Conference Day Two
8:30 am Check-In & Breakfast
9:15 am Chair’s Opening Remarks
Bridging the Gap from Bench to Body: Advancing In Vitro & High-Throughput Screening Models to Accelerate Translational LNP Development
9:20 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
9:30 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?
10:00 am 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
10:30 am 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
11:00 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
11:30 am Morning Break & Networking
Ensuring Smooth Transition from Small Scale to GMP Through Advanced Mixing Technologies & Process Analytical Tools for Accelerated Consistent Manufacturing
12:00 pm Selection Process & Steps to Scale LNP Formulation into the cGMP: Challenges & Mitigation From a Process Perspective
Synopsis
- How to select which LNP to scale: in vivo, in vitro data, LNP stability
- Characterization throughout the process development
- FDA interaction and stability of scaled LNP
12:30 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
1:00 pm Lunch Break & Networking
2:00 pm mRNA-LNP Development, Scale-up & Stability: Challenges & Opportunities
Synopsis
- Overview of mRNA-LNP specific quality attributes and mRNA-LNP development challenges
- Discussing the main scale-up, manufacturing process and thermostability challenges
- Understanding how to define a robust and holistic control strategy
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.