Gene Editing Focus Day

• Compare how EMA, FDA, and MHRA approach transient mRNA-based editing tools versus permanent genome modifications
• Understand classification and risk-based assessment challenges for mRNA-delivered editors (e.g., ATMP designation)
• Learn how early regulator engagement can de-risk trial design, accelerate timelines, and improve approval outcomes

• Examine how modality choice influences safety, efficacy, and scrutiny in clinical development
• Explore competitive advantages of RNA over DNA platforms across specific indications and cell types
• Align modality selection with CMC feasibility, delivery strategy, and reimbursement potential

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

• 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

• 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 localized, adjustable, and repeatable therapies, exactly where transient, programmable mRNA expression can deliver disease-modifying therapies or cures

• Address manufacturing and CMC challenges for ligand-directed and next-gen formulations
• Nanomedicine Platform for Production and application in therapy
• Gain insights from case studies on tissue-specific delivery in rare diseases, cancer and CNS diseases

• Explore how novel delivery platforms are overcoming biological and anatomical barriers to target lung tissue effectively
• Understand the potential of combining mRNA technology with next-generation carriers to achieve precise gene editing in pulmonary indications
• Review emerging preclinical data showcasing safety, efficiency, and translational promise for respiratory gene editing therapies

• 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