Conference Day Two
8:00 am Check-In & Breakfast
9:00 am Chair’s Opening Remarks
Bridging the Gap from Bench to Body: Advancing In Vitro & High-Throughput Screening Models to Accelerate Translational LNP Development
9:15 am Innovative mRNA LNPs with Superior Safety for Vaccination Against Viral Infections
Synopsis
- Presenting the NeoVac library of innovation ionizable lipids and LNPs
- Tailoring activity, organ targeting and immunogenicity according to the intended
- intervention
- Revealing data from a clinical phase I/II study which demonstrate activity and superior safety
9:45 am Predictive in vitro Profiling of LNP-Induced Innate Immune Responses Using an iPSC-Derived Monocyte Model
Synopsis
- How can we accelerate translatable in vitro screening to efficiently identify promising LNP candidates?
- Maximising in vivo screening efficiency by testing multiple LNP candidates using minimal animal models
- What are the current limitations of in vitro models particularly in predicting the immune responses?
10:15 am Roundtable Discussion: The Future of LNP Technologies – Navigating the Frontier
Synopsis
Exploring how to ensure the continued success and adoption of LNP mRNA technologies beyond the initial COVID-19 breakthrough
Discussing the potential of using LNPs in other disease areas beyond infectious diseases, such as cancer vaccines and distal site delivery
Examining how AI can contribute to the development and optimization of LNPs?
10:45 am Morning Break & Networking
11:15 am Anion Exchange Chromatography: A specific Alternative to Determining mRNA Encapsulation Efficiency in Lipid Nanoparticles
Synopsis
- Increased accuracy due to LC method specificity
- Comparison with reference fluorescence method, providing additional insights to mRNALNP characterisation
- A quick and robust approach to encapsulation efficiency monitoring, readily applied to QC GMP environments
11:45 am Built to Deliver: LEON’s Scalable Nanoencapsulation Technology
Synopsis
- At the core: LEON’s FR-JET® modular mixer enables superior process control and product quality
- Maximum consistency: seamless scalability of nanoencapsulation processes from R&D to commercial manufacturing
- A new approach: LEON’s NANOme® for precision medicine
12:15 pm Microfluidic Engineering of Milk Extracellular Vesicles for Oral Delivery of siRNA
Synopsis
- Engineering of milk extracellular vesicle (mEV)-lipid nanoparticle (LNP) hybrids using microfluidics for efficient siRNA oral delivery, overcoming RNA loading limitations of natural EVs
- Demonstration of improved safety, distinct uptake pathways, and enhanced delivery efficiency of hybrid particles compared to conventional mEVs and LNPs
- In vivo biodistribution showing promising colon-targeted accumulation after oral administration, highlighting potential for future clinical translation
12:45 pm Lunch Break & Networking
Ensuring Smooth Transition from Small Scale to GMP Through Advanced Mixing Technologies & Process Analytical Tools for Accelerated Consistent Manufacturing
1:45 pm Exploring Microfluidic Mixing & Alternate Methods to Ensure Equipment Versatility in the Route to GMP
Synopsis
- Are there any alternate methods or variations of current industrial scale methods for mixing which allow for automation?
- Unpacking how microfluidic or alternative mixing methods can fit into continuous flow processes to increase throughput and reduce batch-to-batch variability
- Innovations in real-time monitoring and control of mixing parameters to ensure product consistency
2:15 pm Stealth Lipid-Free LNPs: A New Technology that Offers Colloidal Stability and in vivo Potency, While Sidestepping PEG-Related Liabilities
Synopsis
- Eliminating the need for PEG-lipids by using sodium triphosphate (3P) to achieve colloidal stability, reducing risks of hypersensitivity, anti-PEG antibodies, and accelerated blood clearance
- Preserving essential particle characteristics, such as size, morphology, and polydispersity, while modifying surface charge through 3P binding
- Delivering targeted liver expression with minimal off-target activity and outperforms PEG-LNPs in photon flux six hours post-dose in vivo
- Demonstrating strong tolerability in mice, supporting the use of 3P-LNPs as a viable PEG-free vector for vaccines and the potential to enable chronic intravenous gene replacement therapies
2:30 pm Overcoming the Challenges of Pulmonary RNA delivery: Spray-Dried LNPs as Thermostable & Inhalable Therapeutics
Synopsis
- Gain a deeper understanding of structural CQAs (Critical Quality Attributes) and the emerging analytical metrics, such as structural analogs to pKa, that enable more predictive and robust LNP characterization.
- Explore advanced analytical tools including light and neutron scattering, X-ray techniques, and novel assays for precise quantification of particle size, distribution, and internal structure.
- Learn how innovative quality control strategies go beyond routine HPLC-based checks to improve reproducibility and capture quality dimensions currently overlooked by standard methods.