Explore the Agenda
7:30 am Check In & Morning Coffee
8:20 am Chair’s Opening Remarks
Showcasing the Blockbuster Potential of mRNA Beyond Vaccines to Usher in a New Era of Therapeutic Value
8:30 am Enabling Durable Gene Editing With Transient mRNA Delivery of Editing Machinery
- Engineering compact CRISPR-based gene and epigenetic editors that can be efficiently delivered as mRNA in vivo
- Optimising mRNA architecture and delivery to achieve sufficient intracellular editor expression from a transient dose
- Demonstrating how short-lived editor expression can generate long-lasting gene silencing or therapeutic effects without requiring persistent expression
9:00 am Session Reserved for ProBio CDMO
9:30 am Optimising Peptide-Based Nanoparticles for Functional RNA Delivery to Enable Therapeutic Gene Modulation & Protein Replacement
- Leveraging AI-driven peptide optimisation to design cell-specific targeting motifs and formulate peptide-based nanoparticles without antibody conjugation or additional surface modifications
- Improving targeting specificity and reducing off-target delivery through optimised peptide-based formulations, while supporting repeat dosing for therapeutic applications
- Demonstrating the versatility of peptide-based delivery through in vivo data on T-cell modification for CAR-T applications and protein replacement across tumour models
10:00 am Morning Break & Speed Networking
Join our speed networking session tailored for mRNA and delivery experts, to connect with industry peers and facilitate rapid yet meaningful exchanges of insights and expertise. Elevate your networking experience during this session designed for impactful connections among mRNA experts and stakeholders.
RESEARCH & PRECLINICAL DEVELOPMENT
Demonstrating Therapeutic Proof of Concept Through Preclinical & Clinical mRNA Development
11:00 am Developing FKBPL mRNA Therapeutics for Obesity: From Target Biology to In Vivo Proof-of-Concept
- Defining the role of FKBPL in metabolic regulation and its therapeutic potential in obesity
- Addressing mRNA delivery, protein expression and formulation challenges to enable therapeutic development
- Demonstrating preclinical efficacy and outlining the next steps towards translation
11:30 am Targeting Respiratory Disease: Clinical Progress of ETH47, a Nasally Administered mRNA Therapeutic for Asthma
- Exploring localised respiratory delivery of therapeutic mRNA to achieve dose-dependent protein expression while minimising systemic exposure
- Evaluating the safety, tolerability and biodistribution of nasal and inhaled mRNA formulations to support therapeutic development in respiratory diseases
- Sharing Phase IIa clinical proof-of-concept findings from a rhinovirus challenge study in asthma patients, assessing therapeutic activity and symptom reduction
PROCESS DEVELOPMENT & CMC
Understanding LNP Structure & Stability to Improve Formulation Performance & Product Quality
11:00 am Understanding How Lipid Composition & Manufacturing Conditions Shape LNP Structure, Stability & Transfection Efficiency
- Investigating how different lipid compositions, micromixer geometries and mixing conditions influence LNP morphology, including the formation of BLEB structures
- Leveraging cryo-TEM, dynamic light scattering (DLS) and nano-flow cytometry (nanoFCM) to characterise structural differences across LNP formulations and manufacturing setups
- Demonstrating how the presence or absence of BLEB structures impacts LNP stability and in vitro transfection efficiency, exploring how formulation and micromixer optimisation can influence therapeutic performance
11:30 am Interactive Roundtable: Overcoming mRNA-LNP Stability & Storage Challenges to Reduce Cold-Chain Dependence
An interactive discussion bringing together mRNA formulation, analytical and CMC experts to share practical approaches to:
- Improving formulation stability: Comparing liquid formulations, lyophilisation and spray drying to preserve RNA integrity, LNP structure and biological potency
- Extending shelf life & simplifying distribution: Exploring strategies to enable refrigerated (2-8°C) or room-temperature storage while minimising degradation during storage, transport and handling
- Demonstrating stability for clinical translation: Identifying critical stability-indicating attributes, analytical methods and regulatory considerations to establish shelf life and support GMP readiness
12:00 pm Lunch & Networking
Harnessing AI & Automation to Accelerate RNA & Delivery Platform Innovation
1:00 pm AI-Driven Next-Generation Ionisable Lipid Development & Screening Strategies for Improved Transfection Efficiency & Endosomal Escape
- Leveraging machine learning & AI-driven approaches, from foundation-model pretraining to closed-loop active learning, to accelerate novel ionisable lipid identification from limited datasets
- Addressing endosomal escape & payload–carrier compatibility through rational lipid design & formulation optimisation at reduced RNA doses
- Balancing potency, safety & developability when transitioning from research-scale to GMP-ready lipid platforms, carrying model-derived knowledge into process scale-up & CMC
- Screening novel ionisable lipid structures with multi-objective models to optimise transfection & tissue selectivity
1:30 pm Engineering Next-Generation LNPs for Precise Tissue Targeting, Repeat Dosing & Enhanced Clinical Performance
- Leveraging modular lipid architectures to tune biodistribution, pharmacokinetics, and immunogenicity across diverse therapeutic applications
- Enabling cell- and organ-specific delivery through ligandfunctionalised LNP technologies to improve targeting precision and therapeutic index
- Developing LNP platforms suitable for repeat administration while maintaining favourable safety and efficacy profiles in clinical settings
- Demonstrating versatility across mRNA, saRNA, and other nucleic acid payloads to expand the therapeutic potential of LNP-enabled medicines
2:00 pm Leveraging AI & Automation to Accelerate mRNA Therapeutic Development: Lessons from ABO2203 & Personalised Cancer Vaccines
- Exploring how AI-driven approaches and automated workflows can accelerate mRNA candidate discovery, platform optimisation and selection against target product profiles
- Sharing development insights from ABO2203, an mRNAencoded CD19×CD3 T-cell engager, and Abogen’s personalised cancer vaccine programmes to demonstrate how platform technologies translate into therapeutic applications
- Applying automation and AI across personalised mRNA vaccine development to reduce development timelines and lower cost of goods
Improving RNA Purity, Recovery & Scalability Through Optimised Downstream Processing
1:00 pm Reducing circRNA Manufacturing Costs Through Enzyme-Free Purification, Higher Yield & Scalable Processing
- Replacing reliance on RNase R, where GMP-scale supply can present cost and scalability constraints, with proprietary column chromatography to simplify circRNA processing and reduce product loss
- Increasing recovery to approximately 85% and achieving around 95% purity while scaling manufacturing towards clinical production volumes
- Scaling an enzyme-free purification process from GMP toxicology material towards 30-50 g production while maintaining yield, purity and process simplicity
- Connecting higher yield, simpler purification and lower cost of goods with more affordable RNA therapeutics and broader patient access
1:30 pm Designing Scalable Purification Strategies That Preserve RNA Integrity Across Increasingly Complex RNA Constructs
- Implementing chromatography, tangential flow filtration & affinity-based methods for high-purity circRNA
- Addressing challenges in removing double-stranded RNA to meet regulatory specifications & reduce immunogenicity
- Optimising purification processes to maximise yield whilst achieving target purity levels
- Overcoming bottlenecks in downstream processing when transitioning from research to GMP scale
2:00 pm Developing Scalable mRNA-LNP Formulation, Purification & Process Technologies to Accelerate Clinical Translation
- Enabling both scale-up for larger production volumes and scale-down for individualised therapies
- Leveraging microtechnology-based tangential flow filtration (TFF) for small-scale RNA and LNP purification and concentration, while minimising particle loss and preserving product quality
- Combining machine learning, predictive modelling and process knowledge to optimise mixing parameters, predict target LNP size and reduce consumption of expensive materials
2:30 pm Poster Session & Afternoon Break
This poster session is your go-to session to obtain competitive insights and present your most innovative work to mRNA and delivery thought leaders and stakeholders. For more information or to submit your abstract, please email info@hansonwade.com
Aligning Product Quality, Manufacturing & Regulatory Strategies for Clinical Translation
3:30 pm Leveraging Established mRNA Vaccine Quality Standards to Guide the Transition into Emerging mRNA Therapeutics
- Distinguishing drug-substance CQAs from drug-product CQAs to clarify where controls need to be applied
- Setting phase-appropriate specifications that evolve from Phase I through later-stage development
- Determining what level of heterogeneity is acceptable without compromising safety, potency or manufacturability
4:00 pm Session Reserved for Eclipsebio
4:30 pm Panel Discussion: Navigating Evolving Regulatory Expectations as mRNA Moves Beyond Vaccines
- Clarifying EMA & MHRA expectations for mRNA drug substance, drug product, biodistribution, toxicology & immunogenicity as therapeutic applications diversify
- Understanding how regulatory requirements differ across vaccines, protein replacement, oncology, gene editing & other emerging mRNA applications
- Addressing the regulatory classification of mRNA products, including where non-genome-altering mRNA and mRNAenabled gene editing fit within existing frameworks
- Keeping pace with evolving global guidance while identifying where RNA-specific regulatory expectations remain unclear