Conference Day Two

8:30 am Check-In & Breakfast

9:15 am Chair’s Opening Remarks

  • Fethi Bensaid Head Of Chemistry, Manufacturing, Controls Drug Product Development & Industrialization, mRNA Center of Excellence, Sanofi

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

  • NING DING Researcher, King's College London

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

  • Regina Bleul Head of Nanomedicine group, Fraunhofer ICT-IMM

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

  • Fethi Bensaid Head Of Chemistry, Manufacturing, Controls Drug Product Development & Industrialization, mRNA Center of Excellence, Sanofi

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.

3:30 pm Chair’s Closing Remarks

  • Fethi Bensaid Head Of Chemistry, Manufacturing, Controls Drug Product Development & Industrialization, mRNA Center of Excellence, Sanofi

3:45 pm End of 4th Lipid Nanoparticle Development Summit Europe