mRNA Technology & Innovation Day | Tuesday, 28th January
7.00 Check In & Morning Coffee
8.00 Workshop A
Optimising Non-Viral mRNA-Loaded Formulations to Improve Immunogenicity & Route of Administration
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
9.45 Morning Break & Networking
10.15 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
11.45 Lunch Break & Networking
12.45 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
2.15 Afternoon Break & Networking
2.45 Workshop D
High Quality mRNA at Low Cost – Improving the Process to Design & Produce mRNA Efficiently
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
4:42 pm 4.45 Workshop E
Unlocking the Potential of mRNA within a Collaborative European Ecosystem
Synopsis
YOUR ROADMAP TO EUROPEAN PARTNERSHIPS
The EU is actively building an ecosystem to support the development and commercialisation of RNA therapeutics through various initiatives, funding opportunities, public-private collaborations, infrastructure support and expert networks. Health biotechnology or biopharmaceutical R&I is supported substantially by the EU R&I Framework Programmes, with over 1,250 Horizon 2020 and nearly 600 Horizon Europe projects to date. In March 2024, the European Commission reaffirmed the importance of biotechnology in strengthening the EU’s competitiveness and strategic autonomy and proposed a series of targeted actions to boost biotechnology and biomanufacturing across the EU.
The goal of this two-hour interactive workshop with panellists from industry and academia, organised by the Horizon 2020 project “EXPERT”, is to:
- Provide a roadmap and raise awareness of EU resources supporting development and commercialisation of RNA therapeutics
- Identify current challenges in efficiently utilising the existing infrastructure and services
- Provide user feedback and recommendations to relevant stakeholders
- Showcase main insights and outputs from EU-funded collaborative R&I projects
- Explore – from different perspectives – the approaches, challenges, and issues regarding exploiting the value generated by mRNA R&I projects
- Facilitate collaboration with existing projects and develop synergies with European, national or regional initiatives, funding programmes and platforms