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

  • Ka-Wai Wan Senior Pharmaceutical Assessor, Medicines & Healthcare products Regulatory Agency


  • 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

  • Julia Djonova Head Division Advanced Therapy Medicinal Products, Swiss Agency for Therapeutic Products, Swissmedic, Swiss Medical Group


  • 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


  • 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

  • Julia Djonova Head Division Advanced Therapy Medicinal Products, Swiss Agency for Therapeutic Products, Swissmedic, Swiss Medical Group
  • Maren von Fritschen Head Regulatory Policy Europe, Moderna
  • Marco Cavaleri Head of Biological Health Threats & Vaccines Strategy, European Medicines Agency
  • Martin friede Unit Head, World Health Organization - WHO
  • Ka-Wai Wan Senior Pharmaceutical Assessor, Medicines & Healthcare products Regulatory Agency
  • Duccio Medini R3 Program Director, Wellcome Leap


• 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


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


  • 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


  • 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


Strategising Analytical Development Considerations from mRNA Drug Substance to Drug Products

Chair Moderation:

  • Iulia Oita Chemistry, Manufacturing & Controls Manager, Ziphius Vaccines NV

11:30 am Characterisation of mRNA Using LC & LC MS methods


  • 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


  • 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


  • 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


  • 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:

  • Iulia Oita Chemistry, Manufacturing & Controls Manager, Ziphius Vaccines NV

1:30 pm Panel Discussion: Benchmarking Analytical Approaches to Better Characterise & Optimise the Drug Substance & Drug Product Manufacturing Process


  • 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


  • 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?


  • 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


Session Details TBC

4:30 pm Rational Design of Lipids & LNPs for mRNA Vaccines


  • 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

5:00 pm Chair’s Closing Remarks

5:05 pm End of 3rd mRNA-Based Therapeutics Summit Europe