WEDNESDAY 23RD OCTOBER, 2024
09:00: Registration, welcome refreshments and networking
Developing Precise EV Biomarkers, Diagnostics and Liquid Biopsies
10:00 - OPENING REMARKS
Chair: Konstantin Glebov, Managing Director, Exosome Consulting
10:05 - PRESENTATIONS
Technological considerations for extracellular vesicle-based biomarker applications
- The analysis of EVs as biomarkers has gained widespread interest, especially since EVs are easily accessible in diverse sources, offer perspectives for multi-analyte biomarker measurements, are present in high concentrations and are relatively stable (De Wever and Hendrix, EMBO J, 2019; Hendrix et al., Nat Rev Mol Cell Biol, 2021)
- Novel biomarker identification is often gained through deep omics of EV (Tulkens et al., Gut, 2020; Dhondt et al., J Extrcell Vesicles, 2020; Vergauwen et al., J Extracell Vesicles, 2021)
- I will cover the multi-step process and cover guiding principles and considerations towards EV-based biomarker discovery (Van Deun et al., Nat Methods, 2017; Hendrix et al., Nat Rev Meth Prim, 2023)
An Hendrix, Professor, Ghent University, Principal Investigator, Cancer Research Institute Ghent (CRIG), Principal Investigator, European Liquid Biopsy Society (ELBS)
10:25 - PRESENTATION
Pre and on-treatment response prediction for patients with hematological malignancies using plasma EV transcriptomics
- EV-miRNA networks predict durable response in Myeloma patients
- EV-RNAs faithfully represent their cell of origin
- Deconvolution of EV-RNA in patients with hematological cancer
- Multi-modal analysis of cfDNA and EV-mRNA
Michiel Pegtel, Assistant Professor, VU University Medical Center and CSO, ExBiome
10:45 - PRESENTATION
Are EVs Ready for Prime-Time in Liquid Biopsy Diagnostics?
- EVs are gaining rapid traction in Liquid Biopsy applications
- The distribution of EV sub-types in biofluids should guide the development of new EV-based diagnostic products
- The route-to-market for EV diagnostic products in IVD
- How to validate and regulate different EV omics?
Tomás Dias, Chief Technology Officer, Mursla Bio
11:05 - Technology Focused Talk
Enabling Extracellular Vesicle (EV) biomarker applications with SiMoA® ultrasensitive biomarker technology
- Simoa® digital detection technology overview
- “Best-in-class” sensitivity and simplified workflow for EV applications
- Key example oncology and neurology EV assays and their clinical application
Lindsey Marsh PhD, Senior Field Application Scientist, Quanterix
11:15 - Panel Discussion
Developing Precise EV Biomarkers, Diagnostics and Liquid Biopsies
- Major obstacles and enablers of vesicle-based diagnostic products (Scientific, Regulatory, Commercial)?
- Comparing three major markets (North America, Europe, Asia), which market is more receptive for starting and developing Dx products, why?
- What is the true value proposition of vesicle-based Dx products?
- How do you anticipate the landscape to change in the light of legal and financial challenges (like USA Biosecure Act, EU regional foreign investment protection regulations, high cost of capital etc)?
- Patent protection for Dx products, hurdles, how to develop right IP strategy for academic discoveries?
Moderator: Konstantin Glebov, Managing Director, Exosome Consulting
Michiel Pegtel, Assistant Professor, VU University Medical Center and CSO, ExBiome
An Hendrix, Professor, Ghent University, Principal Investigator, Cancer Research Institute Ghent (CRIG), Principal Investigator, European Liquid Biopsy Society (ELBS)
To Be Confirmed – Paul Slowey, CEO, Oasis Diagnostics Corporation
11:45 - NETWORKING COFFEE BREAK, POSTER VIEWING
Plenary Session: EV Biomarkers for Diagnosis and Prognosis
12:15 - OPENING REMARKS
Chair: Michiel Pegtel, CSO, ExBiome, Assistant Professor, VU University Medical Center
12:20 - PLENARY KEYNOTE PRESENTATION
EV Protein Biomarkers for Diagnosis and Prognosis of Multiple Types of Cancers
- Investigating how EV-mediated intercellular communication drives tumor dynamics and metastasis.
- EVs capture critical molecular signatures of cancer, offering new avenues for early and precise diagnostic assays.
- Leveraging EV protein biomarkers can enhance non-invasive cancer detection and monitoring, improving clinical diagnostics.
- EV protein profiles provide valuable prognostic information, helping to predict tumor progression and patient responses to therapy.
- Clinical trials focusing on EV biomarkers to refine therapeutic strategies and personalize cancer treatment.
David Lyden, Professor of Pediatrics, Weill Cornell Medical College
11:40 - Technology Focused Talk
Please contact chris@precisionmedicineforum.com
13:10 - Panel Discussion
EV Biomarkers for Diagnosis and Prognosis
- How do you see the integration of EV protein biomarkers into routine clinical practice evolving over the next few years?
- How can we overcome major hurdles for implementing EVs in care, such as a lack of standardized and scalable methods for isolation?
- Without cell-of-origin analysis, how will EVs as liquid biopsy markers reach the clinic?
- When will Bulk Plasma EV analysis ultimately outperform tumor-centric cfDNA approaches for precision medicine?
- How do recent breakthroughs in understanding the molecular mechanisms by which EVs contribute to tumor progression and metastasis impact future therapeutic strategies?
Moderator: Michiel Pegtel, Assistant Professor, VU University Medical Center and CSO, ExBiome
David Lyden, Professor, Weill Cornell Medical College
Tomás Dias, Chief Technology Officer, Mursla Bio
13:30 - NETWORKING LUNCH, POSTER VIEWING
Molecular Mechanisms, EV-Engineering and Therapeutic Delivery
14:30 - OPENING REMARKS
Chair: Richard Kelwick, Senior Research Associate & RSE Enterprise Fellow Alumni, Imperial College London
14:35 - PRESENTATION
Extracellular Vesicles; from signalling mechanistic to therapeutic engineering.
- Syntenin fusion efficiently sorts cargo (Cas9) into small extracellular vesicles
- Syntenin supports efficient release of small extracellular vesicle cargo into the cytosol of recipient cells
- Syndecan 1 C-terminal fragment efficiently sorts Nanobodies to the surface of small extracellular vesicles
- Measure of the impact of Nanobody-EV coating for the internalization of small extracellular vesicles by target cells
Pascale Zimmermann, Professor, KU Leuven
14:55 - PRESENTATION
Extracellular Vesicles for Biomedical Applications – Engineering EVs for Targeted Drug Delivery
- Functional delivery by EVs requires endosomal escape and release of cargo
- EVs deliver cargo to recipient cells, in vitro and in vivo, very efficiently only after engineering
- High gene editing can be achieved in vivo by engineered EVs
- By combining simultaneous mRNA and protein delivery by EVs an enhanced therapeutic effect can be achieved
Joel Nordin, Resident in Clinical Immunology and Transfusion Medicine, Karolinska University Hospital, Assistant Professor, Karolinska Institutet and Senior Researcher, Evox Therapeutics
15:15 - PRESENTATION
Vaccine and Therapeutic Delivery by Engineering Recombinant Extracellular Transport Vesicles
- Cell-specific targeting with surface display of antibodies and cell-targeting affibodies
- Genetic engineering improves safety margins and versatility
- Scale up with bioreactors processing and analytical characterization
Christopher Locher, Chief Executive Officer & Co-Founder, Versatope Therapeutics
15.35 - Technology Focused Talk
Please contact chris@precisionmedicineforum.com
15:45 - PANEL DISCUSSION
What are the pivotal challenges and emerging innovations shaping the future of extracellular vesicle-based therapeutics and vaccines?
- What are the primary bioprocess engineering challenges in scaling up the production of EVs for clinical applications?
- How do you ensure the purity and consistency of EVs when producing them for therapeutic use?
- Can you discuss the regulatory hurdles for EV-based therapeutics and vaccines, and how the industry is addressing them?
- How does the source of EVs (e.g., stem cells, or bacterial cells) impact their biodistribution and therapeutic potential?
- Can you share any success stories or significant breakthroughs in the field that you feel has or will pave the way for future clinically useful EV therapeutics?
Moderator: Richard Kelwick, Senior Research Associate & RSE Enterprise Fellow Alumni, Imperial College London
Pascale Zimmermann, Professor, KU Leuven
Joel Nordin, Resident in Clinical Immunology and Transfusion Medicine, Karolinska University Hospital, Assistant Professor, Karolinska Institutet and Senior Researcher, Evox Therapeutic
Christopher Locher, Chief Executive Officer & Co-Founder, Versatope Therapeutics
16:15 - Refreshment Break
16:45 - Short Course and Drinks Reception
Short Course: Regulatory Pathways for the Translation EV Therapeutics from Pre-Clinical Research to the Clinic
16:45 Introduction to the short course and overview of learnings
• Finding clarity on which guidance to follow, what is a Regulatory Binder, and how should they be implemented?
• Cell Lines, GMP and CMC
• Pre-Clinical Efficacy, Safety and Toxicity, Pharmacokinetics and dynamics studies for IND application
• Getting to the clinic…so what’s next, Phase I, II and III, BLA and Approval?
• Regulatory pitfalls to avoid for EV therapeutics
16:55 Introduction of attendees, so each person can say who they are and why they are here?
17:00 Regulatory Binder Presentation, Handout and Q&A
17:15 Cell Lines, CMC and GMP – Presentation and Discussion
17:30 Pre-Clinical studies for IND application – Presentation and Discussion
17:45 In the clinic, Phase I, II and III, NDA and Approval – Presentation and Discussion
18:00 Short course summary and takeaways, final Q&A
Short Course Leader: Maureen Merrifield, Chief Regulatory Officer, Rion
For additional workshops please contact chris@precisionmedicineforum.com
18:15 - End of Short Course
19:00 - End of Drinks Reception and Close of Day One
THURSDAY 24TH OCTOBER, 2024
09:30: Welcome refreshments and networking
Approaching Translational Challenges for Therapeutic EVs
10:00 - OPENING REMARKS
Rachel Crossland, Senior Research Associate in Extracellular Vesicle Biology, Translational and Clinical Research Institute, Newcastle University
10:05 - PRESENTATION
Clinical Potential of MSC-EVs and Translational Challenges
- MSCs are a heterogeneous cell entity with donor-to-donor variations
- MSC-EV product features depend on their parental MSCs
- In addition to inter-donor heterogeneities, intra-donor heterogeneities affect the function of MSC-EV products
- Immortalization and clonal selection procedures appear essential to warrant robust and scalable manufacturing of MSC-EV products
- Improved analytics and potency testing is mandatory to appropriately evaluate MSC-EV product characteristics.
Bernd Giebel, Professor, University Hospital Essen
10:25 - PRESENTATION
Biological standardisation and development of potency reference reagents for EVs
- Bioactivity and potency testing considerations for Biotherapeutics and ATMPs
- Development and establishment of International Reference Reagents
- Process optimisation for manufacturing of MSC-EVs bioactivity standards
Anna Nowocin, Head of Flow Cytometry Standardisation, NIBSC, MHRA
10:45 - PRESENTATION
A Regulatory Pathway to Advance Towards the First Clinically Approved Therapy for Exosomes
- Chemistry manufacturing and control of exosomes
- Pre-clinical regulatory strategy for exosomes
- Clinical trial progression of exosomes
- Interacting with regulatory bodies to progress the exosome field
Maureen Merrifield, Chief Regulatory Officer, Rion
11:05 - Technology Focused Talk
Overcoming Translational Barriers in Exosome-Based Therapies: From Preclinical Proof-of-Concept to Scalable, Optimized Manufacturing
- Translating preclinical efficacy of exosome-based siRNA therapy (ExoPTEN) into clinically relevant outcomes.
- Scaling up exosome production with a focus on process optimization and robustness in maintaining product quality.
- Developing robust analytical assays for exosome characterization, potency, and homing efficiency.
- Addressing regulatory requirements through pre-IND engagement and alignment with FDA feedback.
- Expanding the exosome platform’s therapeutic beyond spinal cord injury- potential for broader clinical applications.
Noa Avni, R&D Director, NurExone
11:25 - Panel Discussion
Approaching Translational Challenges for Therapeutic EVs
- What potency assays are in use for EV products, how do they reflect the product’s MoA and how to ensure the assay’s reproducibility?
- How to measure EV activity, from both a mechanistic and a therapeutic perspective?
- What are the key challenges in ensuring the consistency and scalability of MSC-EV products?
- What are the most critical regulatory considerations when progressing exosome-based therapies from pre-clinical research to clinical trials?
- How can industry collaborations with regulatory bodies be strengthened to expedite approvals?
11:45 - NETWORKING COFFEE BREAK, POSTER VIEWING
Plenary Session: Production of EVs Under GMP Conditions
12:15 - OPENING REMARKS
Chair: Noa Avni, R&D Director, NurExone
12:20 - PLENARY KEYNOTE PRESENTATION
Vesicular secretome fractions manufactured and characterized under GMP – and first clinical data
- Preclinical characterization needed before clinical testing
- Mode of Action: Biologic activity of naïve stromal cell derived EVs
- Regulatory framework for clinical testing of Nanovesikular Therapeutics (NVT)
- First in human application of nanovesicular drug candidates
Eva Rohde, Chair Transfusion Medicine and Director GMP Unit, Paracelsus Medizinische Privatuniversität
12:50 - Technology Focused Talk
Please contact chris@precisionmedicineforum.com
13:10 - Panel Discussion
Production of EVs Under GMP Conditions
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What are the critical steps to ensure consistent large-scale EV production while maintaining biological activity and therapeutic efficacy?
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What analytical methods are essential for characterizing EVs, including potency, purity, and homing capabilities?
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How can batch-to-batch consistency be maintained during scale-up, and what are the best practices to mitigate variability?
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How do regulatory bodies like the FDA view EV manufacturing, and how can companies prepare for evolving guidelines in the absence of formal standards?
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What are the challenges and technical tools in improving EVs as optimized drug delivery vehicles?
Moderator: Noa Avni, R&D Director, NurExone
Eva Rohde, Chair Transfusion Medicine and Director GMP Unit, Paracelsus Medizinische Privatuniversität
13:30 - NETWORKING LUNCH, POSTER VIEWING
Looking Forward, EVs in the Clinic
14:30 - OPENING REMARKS
Chair: Naveed Akbar, Associate Professor of Cardiovascular Science, University of Oxford
14:35 - PRESENTATION
Early clinical experience of a cardiac cell-derived secretome in heart failure
- The production of a GMP-compliant clinical-grade secretome enriched in extracellular vesicles is technically challenging but doable.
- Batch-to-batch reproducibility, quality controls and dosing metrics are the clinically-relevant key issues that still require to be fine-tuned.
- A deeper understanding of the mechanism of action is critical for selecting the most appropriate delivery route.
Philippe Menasché, Professor, Regenerative Therapies for Cardiac and Vascular Disease, Université Paris Cité, Inserm
14:55 - PRESENTATION
Characterization of cell membrane extracellular vesicles in blood products
- Extracellular vesicles (EVs) are released from blood cells and platelets during processing and storage of blood components for transfusion.
- Platelet-derived extracellular vesicles (PEVs) are the most abundant type of EVs in human blood and contribute to many biologically significant processes of clinical relevance.
- Platelet extracellular vesiculome is very complex, composed of exosomes, ectosomes, podiasomes,“free” mitochondria, autophagosomes and some other types of EVs. Different populations of PEVs are investigated for their potential effects on safety and efficacy of platelet transfusion products and as biomarkers of platelet storage lesion.
- In cryopreserved platelets or EV-based hemostatic biologics, different EV populations represent major or main components with a marked impact on the product in vitro procoagulant potency and potentially also on safety and efficacy in vivo.
- More than 90% of EVs present blood products are smaller than 300 nm, most are of exosome size < 100 nm. Quantitative detection of nanoscale EVs by traditional analytical methods, such as conventional flow cytometry, is not possible. There is an urgent need for development of a panel of robust high-resolution assays for analysis of membrane micro- and nanovesicles in blood products and other biologics.
Jan Simak, Principal Investigator, Office of Blood Research and Review, Center for Biologics Evaluation and Research, FDA
15:15 - PRESENTATION
SALIVA- an Ideal Source of High Quality Exosomes
- The simple and very rapid isolation of exosomes from saliva specimens
- The characterization [number of exosomes, size distribution, etc.]
- Comparison of the isolation of exosomes from the current gold standard method [passive drool] and a novel saliva collection kit, Pure•SAL™
- [Hopefully] data generated on exosomes collected from saliva for Lewy Body Parkinson’s Disease
Paul Slowey, CEO, Oasis Diagnostics Corporation
15:35 - Technology Focused Talk
Please contact chris@precisionmedicineforum.com
15:45 - PANEL DISCUSSION
Looking Forwards, EVs in the Clinic
- What is the biggest barrier to EV-therapeutics?
- How do we overcome these barriers?
- What is needed to facilitate discovery to translation?
Moderator: Naveed Akbar, Associate Professor of Cardiovascular Science, University of Oxford
Philippe Menasché, Profesor (PU-PH), Team Leader – Regenerative Therapies for Cardiac and Vascular Disease, Université Paris Cité, Inserm
Jan Simak, Principal Investigator, Office of Blood Research and Review, Center for Biologics Evaluation and Research, FDA
Paul Slowey, CEO, Oasis Diagnostics Corporation
16:15 - CLOSE OF FORUM
If have any questions about the agenda or speaking opportunities, then please email
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