Pre-Conference Day
Ambassador’s Morning
8:50 am Check-in, Coffee & Ambassadors’ Morning Networking
9:50 am Chair’s Opening Remarks
10:00 am Revolutionizing Pandemic Response: Harnessing the Power of mRNA Technology to Achieve 100 Days Mission of Vaccines Development
10:30 am Opportunities & Challenges for Investments & Strategic Partnerships in the Evolving mRNA-Based Therapeutics Market
Synopsis
- Seizing opportunities for investments and partnerships in a thriving mRNA company
- Overcoming challenges within a turbulent funding climate for mRNA companies to gain a scientific advantage
- Key recommendations to avoid risks and ensure success
11:00 am CEOs’ Fireside Chat
Synopsis
With the next wave of mRNA medicines approaching, and companies who were involved pre- pandemic now utilising
validated platforms to develop new therapeutics and vaccines, this leads to the inevitable question: What comes next?
We now need to realise the opportunities of such mRNA- based therapeutic applications, as we hear from the leaders of the field on the future of this technology.
- What does the future look like for the next generation of mRNA- based therapeutics for clinically relevant targets?
- Where do we expect new innovations to come from, and what challenges lie ahead of us?
- What limits actual development, where mRNA could be leveraged to replace existing technologies?
12:00 pm Lunch Break & Networking
Pre-Conference Focus Day:
AI RNA Design & Delivery
Pre-Conference Workshop Day
Over the last two years, mRNA therapeutics and vaccines have undergone a rapid transition from proof-of-concept to realworld impact. Artificial intelligence (AI) algorithms enable drug development from rapid experimentation cycles to help design mRNA and DNA sequences to the automation of quality control analyses and delivery formulations. Join the AI RNA Design and Delivery Focus Day to utilise the capabilities of the flexible, programmable nature of mRNA powered by machine learning (ML) to revolutionise how diseases are treated.
Chair's Opening Remarks
Duccio Medini, R3 Program Director, Wellcome Leap
Unlocking the Potential of Artificial Intelligence & Machine Learning to Accelerate mRNA Vaccine & Therapeutic Development
1.00 Digital Tools & AI/ML to Accelerate R&D for mRNA Vaccines & Therapeutics
• End to end digitalisation and the use of AI/ML in pharma R&D has the potential to accelerate cycle times, and increase the probability of success in bringing novel vaccines and therapies to patients
• Due to its platform nature, mRNA technology is particularly well suited to maximize these benefits
• Discussing applications of AI/ML and digital tools for mRNA platform R&D, with examples on mRNA sequence and lipid nano-particles design and optimisation
Fernando Ulloa Montoya, Head – Data & Computational Science mRNA Center of Excellence, Sanofi
1.30 Trailblazing mRNA Therapeutic Drug Discovery: From Back Translation of Sequence Design Through to LNP Delivery Optimisation
• Addressing the most common question the field is facing
• Examining the complexity of the problems associated with artificial intelligence in mRNA drug discovery
• Sharing which challenges are more amenable to different AI approaches
Wade Davis, VP - Computational Science, Moderna
2:00 Round Table Discussion and Speaker Question & Answers: Leveraging Computational Tools Within Our RNA Discovery Pipelines? Challenges & Opportunities
Workshop A
Developing an mRNA Raw Material Strategy to Accelerate High Quality mRNA Vaccine Manufacturing
1.00 - 3.00
With the validation of the SARS-CoV-2 mRNA vaccines on the global stage, mRNA vaccines and therapeutics are taking the front seat of many biopharma development pipelines. However, there is an ever-increasing demand for trusted raw materials that can lead drug developers safely down the long and winding road to regulatory approval. It is crucial for in-process raw materials to meet the required quality standards, perform consistently, and be scalable for commercial production; especially for low-middle income countries (LMICs). This workshop will address key challenges to ensure the safety and effectiveness of the final therapeutic products.
- Selecting high quality raw materials to meet
scalable demands - Considering the critical process steps to minimise the demand for raw materials and avoid manufacturing delay
- Discussing best practices for safeguarding the manufacturing process against the lack of consistency in the global supply chain, particularly for GMP grade production
- Overcoming regulatory hurdles with raw material quality and comparability to ensure equity for LMICs
Workshop Leaders:
Simone Blayer, Global Head – CMC, PATH
Jens Demand, Director – Product Life Cycle, PATH
3:00 Afternoon Break & Networking
Embracing Artificial Intelligence to Enhance RNA Drug Design & Delivery Optimisation
Pre-Conference Workshop Day
3.30 Developing a Foundational Model System for Multiple Downstream Applications: An mRNA Design Optimisation Case Study
• How can you increase expression of mRNA vaccines in different hosts with host agnostic models?
• How can you determine mRNA sequence homology, and vaccine and recombinant protein expression prediction?
• Benchmarking existing models for new applications
Sven Jager, Computational Science Manager, Sanofi
4.00 Key Early-Discovery Innovations to Include in Your mRNA Therapeutics Screening
• An overview of established strategies for mRNA
therapeutic screening
• How can the latest innovations be utilised during this process?
• Where do we start with high-throughput and machine learning methods?
• Addressing key questions that remain, even when those methods are used
Estelle Beguin, Director - Technology Research, Ethris
4.30 Fireside Discussion: Big Data, Clean Data – Improving Accuracy & Predictability with Data Quality
• Evaluating how AI and ML technologies can accelerate the process of identifying the ideal, most productive mRNA sequence needed to generate those proteins
• Understanding how over time, AI/machine learning refinements will continuously improve the range and effectiveness of this approach by providing ever-larger datasets to draw from
• De-risking the future of AI for development of mRNA therapeutics and vaccines
• Improving accuracy and predictability by avoiding assumptions to cross stitch data sets and fill knowledge gaps
Fernando Ulloa Montoya, Head - Data & Computational Science mRNA Center of Excellence, Sanofi
Sven Jager, Computational Science Manager, Sanofi,
Wade Davis, VP - Computational Science, Moderna
5.30 Chair's Closing Remarks
Duccio Medini, R3 Program Director, Wellcome Leap
Workshop B
Leveraging Experiences of Novel Delivery & Model Systems to Accelerate the Development of mRNA Therapeutics
3.30 - 5.30
With growing interest in mRNA-based technology for the development of medicines for infectious diseases, oncology and beyond, technological advancements in RNA biology, chemistry, stability, and delivery systems have accelerated development pipelines.
However, ensuring potent, long-lasting, and safe immune responses in pre-clinical development remains a significant challenge for human trials to be successful.
Critical appraisal of the recent literature sets out the different model systems to bridge the gap from studies in cell lines to non-traditional animal models towards clinical reality.
This workshop will leverage the cross-learnings of RNA delivery and models to aid the development from discovery through to clinic.
- Extracellular vesicles as RNA drug delivery systems: Opportunities and key challenges
- Transitioning from in vitro models to rodents and non-mammalian models to best predict efficacy in heterogeneous populations
- Cross learnings from cell-penetrating peptide-based microRNA and mRNA delivery strategies to address inflammatory skin and lung conditions: lessons from in vitro and in vivo models
Workshop Leaders:
Ana Rebane, Professor, University of Tartu
Francesca Ungaro, Associate Professor, University of Napoli Federico II
End of Pre-Conference Day