5th mRNA Process Development & CMC Summit

• Building partnership models that go beyond single-CDMO reliance, including how to
• qualify and balance primary and backup manufacturers without overextending budget or timelines
• Aligning early with CDMOs on process characterisation, PPQ strategy, batch sizing and scale-up assumptions to avoid revalidation risks and ensure readiness for rapid progression into pivotal studies
• Integrating supply chain and licensing considerations into partnership strategy, including critical raw materials, IP constraints and long-term commercial implications that are often locked in earlier than anticipated

• Building partnership models that go beyond single-CDMO reliance, including how to qualify and balance primary and backup manufacturers without overextending budget or timelines
• Aligning early with CDMOs on process characterisation, PPQ strategy, batch sizing and scale-up assumptions to avoid revalidation risks and ensure readiness for rapid progression into pivotal studies
• Integrating supply chain and licensing considerations into partnership strategy, including critical raw materials, IP constraints and long-term commercial implications that are often locked in earlier than anticipated

• How early, structured CDMO engagement can reduce development risk and shorten timelines for mRNA, neoantigen and personalised cancer vaccine programmes in cell & gene therapy pipelines
• Aligning process development and CMC expectations between biopharma and CDMOs to avoid scale-up failures and enable smoother translation from preclinical to commercial supply
• Defining effective partnership models for complex modalities, including tech transfer best practices, capacity planning, and managing variability in patient-specific manufacturing workflows

Key questions include:

• How do you structure CDMO partnerships to balance speed, flexibility, and risk without overcomplicating your manufacturing network?
• What are the biggest misalignments between biopharma and CDMOs during scale-up – and how can they be avoided early?
• At what point should companies start designing for commercial-scale manufacturing, and how does that shape CDMO and supply chain decisions?

• Examining how differences in input components influence yield, consistency, and impurity profiles across mRNA manufacturing workflows
• Establishing control strategies to ensure consistency without restricting flexibility- defining specifications, sourcing approaches, and risk-based frameworks that maintain quality while supporting process optimization and platform development
• Embedding raw material considerations into next-generation platform design – ensuring approaches are adaptable across emerging modalities while enabling faster development timelines, improved robustness, and reduced cost of goods

Key questions include:

• How do variations in mRNA structure, impurity profiles (e.g. dsRNA), and LNP formulation impact in vivo expression, immunogenicity, and reactogenicity?
• Which critical quality attributes (CQAs) are most predictive of clinical performance, and how early can these be confidently defined during development?
• How can teams better correlate analytical data with preclinical and clinical outcomes to reduce late-stage failure risk?

• How are emerging mRNA modalities in CGT changing the biggest process development and CMC challenges teams need to solve today?
• Where are current analytical and control strategies falling short when applied across diverse mRNA-enabled CGT applications – and how should they evolve?
• What does a truly scalable and flexible CMC framework look like when supporting multiple mRNA-driven CGT modalities with differing requirements?

• Exploring lessons learned transitioning process development strategies from self-amplifying RNA to circRNA platforms, including upstream IVT optimization,circularization workflows, and downstream purification considerations
• Demonstrating how automated high-throughput experimentation can accelerate reaction chemistry optimization, analytical assay development, and impurity characterization across emerging RNA constructs
• Discussing approaches to building adaptable, cross-functional manufacturing and analytical frameworks capable of supporting the growing complexity of nextgeneration RNA therapeutics and advanced therapy applications

• Exploring key CMC and process development considerations for delivering RNAbased gene editing systems across in vivo (e.g. LNP) and ex vivo manufacturing workflows
• Addressing challenges in achieving consistent editing efficiency while maintaining product quality, potency, and safety across different delivery modalities and target tissues
• Discussing how platform-based development strategies can support scalability, tech transfer, and clinical translation of next-generation genetic medicines

• Persistent virus reservoirs associated with dysfunctional anti-viral T cell responses is a root cause of Long COVID, a debilitating disease affecting >400 million people globally
• We have developed an mRNA-based immunotherapeutic targeting protective T cell antigens of SARS-CoV-2
• Treatment of Long COVID in mice resulted in reversal of T cell dysfunction, clearance of virus reservoirs, and amelioration of cognitive and behavioural deficits

• Exploring key considerations in RNA-LNP process development to improve reproducibility, product quality, and manufacturing robustness across development stages
• Sharing approaches to balancing formulation, process parameters, and analytical insight to reduce variability and support efficient scale-up
• Discussing how integrated CMC strategies can de-risk tech transfer, strengthen process understanding, and accelerate progression from early development to clinical and commercial manufacturing