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
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
