8:00 am Morning Coffee

8:55 am Chair’s Opening Remarks

Pioneering Next Generation RNA Delivery Approaches of Circular RNA & Self-Amplifying RNA to New & Specific Tissues

9:00 am Advancing Next Generation RNA Therapeutics Through Enhancing Delivery to Multiple Non-Hepatic Tissues

Synopsis

  • Gaining insight into the unique RNA therapeutic discovery and conjugation platforms to create groundbreaking treatments
  • Creating RNA therapeutics with improved pharmacology by joining them with antibodies for targeted delivery to disease tissue
  • Using genomic analysis to look at patient outcomes and correlate them with multiomics to improve standard of care

9:30 am Nonviral Delivery of Circular RNA to New & Specific Tissues

Synopsis

  • Introductions to the differences between viral and nonviral delivery approaches for circular RNA
  • Illustrating the potential of non-viral delivery strategies in improved targeted delivery to new tissues
  • Advances in the delivery of circular RNA to new tissues including the lung and bone marrow

10:00 am Overcoming the Challenge of RNA Therapeutics for the Treatment of Cancer & Development of IV Administered, Self-Amplifying RNA

Synopsis

  • Understanding the limitations of first-generation RNA modalities for treating cancer
  • Advantages of IV administered, self-amplifying RNA to get therapeutic amounts of RNA into tumors
  • Using viral machinery to selectively amplify in tumors and attract the immune system
  • Packaging viral machinery in LNPs enables repeat, systemic administration

10:30 am 10.30 Scientific Poster Session & Morning Networking Break

Innovating Delivery Strategies to Reflect the Changing Drug Delivery Needs of Next Generation RNAs

11:30 am Nanoparticle Formulation for Improved Delivery of Next Generation RNA Therapeutics to Brain Tumors

Synopsis

  • Comparing and contrasting the polymeric and lipid nanoparticles for the delivery of miRNA vs. next-generation RNA modalities
  • Understanding how novel nanoparticles are used as delivery vehicles
  • Showcasing results of successful novel therapeutics in brain tumors in vivo
  • Providing an outlook and next steps for optimizing delivery approaches using next generation polymers to target brain tumors

12:00 pm A Next Generation Delivery Approach of CAR-Encoding mRNA to Reprogram T-Cells in vivo

Synopsis

  • Discussing the potential and promise of in vivo delivery of CAR-encoding mRNA as a novel therapeutic
  • Active in vivo reprogramming using novel next generation delivery approaches
  • Transforming CAR-therapies by advancing next generation RNA therapeutics

12:30 pm Revolutionizing Next Generation Targeted LNPs (tLNPs) to Enable Off the-Shelf Immunotherapies

Synopsis

  • Exploring the potential of tLNPs, encapsulating mRNAs, for curative immunotherapies
  • Designing tLNPs for targeted delivery to specific cells for applications in oncology, fibrosis and inflammation related diseases
  • Achieving an advanced first-in-class therapeutic by ensuring tight control of dosage and activity of engineered in vivo cells

1:00 pm Roundtable Discussions:

Synopsis

Topic 1: Shaping the Future Landscape of Cutting-Edge Delivery Approaches by Revolutionizing the Discovery Phase

  • A short presentation providing insights into BioNTech’s delivery innovations, touching on selfamplifying RNA
  • What are the challenges and bottlenecks of existing delivery methods and how to optimize then
  • How is the delivery of next generation RNA to different tissues in the body progressing?
  • What new and innovative approaches are on the horizon?

Topic 2: Leveraging Data from Different Platforms to Ensure Comparability & Compatibility Between Studies

  • Comparing and contrasting the development of mRNA manufacturing platforms for vaccines and therapeutics
  • What analytical aspects of the manufacturing of next generation RNA therapeutics should we focus on and optimize?
  • What are the differences between the quality control of different next generation RNA platforms?
  • How to shape next generation RNA platform requirements to ensure scalable production of different novel RNA modalities

1:30 pm Networking Lunch

Optimizing Next Generation RNA Scalability & Stability Without Compromising Yield & Potency

2:30 pm Unveiling the Promise of Laronde’s eRNA Platform to Transform the Scalable Production of eRNA

Synopsis

  • Understanding the attractions of eRNA from stability, prolonged therapeutic effects and flexibility in formulation and delivery
  • Building modular and scalable eRNA to accommodate the clinical and commercial manufacture of scalable eRNA
  • Supporting the launch of multiple eRNA-based medicines with great therapeutic efficacy

3:00 pm Tackling the Twin Issues of Yield & Stability to Weaponize the Next Wave of Circular RNA Therapeutics

Synopsis

  • Setting the scene with theoretical introductions about the novel technology using to engineer circular RNA using bacteria
  • Manufacturing circular RNA using bacteria to enhance protein expression yields and long-term stability
  • Production of circular RNA aptamers to overcome previous limitations of aptamer-based therapeutics
  • Sharing novel data on the manufacturing process, with insights into challenges and future promise

3:30 pm Afternoon Networking Break

Improving Chemistry Vaccine Efficacy & Affordability of Next Generation RNA Modalities with Clinical & Commercial Needs in Mind

4:00 pm A Development of Dendritic Cell Targeted Lipid Nanoparticles (tLNPs) for Improved Vaccine Efficacy

Synopsis

  • Using data to show how ligand targeted LNPs are successfully used for the development of vaccines in comparison to recently approved nontargeted LNP vaccine formats
  • How the improved vaccine efficiency afforded by targeting allows for improved affordability, scalability, and ready inclusion of multiple mRNA cassettes
  • Improved storage stability through control of ionizable cationic lipid chemical structure in LNP vaccines
  • Taking forward key learnings to ensure successful and efficacious development of next generation modalities

4:30 pm The Development of Small Molecules Targeting Non-Coding RNAs, Including Circular RNA

  • George Calin Professor, Translational Molecular Pathology, MD Anderson Cancer Center

Synopsis

  • Presenting chemistry approaches and the in vitro and in vivo biology of targeting RNA
  • Comparing and contrasting the advantage and pitfalls of such as innovative strategy

5:00 pm Chair’s Closing Remarks

5:10 pm End of 2nd Annual Next Generation RNA Therapeutics Summit 2023