This presentation will highlight accomplishments in RNA vaccine and therapeutic development, including innovations in cap structures, nucleotides, gene editing, and self-amplifying modalities. Explore how insights and lessons learned from early innovators can be applied to the development of clinic-bound constructs to continue to advance RNA medicines. 

Speaker:

Ben Hudson, Ph.D.
Associate Director of mRNA Research & Development
TriLink BioTechnologies

mRNA vaccines contributed significantly to controlling the COVID-19 pandemic, and as a result, numerous low- and middle-income countries have begun to acquire the know-how for production and to build mRNA vaccine manufacturing facilities. However, in the absence of routine manufacture and reliable sales most of these facilities will not be sustainable and as a result will not be ready to respond in the event of a future outbreak. R&D to apply mRNA technology to infectious disease targets of importance to the populations in LMICs is therefore required. The presentation will review the potential application of mRNA technology to a variety of infectious diseases and discuss the challenges to technical and regulatory success, as well as the probability of policy development and procurement for each of the targets.

Speaker:

Martin Friede, Ph.D.
Head, Vaccine Research Unit
World Health Organization

mRNA modalities are playing an increasingly important role in drug development. Key to their stability and translational performance is the chemistry of the 5’end cap. This presentation delves into innovative cap analog designs that enhance mRNA immune evasion and translation efficiency. The talk explains the role of 5’mRNA end capping in distinguishing self from non-self RNA and how various cap structures affect immune recognition. Using structural insights from literature studies, novel cap structures are proposed that are hypothesized to evade IFIT1 binding, thereby improving translation under immune stress. The efficacy of these designs is showcased through in vitro and in vivo models of inflammation, demonstrating their potential to improve mRNA-based therapeutic outcomes. Attendees will gain valuable knowledge on the rational design of mRNA cap analogs and their potential impact on RNA therapeutics.

Speaker:

David Butler
CTO
Hongene Biotech Corporation

Quality by digital design (QbDD) is an improvement over the quality by design paradigm; here, computational models are used to characterize, monitor, control, and improve manufacturing processes. In this session, we will look at the current mRNA-based product manufacturing and determine how the concepts/tools of QbDD can be utilized to improve it further. The mRNA manufacturing process has the inherent advantage of being disease agnostic, and this complements the principles of QbDD, especially with respect to the process of knowledge transfer from one product to another. Moreover, QbDD acts as the natural successor to the conventional quality by design paradigm in the era of bioprocessing 4.0.

Speaker:

 Adithya Nair
Post-Doctoral Research Associate (UK-SEA Vax Hub)
University of Sheffield