mRNA Technology & Innovation Day

7.00 Check In & Morning Coffee

8.00 Workshop A

Optimising Non-Viral mRNA-Loaded Formulations to Improve Immunogenicity & Route of Administration

  • Roland Brock Professor of Biochemistry, Radboud University Medical Center
  • Neill Liptrott Professor of Pharmacology & Therapeutics, University of Liverpool
  • Samir El-Andaloussi Professor of Biomolecular Medicine & Advanced Therapy & Co-Founder, Karolinska Institutet & Evox Therapeutics

Synopsis

Drug delivery remains the biggest challenge to truly unlocking mRNA potential, yet the current delivery systems in the market do not offer effective targeting, reduced immunogenic responses and ultimately not enough clinical validation for new therapies and vaccines.

With the rise of creative LNP formulations, modifications and structures along with novel carriers from peptides and extracellular vesicles offering more choice, this workshop will help you overcome the bottlenecks in translatability and targeting of mRNA drugs to advance from research to clinic.

Case-Study Examples Include:

  • How to develop the ultimate LNP toolkit latest methods for routes of administration to ensure delivery success every time
  • Unlocking peptide-based nanoparticles to understand and advance mRNA delivery
  • Development of engineered exosomes for delivery of RNA therapeutics and gene editors

10.00 Morning Break & Networking

10.30 Workshop B

De-LIVER – Solving Extra-Hepatic Targeting Bottlenecks With Delivery to Combat Rare Diseases

Synopsis

Recent advances in mRNA technology and its delivery have enabled mRNA-based therapeutics to enter a new era in medicine with unlimited potential. The rapid, potent, and transient nature of mRNA-encoded proteins, without the need to enter the nucleus or the risk of genomic integration, makes them desirable tools for treatment of a range of diseases.

Nonetheless, challenges remain with regards to mRNA expression, delivery efficiency, and targetability, to broaden the applicability of mRNA therapeutics. This workshop brings together key experience from novel proof-of concept and pre-clinical programs venturing into new target areas including the kidney and lung to optimize mRNA platforms to meet clinical needs of each disease.

Case-Study Examples Include:

  • Strategies for active targeting of LNPs to the kidney
  • Respiratory delivery of mRNA – overcoming hurdles for therapeutic application

12.30 Lunch Break & Networking

1.30 Workshop C

mRNA & Gene Editing – Delineating the Convergence of mRNA’s Potential as a Tool to Treat Genetic Diseases

Synopsis

With the UK approval of the world’s first gene-editing treatment for blood cancer, the race is now on to develop next-generation gene editing technologies leveraging mRNA technology with the mission to develop curative therapies for some of the most challenging genetic diseases.

With a convergence of academic and industry collaborations to focus on bringing these drugs to patients with minimal toxicity and affordability, this workshop will deep-dive into novel gene editing technology and delivery with the mRNA payload at center focus to truly unlock future applications of this technology.

Case-Study Examples Include:

  • Unleashing RNA-based gene editing and delivery to minimize toxic effects for improved therapies
  • Achieving low-cost mRNA-based gene therapies for retinal diseases

3.30 Afternoon Break & Networking

4.00 Workshop D

High Quality mRNA at Low Cost – Improving the Process to Design & Produce mRNA Efficiently

  • Zach Zhu Chief Technology Officer, Innorna
  • Marcus Wilhelmsson Professor of Physical Chemistry, Chalmers University of Technology
  • Zoltán Kis Associate Professor – Department of Chemical & Biological Engineering, University of Sheffield

Synopsis

With the rise in discovery tools, sequence optimization and process advancements to accelerate and economise drug development, the mRNA field is undergoing a radical transformation to improve mRNA vaccine and therapeutic production. However, there remain significant challenges to adopt and translate this knowledge into real-world applications and to leverage these platforms with the potential to power a new era of mRNA medicines.

This workshop will leverage innovative solutions in mRNA labelling, design optimisation and product digitalisation to drive the future mRNA medicine development with application examples from concept to clinic.

Case-Study Examples Include:

  • Harnessing fluorescence labeling methods for RNA-based therapeutics
  • Deep-diving on mRNA capping technologies
  • Novel cost reduction strategies, intensified, continuous and digitalised production of high-quality mRNA medicines at low cost 

6.00 End of mRNA Technology & Innovation Day