Conference Day One | Wednesday, 29th January
7.15 Check In & Morning Coffee
8:15 am Chair’s Opening Remarks
Visions for the Future of mRNA & Beyond: The Leadership Perspective
8:25 am CEO & Global Leaders Think Tank: Unlocking the Next Frontiers of mRNA-Based Vaccines & Therapeutics to Ensure Better Drugs Reach Patients Faster
Synopsis
- What are the future opportunities for the mRNA field towards best-in-class drugs into 2025?
- What are the biggest challenges you face on a day-to-day basis from asset, deals to risk-management and clinical strategy?
- What can biotechs get most out of an investment to extend cash-flow and nuances of striking a deal for a new pipeline?
9:15 am Current Cancer Vaccine Strategies Including Initial Phase 1 Study Data of CVGBM, an Off-the-Shelf Cancer Vaccine in Glioblastoma
Synopsis
- Cancer vaccines based on mRNA technologies have shown encouraging clinical data over the last couple of years
- Showcasing current strategies to develop cancer vaccines focus on either personalized cancer vaccines or off-the-shelf cancer vaccines
- Presenting CVGBM, an off-the-shelf cancer vaccine, that demonstrated encouraging immunogenicity in a phase 1 study in patients with GBM after tumor resection
9:45 am Showcasing Clinical Trial Experience & Data from BNT116 – The UK’s First mRNA-Based Lung Cancer Vaccine Trial
10:15 Morning Break & Speed Networking
TRACK A: DISCOVERY & PRE-CLINICAL DEVELOPMENT
Synopsis
Chair: Nigel Horscroft, Chief Technology Officer, Stealth Mode mRNA Biotech
Showcasing New Discoveries in mRNA Medicine From Asia to Unlock Platforms for Obesity & Antibody Production
11:15 am Advancing mRNA Therapeutics: A New Frontier in the Treatment of Obesity & Metabolic Disease
Synopsis
- Presenting new preclinical data from WestGene’s obesity program, demonstrating significant weight loss and metabolic improvements
- Highlighting how WestGene’s proprietary mRNA-encoded antibody platform drives these results and opens new possibilities for therapeutic innovation
- Exploring the platform’s broader potential, using the obesity project as a key example of its applications in metabolic diseases, oncology, and other critical areas
11:45 am Development of Novel & Efficient Cap Analogs & Ionizable Lipids for mRNA Based Vaccines & Therapeutics
Synopsis
Novel trinucloside AG and AU cap analogs amenable to synthesizing mRNAs and saRNAs with an enhanced protein expression profile
- One-pot synthesis of fluorescently labeled mRNA for real-time in vitro and in vivo tracking, and mRNA delivery system screening
- Efficient and fast degrading ionizable lipid for mRNA LNP delivery
- Application of LZCaps in mRNA vaccines and protein replacement therapies
12:15 pm Harnessing In Vivo Therapeutic Antibody Production Enabled by mRNA-LNP Technology
Synopsis
- Outlining novel mRNA-LNP platform for mRNA therapeutic antibody production
- Exploring therapeutic antibody potential from discovery to pre-clinical development
- Overcoming key challenges of in vivo protein expression for successful translation of mRNA-based therapeutics
12:45 pm Unlocking the Future of RNA Based Vaccines: Tebu-bio’s Production Solutions
Synopsis
- Implementing mRNA mini-scale production for preclinical studies
- Developing mRNA encapsulation in LNP for therapeutic applications
- Assessing the effect of mRNA in physiologically-relevant cell models
TRACK B: CLINICAL DEVELOPMENT & MANUFACTURING
Synopsis
Chair: Ruben Rizzi, Senior Vice President Global Regulatory Affairs, BioNTech
Deciphering a Regulatory Framework & Guidance for Future mRNA Platforms to Harmonise Clinical & Manufacturing Development
11:15 am The Value of a Harmonised Regulatory Framework for mRNA Medicines
Synopsis
- mRNA platform technology approaches supported by regulatory provisions
- Diverse regulatory understanding of platform technology from various perspectives
- Examples of platform technology – platform technology approaches today and in the future
11:45 am Swiss Regulatory Aspects & Evaluation for mRNABased Vaccines & Therapeutic Pathways to Approval
Synopsis
- Exploring harmonisation regulatory challenges and expectations for new mRNA drugs applications and documentation
- Delineating the mRNA platform definition for vaccine, therapeutic and broader applications
- Highlighting regulatory progress in personalised mRNA cancer vaccines and manufacturing considerations
12:15 pm Fireside Discussion: Leveraging the mRNA Platform to Advance Development & Manufacturability Successfully at Clinical Stages
Synopsis
- What is required to ensure mRNA vaccine development at speed with a coordinated effort?
- How to accelerate mRNA vaccine process manufacturing to combat future pandemics?
- How can we apply learnings to promote global access to mRNA technology that will be key for pandemic preparedness?
12:45 pm Technology Transfer of mRNA/LNP Platform, from CMC Development to Process Performance Qualification
Synopsis
- Case Study: Transfer of self-amplifying mRNA Covid-19 vaccine in Japan and pandemic readiness
- Good practices to ensure a successful technology transfer
- Key points to select a CDMO partner
12.55 Lunch Break & Networking
TRACK A: DISCOVERY & PRE-CLINICAL DEVELOPMENT
Synopsis
Chair: Nigel Horscroft, Chief Technology Officer, Stealth Mode mRNA Biotech
Turbocharging new mRNA-LNP Programs to Expand Therapeutic Potential with European Research Initiatives & Collaborations
2:00 pm REGeRNA Project: Identifying & Developing mRNAEncoded Proteins Towards Novel Therapeutics for Cardiac Tissue Regeneration
Synopsis
- Establishing the need for new treatments of heart failure
- Deciphering a rationale for an mRNA-based strategy for reinducing proliferation of cardiomyocytes Showcasing first results and remaining challenges
2:30 pm Multidimensional Characterisation of RNA-Based Vaccines & Therapies
Synopsis
- Evaluate potency through the identification of ribosome stalls that reduce protein production
- Examine purity by discovering the sequences and structures that lead to dsRNA by-products
- Measure safety through studies of off-target changes in transcription and translation
3:00 pm BAXERNA Project: On a Mission to Develop Novel Bacterial mRNA Vaccines
Synopsis
- Introducing BAXERNA’s aim to develop mRNA vaccines against bacterial infections like TB and beyond Showcasing the development of a novel antigen discovery to Phase I pipeline for fully realised bacterial vaccines
- Showcasing the development of a novel antigen discovery to Phase I pipeline for fully realised bacterial vaccines
- Unlocking LNP formulation, mRNA format, and analytical development for novel mRNA vaccines
3:30 pm EXPERT Project: Immunostimulatory Triplet mRNA Cocktail in Lipid Nanoparticles for Cancer Therapy
Synopsis
- Developing a new LNP with a mRNA-encoded cytokine mix
- Demonstrating scaled-up LNP production and characterization
- Unleashing new delivery system on the horizon: Cell penetrating peptides and extracellular vesicles
TRACK B: CLINICAL DEVELOPMENT & MANUFACTURING
Synopsis
Chair: Andreas Kuhn, Senior Vice President – RNA Biochemistry & CMC, BioNTech SE
Forging New Commercial Partnerships to Ensure Smooth Transfer into Trials & Manufacturing at Scale
2:00 pm Unlocking mRNA Vaccines: From Platform to Products
Synopsis
- Developing an mRNA competitive platform requires a high level of collaboration between designers and developers of mRNA and LNPs
- Evolving from platform to product needs to build the desired biological performance and the CMC performance into the design of mRNA and LNPs
- Data science is the continuum that provides acceleration to the overall mRNA Vaccines development
2:30 pm Vinylphosphonic Acid Modified Cap Analogs and Their Performance in mRNA Vaccines
Synopsis
- Development of Vinylphosphonic Acid Modified Cap GAG (CAP5011) and GAU (CAP1811)
- Synthesis of mRNA using CAP5011 and CAP1811, and encapsulation in LNP
- Testing of mRNA capped with CAP5011 and CAP1811, including protein expression, vaccine induced immune response in vivo and mRNA safety in NHP.
3:00 pm Panel Discussion — Finding the Right Partnership to Build a Robust & Trusting Relationship to Accelerate mRNA Development Into the Clinic
Synopsis
The mRNA community thrives on strategic alliances, which allow for cross-functional sharing of knowledge, skills, and experience across different disciplines to truly advance mRNA technologies to accelerate drug development to patients faster.
This panel will explore:
- How to overcome the complexities of decision-making and cultural boundaries for developing, acquiring, licensing and advancing innovative approaches
- Where are the gaps when using mRNA approaches that the service provider, biotechnology and academic sector can fill?
- Developing successful expertise in house vs external sourcing management vs asset acquisition partnering strategies
4.00 Afternoon Break & Poster Session
Synopsis
As the landscape of innovation that is supercharging the next wave of efficacious mRNA vaccines and therapeutics, it is more important than ever to collaborate and learn for the growth of this field. Join our dedicated poster session to share your latest data and get the first look into what your peers are working on!
Accelerating Effective & Safe Personalised mRNA Cancer Immunotherapies Towards Clinical Success & Achieving Market Approval
4:30 pm Advancing RNA Therapeutics Development: Novel Reagents for DNA Template Production by Rolling Circle Amplification
Synopsis
- The rolling circle amplification (RCA) is an isothermal amplification technique that shows promise for generating DNA template for in vitro transcription (IVT) reaction used in therapeutic RNA production. Compared to using bacterial plasmids as DNA templates, RCA offers several advantages as speed, simplicity and safety
- For mRNA therapeutics development the AOF (animal origin free) reagents are of interest due to the reduced risk for impurities associated with animal origin materials. Recently, we have developed the TheraPure 29 DNA polymerase (AOF), and investigated the enzyme’s performance under various RCA conditions, including different reaction times, temperatures, different sizes of DNA templates. RCA generated DNA template was used for IVT reaction and compared to plasmid DNA. RCA scalability was evaluated
- Our results support the use of TheraPure 29 DNA polymerase and other Thermo Fisher Scientific reagents for DNA template preparation by RCA for RNA based therapeutics development
5:00 pm Presenting MHRA’s Perspectives & Draft Guidance on the Licensing of Individualised mRNA Cancer Immunotherapies
Synopsis
- Individualised mRNA cancer immunotherapies (cancer vaccines) pose new regulatory challenges since the mRNA sequence is matched to the patient’s unique tumour neoantigen profile and HLA type
- The MHRA aims to publish draft scientific and regulatory guidance for developers of individualised mRNA cancer vaccines by the end of 2024. This will include sections on regulations, manufacturing, non-clinical, clinical and post-authorisation aspects
- An overview of the key points will be presented
5:30 pm Panel Discussion – A European Industry & Regulatory Perspective on the Development & Approval of mRNA Medicines
Synopsis
- Uncovering how regulatory provisions can support mRNA medicines’ approval
- Reducing the regulatory approval timeline through platform technology approaches
- Moving towards a harmonized approach for mRNA medicines for the benefit of patients