* 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

  • Anwar Hossain Senior Scientist- Neurosurgery Research, MD Anderson Cancer Center

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

  • Anna Duboff Associate Scientist II, Codiak BioSciences

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

  • Gary Hao VP- Analytical Sciences, Codiak BioSciences

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

4:00 pm Chair’s Closing Remarks

5:00 pm End of 4th Extracellular Vesicle- Based Therapeutic Development Summit