Day Two

• Understanding how personalized and patient-specific mRNA products are being approached by EMA and MHRA
• Learning from ongoing oncology-focused mRNA trials navigating the current patchwork of global expectations
• Discussing gaps in guidance for non-infectious mRNA indications and how companies are addressing them

• Discovering how saRNA and circRNA are redefining RNA’s potential with longer lasting expression, lower dosing, and expanded therapeutic applications
• Understanding the scientific and clinical implications of modality-specific challenges, from immunogenicity to delivery complexity
• Learning from pioneering case studies translating next-generation RNA formats into viable therapies across oncology, rare diseases, and beyond

• Explore how engineered Cas9 variants are expanding precision, efficiency, and safety in gene editing applications
• Review strategies for tailoring Cas9 platforms to address the unique challenges of rare disease indications
• Learn from case studies on building a diversified rare disease pipeline through Cas9- based therapeutic development

This networking session is your opportunity to get face-to-face with many of the brightest minds working in the mRNA field and establish meaningful business relationships to pursue for the rest of the conference

• Understand best practices of safety monitoring during clinical trials to ensure robust safety oversight
• Learn how post-marketing studies and close interaction with health authorities shape ongoing patient safety and regulatory compliance.

• Optimising CRISPR/Cas9-loaded LNPs as a novel therapeutic strategy to target specific mutations in lung cancer
• Presenting in vitro and in vivo data demonstrating editing efficiency, safety, and the therapeutic potential in preclinical models
• Discussing the translational implications for moving CRISPR-based lung cancer therapies toward the clinic

• Explore how barcoded nanoparticles generate high-resolution datasets to map RNA delivery efficiency across tissues
• Learn how machine learning algorithms decode complex delivery patterns to optimise formulation design
• Understand how this approach accelerates the translation of RNA therapeutics with improved targeting and efficacy

• Accelerating Neoantigen Discovery – How AI integrates genomic, transcriptomic, and immunological data to rapidly identify and prioritize tumor-specific targets
• Optimizing Vaccine Design – Using predictive models and adaptive strategies to anticipate immune response and iteratively refine personalized formulations
• Translating Innovation into Impact – Case studies where AI platforms have shortened development timelines, improved patient outcomes, and opened new horizons for immunotherapy

• Explore how CycloPhore™ and SemaPhore™ overcome delivery barriers to enable the extrahepatic delivery of circular RNA and mRNA
• Understand the platform’s potential for improving stability, targeting, and translational efficiency of RNA therapeutics
• Learn how patented delivery technologies are advancing the development of novel RNA modalities

• Exploring how quality benchmarks are evolving as mRNA, saRNA, and circRNA platforms mature beyond first-generation vaccines
• .Understanding the key factors that determine vaccine quality and potency, across different RNA formats
• .Discussing how improved analytical approaches are helping ensure consistency and reliability from lab development to large-scale manufacturing

• Learning from vaccines: Exploring how mRNA’s transient biology is not a drawback but an asset
• Building on innovation: Reviewing how advances in mRNA topologies, stability, and delivery systems are expanding what mRNA can achieve
• Why Ophthalmology?: Discussing how eye diseases demand localised, adjustable, and repeatable therapies, exactly where transient, programmable mRNA expression can deliver disease-modifying therapies or cures

• Exploring next-generation RNA modalities from programmable nucleases to emerging enzyme systems for therapeutic use in the lung
• Case study: Leveraging a Cas13 platform to counter respiratory viral infections
• Designing inhalable delivery vehicles for safe, efficient deposition throughout the airways

• Address manufacturing and CMC challenges for ligand-directed and next-gen formulations
• Review a nanomedicine platform for production and application in therapy
• Gain insights from case studies on tissue-specific delivery in rare diseases, cancer and CNS diseases