Day Two Agenda

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8:30 am Morning Networking Coffee

8:50 am Chair’s Opening Remarks

  • Andrew Geall Co-founder & Chief Development Officer, Replicate Bioscience

Innovating Immunotherapy Approaches through Next Generation RNA Therapeutics

9:00 am mRNA 2.0 Immunotherapy for Cancer

  • Yusuf Erkul Co-founder & Chief Executive Officer, Kernal Biologics Inc.

Synopsis

  • Improvements in innate immunogenicity of mRNA therapeutics can expand their
    therapeutic window
  • Cancer ribosomes provide a novel opportunity for the design of onco-selective
    mRNAs
  • Stealth, onco-selective, and oncolytic mRNAs can induce tumor regression in
    syngeneic mouse models resistant to check-point inhibitors

9:30 am Next Generation Self-Replicating RNA Vectors to Enable New Approaches to Immunotherapy

  • Nathaniel Wang Chief Executive Officer & Co-Founder, Replicate Bioscience

Synopsis

  • Next generation synthetic srRNA vectors can lower dose by orders of magnitude
    compared to current srRNA designs
  • Developing new srRNA products requires independent optimization of vectors,
    gene inserts, and delivery
  • Improved srRNA bioactivity enable new approaches for immunotherapies in
    oncology

10:00 am Programming mRNA for Cancer Immunotherapy

  • Tasuku Kitada President, Director, Co-founder, Head of Research & Development, Strand Therapeutics Inc.

Synopsis

  • Outlining current preclinical and clinical data indicating efficacy of mRNA cancer
    treatments
  • Discussing why Strand’s approach may be superior to current mRNA drugs in the
    clinic
  • The future of mRNA oncology therapy may involve precise programming of
    protein expression using synthetic biology

10:30 am Morning Networking Break

RNA Translation & Regulatory Consideration for Next Generation RNA Therapeutics

11:30 am Engineering New Modalities to Control RNA Translation

  • Velia Siciliano Principal Investigator - Synthetic & System Biology for Biomedicine, Italian Institute of Technology

Synopsis

  • Use of RNA binding proteins to encode RNA circuits
  • Design of RNA-encoded genetic devices by RBP
  • Re-engineering RBPs to respond to protease as indication of viral infection

12:00 pm Roundtable Discussion: Discussing Regulatory Considerations for Next Generation RNA Therapeutics & Vaccines

1:00 pm Networking Lunch

Emerging & Next Generation Formulation & Delivery Strategies to Maximize Therapeutic Potentials

2:00 pm Unravelling Viral & Non-viral Delivery Approaches for Next Generation RNA Therapeutics

  • Anna Blakney Assistant Professor, University of British Columbia

Synopsis

  • Understanding the stability and quality differences between viral and non-viral
    delivery strategies
  • Better understanding biological mechanism of non-viral delivery strategies to
    improve targeted delivery
  • How to choose the most appropriate delivery system for different types of RNA?

2:30 pm Panel Discussion: Discussing Emerging LNP Modifications & Polymer-Based RNA Delivery for Next Generation RNA Therapeutics

  • Andrew Geall Co-founder & Chief Development Officer, Replicate Bioscience
  • Thomas Colace Director, LNP Formulation, Tessera Therapeutics
  • Allen Horhota Senior Director & Head of Delivery, Orna Therapeutics
  • Anna Blakney Assistant Professor, University of British Columbia

Synopsis

  • Outlining the advantages of different LNP modifications to enhance target specificity of next generation RNA
    therapeutics
  • Describing the biological mechanism of LNP and polymer-based delivery to increase target specificity
  • Discussing the challenges of maintaining sufficient extrahepatic protein expression

3:30 pm Next Generation Nebulizer Formulations for mRNA-Based Therapeutics for the Lung

Synopsis

  • Development of nebulization screening platform for mRNA delivery to the lung
  • Polymer versus LNP for lung delivery via nebulization
  • Demonstration of efficacy against SARS-CoV-2 and influenza

4:00 pm Chair’s Closing Remarks

  • Andrew Geall Co-founder & Chief Development Officer, Replicate Bioscience

End of Next Generation RNA Therapeutics Summit