will be able to ask the presenter questions and view their poster presentation.

11:00 am A-to-I RNA Editing in Cardiovascular Disease

Synopsis

• Assessing how the expression of ADARs increases in the diseased hearts compared to the healthy ones, which contributes to the increased A-to-I RNA editing sites
• Adar1 has important functions in developing hearts by controlling cardiomyocyte survival and proliferation
• The cardiomyocyte-specific deletion of Adar1 in adult murine hearts causes upregulation of genes coding for structural proteins, increases myocyte apoptosis, and worsens heart function partially due to mitochondrial dysfunction

Evaluating Delivery Mechanisms

11:30 am Novel AAV Capsid Engineering for Delivery of RNA-Targeted Payloads & Beyond: the ShapeTX AAVidTM Directed Evolution Platform

12:00 pm Online Networking Lunch

Identifying the Clinical Application of RNA Editing

1:00 pm Targeting Cancer-Driven RNA Editing Events: an Oligonucleotide-Based Antisense Therapy

  • Polly Leilei Chen Principle Investigator and Assistant Professor, National University of Singapore

1:30 pm How ADAR1 can drive Disease Progression by Acting Both as an RNA Editor of Specific Transcripts & as a DNA Mutator of their Cognate Genes

  • Nina Papavasiliou University Professor and Division Head
    German Cancer Research Center, Heidelberg, Germany, Visiting Professor
    Rockefeller University, New York

Synopsis

• Evaluating RNA editing vs DNA mutation in cancer
• (Some) deaminases can act as RNA editors and DNA mutators
• Exploiting dual function enzymes for therapeutic purposes

2:00 pm Online Networking Break

Synopsis

This is your last opportunity to meet with other attendees and share virtual business cards!

2:30 pm Quantification of Ongoing APOBEC3A Activity in Tumor Cells by Monitoring RNA Editing at Hotspots

  • Remi Buisson Assistant Professor, University of California Irvine

Synopsis

• Identifying hotspot RNA mutations in APOBEC3A-positive tumors
• Sharing how we developed a droplet digital PCR assay to quantify the RNA-editing activity of APOBEC3A
• Demonstrating that the RNA mutation-based APOBEC3A assay is applicable to clinical samples from cancer patients

3:00 pm Path to Translating Natural RNA Editing into New Possibilities for Patients; Non-Clinical & Clinical Considerations

  • Yong Chang Senior Vice President of Development, Korro Bio

Synopsis

• Overview of development considerations including safety, regulatory and CMC
• Application and potential early indications

3:30 pm Chair’s Closing Remarks & End of Conference