* All times shown in EST
8:30 am
Morning Networking Coffee
8:50 am Chair’s Opening Remarks
Exploring Latest Advancements of EV-Based Cargo Delivery
9:00 am Payload Delivery & Cargo Loading of EV-Based Neural Stem Cells in CNS
Synopsis
- Maximizing the therapeutic potential of EV- based neural stem cells through nucleic acid and protein payload delivery
- Loading cargos exogenously and endogenously in CNS applications
- Outlining important considerations when filing IND for a exosome product
9:30 am Engineered Exosomes as Gene Delivery Tools for the Treatment of Glioblastoma
Synopsis
- Developed engineered exosomes (eExos) that incorporate viral proteins to successfully package, deliver, and express episomal plasmid DNA in human glioma cells in vitro and in vivo
- Screening microRNA library for glioblastoma and then delivery miRNA via exosome
- How packaging 3 miRNAs in one plasmid leads to successful delivery to tumours
10:00 am Targeted Delivery of Therapeutic Payloads Using Engineered Stem Cell Exosomes
Synopsis
- Loading exosomes with different cargos through surface engineering or luminal loading of various proteins and nucleic acids, nucleases
- Exploring the effects of using different producer cell lines on EV platform
- Pre-clinical data on the application of EV-platform to CNS
10:30 am
Morning Networking Break
Track A: Discovery
Exploring Characterization Strategies of EVs
11:30 am Single Particle Characterization & Analytical Techniques for EVs in Immuno- Oncology Therapeutics
Synopsis
- Unlocking single particle characterization as a method for understanding product heterogeneity and structureactivity relationship in EVs for IO therapeutics
- Leveraging single particle characterization for driving process development and cell line development decisions
- Comparing various particle analytical techniques for EV characterization
12:00 pm Advancing Proteomics on Exosomes to Improve Protein Identification
Synopsis
- Delving into proteomics methods on exosomes
- Using mass spectrometry on exosomes to identify and profile proteins
- Exploring tube digestions as an alternative to bands to avoid the loss of proteins
Track B: Translation
Improving Efficacy & Biodistribution of EVs
11:30 am Exploring the Therapeutic Efficacy & Biodistribution of Exosomes Using an Organoid- Based Multi Organ on a Chip Platform
Synopsis
- Exploring alternatives for the use of animals in extracellular vesicle research by leveraging a multiorgan chip
- Unraveling the development of a chip that combines liver and kidney
- Investigating whether this multi-organ-on-a-chip models are superior to using animals when assessing therapeutic efficacy and biodistribution of MSC-derived extracellular vesicles
12:00 pm Scalable Production & Characterization of Non- Immunogenic ARMMs Particles
Synopsis
- Using scalable, GMP-compliant processes, we can produce ARMMs efficiently payloaded with biomolecules of interest such as gene editors.
- ARMMs in vivo: how the route of administration affects biodistribution and the specific cell types which take up ARMMs
- Repeated administration in immunocompetent mice is safe, supporting their use as a non-viral delivery method
12:30 pm
Networking Lunch
Delving into Precision Engineering of EVs
1:30 pm Engineered Exosomes for Therapeutic Applications
Synopsis
- Loading exosomes with nucleic acids
- Clinical applications of exosome-based therapeutics
2:00 pm Applying Engineered Exosomes in Regenerative Medicine
Synopsis
- Leveraging the engineering of exosomes in regenerative medicine therapeutics
- Exploring the EVOGEX platform as a method to advance key exosome properties and increase efficacy
- Exploring the platform’s lead indications in GI
Establishing Clinical Success with Emerging Assays & Analytical Tools
1:30 pm Advancing Platelet-Derived Exosomes for Clinical Applications
Synopsis
- Unraveling the characterization and purification methods of platelet-derived exosomes
- Exploring different technologies and emerging assays for exosome characterization
- Engineering approaches for platelet-derived exosomes
2:00 pm Boosting Exosome – Based Therapeutic Development for Regenerative Medicine & Beyond
Synopsis
- Establishing different analytical tools to standardize EV quality control
- Unraveling the therapeutic potential of an exosome based product for regenerative medicine applications, and inflammatory/autoimmune diseases
- Present our business platform established to boost the development of exosome-based therapeutics through industry partnerships
2:30 pm
Afternoon Break
Understanding Regulatory Expectations for Accelerated Approval of EV-Based Therapeutics
3:00 pm Considerations on Regulatory CMC Dossier Preparation of EV- Based Therapeutics
Synopsis
- Overview on challenges of CMC development and regulatory compliance for EV based therapeutics
- Case studies on CMC modules for regulatory filing of EV based therapeutics
- Future trend and perspective
3:30 pm Regulatory Considerations to Advance EV Therapies to Licensure
Synopsis
- How exosome companies are applying RMAT breakthrough designation
- Unravelling regulatory strategies towards achieving BLA
- Outlining important regulatory considerations for accelerated clinical approvals