Agenda

• Honing Rion’s platelet-derived exosome technology and application in regenerative medicine
• Reviewing outcomes from ongoing clinical programs in advanced wound care and musculoskeletal diseases
• Presenting additional, future applications of exosome-based technologies as a therapeutic platform

• Characterization of EV-based therapies
• Clinical manufacturing, loading and decoration technologies
• Productivity increases for competitive cost-of-goods
• Early assessment to build investor confidence

• Exploring recent update of deramiocel Phase 3 trial
• Reviewing direct evidence deramiocel exosome homing to skeletal muscle
• Understanding MOA of exosome on anti-fibrosis, anti-inflammatory and potential targeting mechanism

This session is your opportunity to get face-to-face with many of the brightest minds working in the exosome and extracellular vesicle therapy field, and establish meaningful business relationships to pursue for the rest of the conference.

• Evaluating exosome dosing strategies: concentrations, delivery methods, and clinical outcomes across multiple conditions
• Understanding Why FDA drug approval models don’t fit acellular biologics — and how the ADDSB framework offers a risk-proportional alternative
• Honing state-level exosome regulation trends and the legislative gap excluding acellular products despite their safety profile and signalling role

• Optimizing bioprocess design and orthogonal analytical strategies to achieve high-purity extracellular vesicle products
• Enabling reproducible, scalable manufacturing while addressing batch-to-batch variation and quality control challenges
• Advancing functionality and potency assays to support extracellular vesicle characterization and therapeutic development

• AGLE-102 showed meaningful improvements in wound healing and skin integrity in patients with recessive dystrophic epidermolysis bullosa (RDEB), indicating strong therapeutic potential
• The treatment was well tolerated across the study population, with no significant safety concerns or dose-limiting toxicities reported
• Phase 1/2a results support further development, demonstrating both biological activity and clinical benefit in this high unmet-need genetic disorder

• Examining the key barriers limiting Big Pharma investment and participation in the exosome therapeutics landscape
• Evaluating the impact of regulatory uncertainty, manufacturing challenges, clinical validation requirements, and commercial considerations on adoption decisions
• Identifying the scientific, clinical, and business milestones needed to transition exosome therapeutics into a mainstream pharmaceutical modality

• Breaking down major cost drivers including CMC development, GMP manufacturing scale-up, patient recruitment, and long-term safety monitoring requirements
• Reducing inefficiencies through smarter trial design, including adaptive protocols, streamlined endpoints, and biomarker-informed patient selection
• Leveraging scalable manufacturing and early regulatory alignment to minimise delays, rework, and costly late-stage development changes

• Selecting fit-for-purpose animal models that reflect disease relevance, biodistribution, and immunological response to exosome-based interventions
• Establishing translational pharmacology frameworks linking dose, exposure, and functional response across in vitro and in vivo systems
• Integrating safety pharmacology, toxicology, and biodistribution studies to de-risk first in-human clinical trial design

• See beyond light scattering and flow cytometry for EV particle analysis
• Spectradyne’s ARC particle analyzer offers a truly orthogonal approach
• The ARC yields unique insights for EV size, concentration and phenotype

• Implementing robust labelling and detection strategies (e.g., fluorescent, radioactive, or molecular tagging) to enable accurate in vivo tracking of exosome fate
• Addressing methodological challenges including signal dilution, label stability, and distinguishing free label from intact vesicles
• Correlating biodistribution profiles with pharmacodynamic effects to inform dosing, safety assessment, and tissue targeting strategies