Lipid nanoparticle encapsulated RNA (RNA-LNP) vaccines have played a pivotal role in the global response to the COVID-19 pandemic and are now at various stages of development against a wide-range of other infectious diseases. Limited access to potent ionizable lipids, a key component in LNPs, and expertise for formulation and scale-up, present real barriers to entry in this field. To overcome these challenges, we developed and optimized a novel ionizable lipid and a corresponding LNP composition for the delivery of RNA in vaccine applications. In this webinar we will discuss the following aspects of RNA-LNP vaccine formulation.

1. Current landscape of genomic medicines and vaccines
2. LNP formulation and physiochemical characteristics with both messenger RNA (mRNA) and self-amplifying RNA (saRNA)
3. Current landscape of genomic medicines and vaccines
4. LNP formulation and physicochemical characteristics with both messenger RNA (mRNA) and self-amplifying RNA (saRNA)
5. In vivo immunogenicity of RNA-LNP vaccines against influenza A and SARS-CoV—2
6. Overcoming scale-up and process development challenges in vaccine development

Speakers:

Natalie Orr
Product Development Scientist
Precision NanoSystems

Manufacture of LNPs and other nanomedicines is one of the significant challenges in successfully getting these novel modalities rapidly to market. This talk will discuss some of the problems and outline possible solutions from early stage discovery to fully compliant GMP manufacturing.

Speaker:

Dai Hayward
CEO
Micropore Technologies Ltd 

Optimizing lipid nanoparticle manufacture can be complex given the vast number of variables. Oftentimes the change of one variable can change other variables concomitantly, making it difficult to isolate the effect of one variable. This presentation will detail considerations needed for initially developing a robust process, while keeping scale up feasibility in mind. Firstly, it would be ideal if a desired set of particle characteristics is known for a specific project. Topics include:

• Mixing step – what experiments can you do to learn your LNP?
  • Ways you can tailor particle size
  • Considerations for encapsulation efficiency of RNA
  • Mixing technology comparison
• Small vs large scale
  • Differences between small- and large-scale manufacturing
  • How to keep large scale in mind for a seamless transition
  • Dilutions
• Downstream processing
  • What state does your LNP need to be in for successful TFF?
  • Process parameters for TFF

Speaker:

Dr. Paul Stainton
Senior Scientist – Advanced Drug Delivery
AstraZeneca

As a class of therapeutics, peptide drugs are currently experiencing a significant growth. Nevertheless, from the sustainability standpoint these modalities suffer from the reliance on various hazardous substances as well as from the large amount of waste generated during manufacturing. In this regard, despite the numerous studies aimed at minimizing the footprint of peptide production, the role of quality of the starting materials on the overall sustainability of peptide manufacturing has been less investigated. Through a series of case stories, we will illustrate the profound impact of the quality of starting materials on the sustainability of industrial production of therapeutic peptides.

Speaker:

Jan Pawlas, Ph.D.
Scientist, Global Development
PolyPeptide

This session will describe a new method for tracking and labeling RNAs and RNPs within an intracellular context, called URIL tagging, based on our approach for targeting U-rich internal loops (URILs) in structured RNAs. URIL tagging is a versatile chemical approach to RNP tracking, labeling and degradation that has unique advantages in the investigation of RNA biology. This protected intellectual property is available for licensing and development into novel diagnostic platforms focused on RNP granule biology, which is dysregulated in the majority of neurodegenerative diseases such as ALS. Notably, there are few therapeutic avenues in this space and an RNA-centric strategy for RNP granule tracking, interrogation and diagnostic evaluation would be impactful within this challenging and underserved domain.  

Speakers:

Dennis Bong, PhD
Professor, Department of Chemistry & Biochemistry
The Ohio State University