Conference Day Two
7:30 am Morning Coffee & Networking
8:20 am Chair’s Opening Remarks
Driving the Vision of the European mRNA Regulatory Landscape for First-in-Class mRNA Therapeutics & Emerging Health Threats
8:30 am Regulatory Considerations for mRNA Applications & Use of mRNA-LNP Technology
Synopsis
- Overview of key regulatory guidelines for different mRNA applications within the UK to accelerate mRNA products into the clinic
- Key considerations for mRNA-LNP technology and development of novel platforms
- The future directions with a regulatory lens and working with the industry for the benefit of patients
9:00 am Swiss Regulatory Aspects & Evaluation Considerations for mRNA Technology & Future Therapeutic Potential
Synopsis
- Swissmedic Innovation Office – Visibility near the innovation, promote innovation
- Swissmedic’s Competence Center Advanced Therapy Medicinal Products (ATMP): ATMP Risk Based Approach
9:15 am Showcasing WHO’s mRNA Vaccine R&D Priorities & Requirements for Low- & Middle- Income
Synopsis
- Numerous LMICs are acquiring mRNA vaccine production capacity as a tool to enable rapid response to future regional or global outbreaks. Maintaining this infrastructure requires that countries develop and manufacture mRNA vaccines for routine targets
- WHO, MPP, and their partners are building research networks to help these countries undertake R&D for mRNA vaccines against priority diseases in LMIC countries
- Reviewing the technical feasibility (PTRS) of developing the vaccines against these diseases, and also the probability that such vaccines would be procured (probability of policy development and procurement). This is a framework for R&D prioritisation to support these regions
9:45 am Panel Discussion: Realising The Impact of mRNA Medicine Perception on Public Health
Synopsis
• Understanding the complexity of the regulatory and scientific classification of innovative RNA products
• Gaining perspective on the intersection of public health and regulatory landscape from drug discovery to patient access
• What is the value for patients of more granularity in medicine classification?
• How can we increase public health benefits of mRNA medicines by improving the understanding of this new technology?
10:30 am Session Reserved: ReciBio Pharm
11:00 am Morning Networking Break
TRANSLATIONAL DEVELOPMENT TRACK
Developing Next Generation Delivery Vehicles to Improve Translational Potential
Chair Moderation:
11:30 am Delivery of Therapeutic RNA in Inflammation & Cancer Using Peptide-Based Nanoparticles
Synopsis
- Structure and assembly of peptide-based nanoparticles
- Advantages of peptide-based polyplexes versus other delivery systems
- Flexibility and tunability of SemaPhore as a delivery vehicle
12:00 pm Adjuvant-Modified Lipid Nanoparticles for mRNA Vaccine Delivery
Synopsis
- Inclusion of adjuvants in the lipid nanoparticIe-based carrier is a promising strategy for increasing the potency of mRNA vaccines
- Partial substitution of LNP components with an adjuvant result in colloidally stable LNPs with high mRNA entrapment and transfection efficiency
- The adjuvant substitution strategy works well for a clinically relevant mRNA-LNP vaccine, demonstrating its translational potential
CMC
TRACK
TRACK
Strategising Analytical Development Considerations from mRNA Drug Substance to Drug Products
Chair Moderation:
11:30 am Characterisation of mRNA Using LC & LC MS methods
Synopsis
- Direct sequence mapping of mRNA using LC MS methods
- Analysis of poly-A tail length and heterogeneity using LC MS
- Parameters which affect poly-A tail length and heterogeneity using LC MS
12:00 pm Advancing Analytical Strategies for Drug Product Characterisation & Understanding
Synopsis
- Outlining mRNA-LNPs within the QbD framework
- Improving mRNA-LNP sizing
- Optimising payload characterisation
12:30 pm Lunch Break & Networking
Achieving Effective Cell & Tissue Specificity in vivo for Next Generation mRNA-Based Therapeutics
1:30 pm Panel Discussion: Tissue-Specific Targeting of mRNA to Increase Therapeutic Efficacy
Synopsis
- Analysing cellular and tissue characteristics for successful uptake of mRNA- based therapeutics without inflammation
- Improving formulations to enhance mRNA specificity and eliminate mis-targeting to alternative cellular populations
- How can understanding biodistribution overcome challenges of target specificity?
2:30 pm Opportunities & Challenges for Extra-Hepatic Delivery of Therapeutic mRNA
Synopsis
- Physicochemical properties of Lipid-nanoparticles impact on their delivery capabilities depending on the administration orphan
- Discussion of extrahepatic delivery and therapeutic opportunities in the lung
- Challenges for preclinical translation associated with LNP delivery capabilities (Example PAN004)
Overcoming mRNA Quality Control Considerations to Ensure Biological Efficacy of mRNA Candidates
Chair Moderation:
1:30 pm Panel Discussion: Benchmarking Analytical Approaches to Better Characterise & Optimise the Drug Substance & Drug Product Manufacturing Process
Synopsis
- Considering the key factors when selecting analytical assays in the context of drug substance and product quality and efficacy
- Assessing the status of different analytical techniques for efficient scale-up and harmonising with current regulatory standards
- Implementing a predictive quality matrix as an added process control measure for concentration and final product evaluation
2:30 pm Developing Synthetic DNA Template (SDT)-Based Platforms for Quick in Vitro Transcription, Screening & Validation of Neoantigen & T-Cell Receptor mRNA Candidates
Synopsis
- Showcasing a synthetic DNA template for fast manufacturing of versatile single epitope mRNA
- From cap to tail: A molecular analysis and optimisation of antigen mRNA
- Long Synthetic DNA template (LSDT)-based platform for the in vitro transcription and
3:00 pm Afternoon Break & Networking
Unlocking Diseases of Tomorrow with Disruptive Companies & Next-Generation RNA Therapeutics & Vaccines
3:30 pm An Overview of the mRNA Landscape – Where Are We Now?
Synopsis
- Analysing the mRNA drug and trial landscape
- An overview of deals and companies in the mRNA space
4:00 pm Showcasing the Future Potential for mRNA-Based Vaccines & Therapeutics to Effectively Treat Cancer
Synopsis
Session Details TBC
4:30 pm Rational Design of Lipids & LNPs for mRNA Vaccines
Synopsis
- Rationally designed ionizable lipids that induce up to 20-fold neutralizing antibodies compared to clinically relevant mRNA vaccines for infectious diseases
- Novel ionizable lipids and LNPs for superior efficacy in tumor models
- Method to address manufacturing challenges of LNPs to improve mRNA vaccine accessibility