Conference Day One
7:20 am Check-in & Coffee
Showcasing Clinical Updates to Accelerate the Next Wave of mRNA Vaccines for Infectious Diseases & Oncology
8:20 am Chair’s Opening Remarks
8:30 am Local Delivery of mRNA Therapeutics for Treatment of Respiratory Diseases
Synopsis
- Unlocking local delivery of mRNA therapeutics into the upper and lower respiratory tract to enable production of the target protein, directly at the site of desired pharmacological activity
- Addressing the technical challenges of inhaled mRNA delivery and how to overcome them
- Revealing the first clinical trial results using mRNA for immunomodulation in the respiratory tract
9:00 am Updates & Interim Data of mRNA-Based Cancer Vaccines to Treat EBV-Related Cancers
Synopsis
- Present breakthroughs in the treatment of EBV-related cancers, including NPC and NKT lymphoma
- Highlight safety and efficacy based on ongoing enrollment and interim data
- Demonstrate the transformative potential of mRNA therapeutics in the treatment of viral-related malignancies
9:30 am Optimised Prime: A Transformative RNA-LNP Vaccine Composition that Scales
Synopsis
- LNP formulation and physicochemical characteristics with both messenger RNA (mRNA) and self-amplifying RNA (saRNA)
- In vivo immunogenicity of RNA-LNP vaccines against influenza A and SARS-CoV-2
- Overcoming scale-up and process development challenges in vaccine development
10:00 am Morning Break & Speed Networking
TRANSLATIONAL DEVELOPMENT TRACK
Chair Moderation:
Unlocking Novel RNA Technology Approaches to Expand Vaccine & Therapeutic Application
11:00 am Extracellular Vesicles (Exosomes) Spread the Lipid Nanoparticle(LNP)-mRNA Between Cells: Do Exosomes Change the Fate of mRNA-Vaccines in Recipients?
Synopsis
- After internalization (endocytosis) of mRNA-LNPs, the mRNA of LNPs is repacked into exosomes via endosomal-exocytosis route and transported to neighbouring cells or distant organs
- All cells at the administered area do not receive mRNA directly from LNPs but part of the mRNA of LNPs is distributed by exosomes, thus, exosomes contribute to expression of proteins or antibodies from this mRNA
- Exosomes carrying the mRNA of LNPs, transport this mRNA to organs differently than delivery via LNPs themselves, both for function and inflammatory response
11:30 am Droplet Digital PCR – A Key Tool for Advancing mRNA Therapeutics
Synopsis
- The emergence of mRNA therapeutics offers novel treatment options for numerous diseases. However, the development and optimisation of mRNA therapeutics require precise and reliable methods for quantifying mRNA expression and assessing treatment efficacy
- Droplet digital PCR (ddPCR) has emerged as a powerful tool for mRNA therapeutics development, offering unparalleled sensitivity, precision and absolute quantification capabilities
- Investigating how ddPCR is helping to transform the development process of cell, gene and mRNA therapies and how it is rapidly replacing traditional qPCR methods
12:00 pm Trans-Amplifying RNA as a Versatile Vaccine Platform Having Unique Advantages for Improving Pandemic Preparedness
Synopsis
- Showcasing in vitro taRNA development and optimisation
- Sharing In vivo experiences with taRNA
- Providing future perspectives on the potential contribution of taRNA for improved pandemic preparedness
12:30 pm Innovations in RNA+LNP Drug Product Manufacturing: The Journey from Lab Formulation to GMP Production
Synopsis
- Exploring Cutting-Edge RNA+LNP Formulation Techniques: Discover how our proprietary LNP manufacturing platform, the Nova BT IJM system, revolutionises the art of lipid nanoparticle formulation with an optimal turbulent mixing approach
- Bridging the Gap: Learn how we excel at transitioning drug products from concepts to initial laboratory formulation to scalable GMP production, with a special focus on helping clients migrate from microfluidic processes
- Advantages and Implications: Gain insights into the advantages of our innovative approach and the cost-effectiveness it brings, especially for complex formulations and next generation RNA+LNP drug products
CMC
TRACK
Chair Moderation:
TRACK
Translating CMC Methods to Advance mRNA Products from Bench to Clinic with Ease
11:00 am Advancing CMC Strategies for Self-Amplifying RNA to Achieve a Transition from Discovery through to Clinic
Synopsis
- How to optimise CMC methods for self-amplifying RNA whilst de-risking failures
- Examining platform requirements for a scalable production process to meeting the needs at each phase
- Maximising volume and speed to ensure future clinical success
11:30 am Democratising mRNA Therapeutics with Novel Cap Analogs & Modified NTPs
Synopsis
- Showcasing novel cap analogs and their discovery
- Unravelling novel modified NTPs
- Leveraging mRNA capping assays and paired capped/ upcapped mRNA standards
12:00 pm mRNA-Based CRISPR/Cas9 Delivery for Gene Editing in Muscle Stem Cells
Synopsis
- Exploring muscle wasting and muscular dystrophy
- Assessing clinical trial, ATMP and regulatory considerations
- Advancing CMC and ex-vivo editing
12:30 pm Reduction of Double-Strand mRNA in Process Development
Synopsis
- dsRNA is a fatal immunogenic impurity generated through mRNA IVT process
- Highlighting the importance to reduce the dsRNA to a safe range for large dose or repeated dose of mRNA-based therapeutics
- CATUG DS100 and DS20 platform emphasises different strategies to control dsRNA <100ng/mg or even <20ng/mg
12:40 pm Lunch Break & Networking
Translating Preclinical to Clinical Success in the Context of mRNA-Based Therapeutics & Vaccines
Chair Moderation:
1:45 pm Beyond COVID Vaccines – mRNA Therapeutics & Lean Strategies for their Non-Clinical (Safety) Assessments
Synopsis
- Introduction into special considerations for mRNA therapeutics with regards to their non-clinical PD/DMPK and safety assessments
- Introduction of the platform toxicology concept
- Challenges and opportunities for mRNA therapeutics in the non-clinical (safety) space
2:15 pm Harnessing mRNA Therapeutic Platforms to Accelerate Product Development
Synopsis
- Describing different platform components from design to manufacturing, and their importance to accelerating product development
- Leveraging critical elements in protein and mRNA design to drive clinically relevant PK and purity behaviours in vivo
- Transitioning from in vitro to rodents to non-human primates to best predict clinical efficacy in heterogeneous populations
2:45 pm Impingement Jets Mixing for Scalable Production of Lipid Nanoparticles
Synopsis
- Showcasing KNAUER’s NanoProducer Systems with Impingement Jet Mixing have shown great success for large-scale production of mRNA vaccine
- Successfully using laboratory Scale NanoScaler for method development and small batches
- Achieving scalability between both ranges with our range of Impingement Jets Mixers
Unravelling mRNA-Delivery Thermostability to Produce Best-In-Class Drug Formulations
Chair Moderation
1:45 pm Combating Thermostability with the World’s First Approved Lyophilised mRNA-Based Vaccine
Synopsis
- Developing the first self-amplifying mRNA vaccine to gain European approval
- Benchmarking the convenience of 2-8 degrees Celsius storage and supply, pharmacoeconomics and reachability
- Advancing needle free delivery
- Showcasing self-sufficiency of raw materials, ease of manufacturing and stockpiling
2:15 pm A Novel Unit Operation Successfully Drying mRNA/ LNP by Mass Transfer
Synopsis
- Presenting a unique LaminarPace ambient drying, based on mass transfer principle
- Turning liquid biopharmaceuticals into thermostable dry powders
- Examining how the well-defined powder may be reconstituted or used for novel administration routes
2:45 pm Advances in Development of Thermostable mRNA-LNP Formulations
Synopsis
- Showcasing development of the first mRNA vaccine developed by Abogen in China, but approved in Indonesia for emergency authorisation use
- Sharing technical insights into Abogen’s mRNA COVID vaccine which can be stored at 2-8 degrees Celsius whilst similar vaccines from BioNTech-Pfizer and Moderna require much lower storage temperatures of -70 and -20 degrees Celsius
- Highlighting Abogen’s recent progress in development of frozen and lyophilised mRNA-lipid nanoparticle formulations for conventional use
3:15 pm
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 the mRNA field. Join our dedicated poster session to share your latest data and have first look into what your peers are working on!
Delving into Novel Applications of mRNA-Based Therapeutics to Expand Potential Beyond Infectious Diseases
4:00 pm Small but Mighty: How Digital Technology can Support Small Batch Manufacturing of mRNA Therapeutics
Synopsis
- Small and flexible manufacturing allows optimized solutions for personalized therapeutics and small-batch manufacturing
- Digital technology can support mRNA therapeutics by helping to accelerate batch release and improve compliance
- Using paper during development can carry hidden costs
4:30 pm Targeted mRNA Therapeutics for Treatment of Kidney Diseases
Synopsis
- Mercurna’s technology platform combines unique cell-targeting moieties, with a lipid-based delivery vehicle to deliver engineered mRNA with high specificity
- Active targeting strategies through optimised technology on targeted LNPs
- Showcasing examples on how Mercurna leverages its platform for development of targeted mRNA therapeutics to treat kidney diseases
5:00 pm Synthesis of Nucleic Acid Therapeutics – End-to-end Workflow & Process Optimisation
Synopsis
- Phi29 DNA polymerase utilizes rolling circle amplification to produce cell-free linear DNA template for RNA synthesis with the flexibility to work at the RUO and GMP grade
- Therapure IVT reagents were able to consistently synthesise RNA at multiple volumes with Therapure GMP reagents reaching 1 gram of RNA at the 100 mL synthesis scale
- Design of experiments were used to determine the buffer components and reagent proportions that result in optimal IVT performance
5:30 pm Delivery of Synthetic Tropoelastin mRNA for the Regeneration of Elastic Tissues in Rare Diseases
Synopsis
- Showcasing protein replacement therapy using synthetic mRNA
- Accelerating mRNA therapeutics for rare diseases
- Unleashing targeted delivery of synthetic TE mRNA