8:00am | Coffee & Networking

8:45am | Chair’s Opening Remarks

Optimising Analytical Development for Nucleic Acid-based Vaccines: mRNA & DNA Vaccines

9:00 am Analytical Characterization of mRNA Impurities in LNP Delivery Systems

Synopsis

• Recent advances towards mRNA production and delivery prompt a need for robust analytical methods capable of characterizing this new class of drugs.

• In this case study, the analytical characterization of a novel class of lipid-modified mRNA impurities that impact mRNA activity in LNP delivery systems will be discussed.

9:30 am mRNA-lipid Nanoparticle COVID-19 Vaccines: Structure & Stability

  • Daan Crommelin Professor Emeritus of the Dept Pharmaceutics, Utrecht Institute for Pharmaceutical  Sciences, UIPS

Synopsis

• Understanding the root cause of mRNA instability to help rationally improve mRNA-LNP product stability

• What is the structure of mRNA-lipid nanoparticles?

• What can be done to increase stability of the mRNA vaccines?

10:00 am An RNA/ DNA-carrying and DC targeting nanocarrier with intrinsic co-stimulatory effects and metrics of immunomonitoring

10.30am | Morning Break

Optimising Analytical Development for Nucleic Acid-Based Vaccines: mRNA & DNA Vaccines

11:00 am Nice-and-easy nanoparticle characterization with Stunner

Synopsis

•Check out how Stunner delivers one-step quant & sizing for any nanoparticle

•See how UV/Vis can quantify any nanoparticle payload, without dyes or standards

•Learn how to get tons of size and aggregation data with high-throughput DLS, from just 2 uL

11:15 am Panel Discussion: Advancing the Pharmaceutical Science of Lipid Delivery Systems and their Translation into the Clinic

  • Aalok Shah Director, Formulation & Drug Delivery , Strand Therapeutics
  • Daan Crommelin Professor Emeritus of the Dept Pharmaceutics, Utrecht Institute for Pharmaceutical  Sciences, UIPS
  • Meredith Packer Scientist, Process & Analytical Development , Moderna

Synopsis

• Despite huge advances made in mRNA vaccines, the development of LNP-based therapeutics and vaccines still presents unique and significant pharmaceutical and regulatory challenges.

• This panel will discuss the specific challenges associated with the pharmaceutical development of lipid-based therapeutics, along with discussion on the biophysical design and characterization capabilities, as well as the engineering expertise required to build the fundamental understanding for successful clinical development.

12:00pm | Lunch

1:30 pm Lyophilization of Lipid-Nucleic Acid Complexes

  • Tom Anchordoquy Professor, Pharmaceutical Sciences, Skaggs School of Pharmacy and Pharmaceutical Sciences, University of Colorado

Synopsis

• Stability optimization during acute freezing and drying stress

• Identifying meaningful methods for assessing recovery

• Stability optimization during storage

2:00 pm Optimized Plasmid Design & Delivery with Robust Analytical Methods for DNA-based Vaccines

  • Jean Boyer Vice president, Analytical Sciences , INOVIO

Synopsis

• Insight into INOVIO’S DNA-based technology to show induction of virus-specific cellular and humoral immune responses.

• Insight into the analytical methods used to allow for optimized design and development of a wide range of DNA-based vaccines.

2:30 pm Analytical Insights Supporting the Development of a CoVID-19 saRNA LNP Vaccine

  • Adam Crowe Manager, Analytical Development, Precision NanoSystems

Synopsis

• Precision NanoSystems Inc.’s (PNI) self-amplifying RNA (saRNA) offers numerous advantages over smaller mRNA payloads in SARS CoV2 LNP vaccines due to its low therapeutic dose and prolonged antigen expression in vivo; however, its size (>9000 nt) poses unique analytical challenges requiring innovation
• Assessment of saRNA quality attributes including integrity, 5’ cap structure, and potency was accomplished by adapting methodologies for capillary electrophoresis (CE), LC-MS, and in vitro expression from a model eGFP saRNA, respectively
• Further analysis of the LNP drug substance’s excipient content, sizing, and payload encapsulation have provided vital insights into the development of PNI’s SARS CoV2 LNP vaccine

3:00pm | End of Day One