LNP 101 Day: Monday, April 14

8.30 Check-In & Coffee With a Light Breakfast

9:20 am Chair’s Opening Remarks

  • Vinu Krishnan Principal Scientist - Research, Chemistry, Manufacturing Controls, Molecular Profiling & Drug Delivery, Abbvie

Deep Diving into the History, Early Successes & Key Components of LNPs to Gain a Solid Foundation for Leveraging LNPs to Deliver Therapeutics & Vaccines

9:30 am Delving into the History of Lipid Nanoparticles to Understand the Evolution of this Innovative Delivery Vehicle

Synopsis

  • Deep diving into the discovery of LNPs to understand how they were initially invented
  • Uncovering the initial formulations and purposes of LNPs through evaluation of the first patent filings which took place
  • Reviewing academic and pharmaceutical progress of LNPs, including the uncovering the development of the first FDA approved drug utilizing LNPs

10:00 am Roundtable Discussion: Uncovering the Roles of the Components in LNPs for Developing Stable Delivery Vehicles

  • Delai Chen Director, Nanoparticle Formulation, Beam Therapeutics

Synopsis

• Evaluating the 4 components in LNPs; ionizable lipid, phospholipid, sterol, and PEGylated phospholipid, for developing functional LNPs

• Exploring the potential of adding in a 5th component, such as polymers, to improve stability

• Improving targeting of LNPs with conjugates, peptides, and more for enabling extrahepatic delivery

10:30 am Evaluating the Fundamental Analytical Methods for Characterizing & Identifying Effective LNPs with Good Encapsulation Efficiency, Morphology & Stability

Synopsis

• Delving into the current go-to analytical methods for understanding and characterizing LNPs

• Uncovering the limitations and benefits of each analytical method for a better understanding of LNPs

• Utilizing Ribogreen assays and DLS to their full advantage for accurate measurement of particle size and encapsulation efficiency

11.00 Morning Networking Break

Gaining Knowledge on the Fundamental Strategies for Successfully Formulating & Manufacturing Traditional & Innovative LNPs

11:30 am Getting up to Speed on the Formulation Methods for Development of Traditional & Innovative LNPs

Synopsis

• Delving into the past, present, and future formulation development of LNPs

• Deep diving into the best formulation methods for reliably developing LNPs

• Revealing a successful roadmap for translating LNPs from concept to the clinic

12:00 pm Uncovering the Fundamentals for Manufacturing LNPs with Reliable Mixing, Filtration & Purification Techniques

Synopsis

  • Delving into generic routes for scaling-up LNPs
  • Uncovering the critical quality attributes which need to be considered to enable successful scale up
  • Evaluating the currently strategies for mixing, filtering and purifying LNPs

12:30 Lunch & Networking

Unleashing the Huge Potential of LNPs to Encapsulate Many Payload Types & Target Specific Cells for Developing Cures to a Large Disease Landscape

1:30 pm Advancing Preclinical Models to Accelerate the Clinical Translation of Cell Type -Specific LNPs

  • Tobias Raabe Research Assistant Professor, University of Pennsylvania

Synopsis

• Charting the progression of lipid nanoparticle (LNP) technologies through insights gained from preclinical mouse models

• Highlighting breakthroughs in organoid engineering that are accelerating the path from LNP design to clinical application

• Advancing cell-type-specific LNP delivery by integrating the strengths of both animal and human organoid models

2:00 pm Panel Discussion: Leveraging LNPs to Deliver a Range of Payloads to Treat a Wide Landscape of Disease

Synopsis

  • Highlighting the range of payloads being delivered by LNPs, including mRNA, siRNA, CAR DNA, gRNA and beyond
  • Showcasing the different characteristics and specifications required to be met by LNPs to deliver the different payload types
  • Uncovering the different challenges and opportunities it comes to using LNPs for both vaccines and therapeutics

2:45 pm Fast-Tracking LNP-Based Therapeutics – Insights from mRNA Vaccine Development

Synopsis

  • Translating the Success of mRNA Vaccines into Broader Therapeutic Applications
  • Applying mRNA-LNP Lessons to Accelerate Next-Gen Drug Development
  • Developing safe and scalable LNP therapeutics

3:15 pm Reviewing the Disease Applications of LNPs to Unleash their Full Potential for Treating a Large Variety of Patients

  • Annette Bak Head of Advanced Drug Delivery, AstraZeneca

Synopsis

  • Uncovering the potential of developing innovative LNPs for treating a wide landscape of disease
  • Delving into the current clinical trials happening using LNPs to deliver genetic medicine, cell therapy, cancer and autoimmune treatments
  • Discussing the bottlenecks faced when using LNPs for diseases outside of the liver

3:45 pm End of LNP 101 Day

  • Vinu Krishnan Principal Scientist - Research, Chemistry, Manufacturing Controls, Molecular Profiling & Drug Delivery, Abbvie