ST Pharm is mostly known for global API manufacturing company and has a strong foundation of manufacturing nucleotides and oligonucleotides. In parallel to oligonucleotide CDMO business, ST Pharm has expanded its business to another gene therapy area, the mRNA. Since 2018, ST Pharm has set up its own mRNA platform technology including novel 5’-capping reagent SMARTCAP® and lipid-nanoparticle drug delivery system SmartLNP®. These mRNA technologies enable ST Pharm to expedite the development of mRNA vaccine as well as mRNA CDMO business. In this presentation, potency of ST Pharm’s patented SMARTCAP® and delivery efficiency of SmartLNP®, and further capability and capacity as mRNA CDMO will be presented. 

Speaker:

Joo-Sung James Yang, Ph.D.
VP, Head of mRNA R&BD, Head of Biotechnology R&D
ST Pharm Co. Lt d.

Optimizing the IVT reaction for high mRNA yield requires construct-specific considerations. At first the IVT reaction might seem simple compared to cell derived samples, containing mostly nucleotides and their by-products. Effective mRNA process development needs to separate & identify these species, so they can be tracked & removed. The PATfix mRNA analytical platform is specifically designed to tackle these challenges.

Speaker:

Blaž Bakalar
Product manager PATfix platform
BIA Separations, now part of Sartorius

AstraZeneca has a rising number of polynucleotide and mRNA therapy projects in the portfolio. This presentation will focus on CMC analytical approaches for mRNA in a buffer solution formulation. The quality attributes and approaches for release and stability testing will be outlined. We will describe the main impurities and degradation pathways to consider for a drug product. For mRNA, many structures related impurities pose a challenge, and we will share cases of separation-based methods for characterization of mRNA.

Speaker:

Eivor Örnskov, Ph.D.
Principal Scientist, Pharmaceutical Sciences
BioPharmaceuticals R&D, AstraZeneca

Though much of the regulatory filing requirements for mRNA-based therapeutics can be compared with those for traditional biologics, others are not yet well defined. Like traditional biologic products and processes, mRNA-based products require controls on the quality of the starting material (including cell banking), consistency of manufacturing processes, identification of critical process parameters, clearance of impurities, establishment of environmental controls to meet safety requirements, and the thorough characterization of structure, purity, and efficacy. This has posed some challenges in analytical method development to ensure qualification for intended use. We discuss some of these challenges and solutions to support the requirements for characterization of structure, purity, and efficacy of an mRNA Drug Substance.

Speaker:

Khaled Yamout
Senior Director Quality Control
TriLink BioTechnologies

 Lipid nanoparticles (LNPs) are the leading vehicles for mRNA therapies as the approved SARS-CoV-2 vaccines. In order to design safe and effective LNPs, we investigated their structure in the absence and presence of proteins using high resolution techniques as small angle neutron scattering. Apolipoprotein E shows high binding affinity towards LNPs and induces lipid redistribution. These findings are relevant to understanding cellular mechanisms involved in LNP delivery as endosomal escape.

Speaker:

Marianna Yanez Arteta, Ph.D.
Associate Principal Scientist, Pharmaceutical Science
Advanced Drug Delivery