Cambridge Healthtech Institute’s 2nd Annual

Extracellular Vesicles

Biomarkers, Drug Delivery and Therapeutics

June 17, 2019

A rapid expansion of research into Extracellular Vesicles is yielding numerous healthcare industry applications including their use as biomarkers, in diagnostics, as therapeutic delivery vectors and as therapeutics themselves. Yet many challenges are yet to be overcome, including a lack of standardization for sample preparation and isolation techniques, too little regulatory guidance, scale-up and GMP manufacturing difficulties and not enough clinical evidence. At Cambridge Healthtech Institute’s 2nd Annual Extracellular Vesicles Symposium, discover the latest findings of leading researchers from industry and academia who are endeavouring to realize their true importance. Receive expert opinion from industry leaders on microvesicles and exosomes in biomarkers, diagnostics and precision medicine, therapeutics and drug delivery, without being limited to any one specific therapeutic area.

Final Agenda

Monday, June 17

8:30 am Registration Open and Morning Coffee

Waterfront 2

9:00 Welcome and Opening Remarks

Raghu Kalluri, MD, PhD, Professor and Chair, Department of Cancer Biology, University of Texas MD Anderson Cancer Center

9:10 KEYNOTE PRESENTATION: Current State of Exosome Based Diagnostics

Skog_JohanJohan Skog, PhD, CSO, Exosome Diagnostics, a Bio-Techne brand

Exosomes and other extracellular vesicles (EVs) have emerged as an immensely versatile source of biomarkers for molecular diagnostics, enabling and enhancing the field of liquid biopsy, from mutation detection to RNA profiling and/or protein analysis. The field of liquid biopsy and EV based diagnostics has made substantial progress recently and we have now seen the first commercially available exosome based tests help doctors and patients in their clinical decision-making process.

9:40 Exploiting Extracellular Vesicles as Biomarkers in Liver Diseases

Szabo_GyongyiGyongyi Szabo, MD, PhD, FAASLD, AGAF, FACP, Professor, Vice Chair for Research, Medicine, University of Massachusetts Medical School

10:10 Biochemical Architecture and Morphological Release Pathways of the Human Platelet Extracellular Vesicles

De_Paoli_SilviaSilvia De Paoli, PhD, Staff Scientist, Center for Biologics Evaluation and Research (CBER), Food and Drug Administration (FDA)

Platelet extracellular vesicles (PEVs) have emerged as potential mediators in intercellular communication. PEVs exhibit several activities with pathophysiological importance and may serve as diagnostic biomarkers. Here, I will discuss the structure, composition, and surface properties of PEVs derived from human platelets (PLTs). In addition, I will demonstrate the different pathways for PEVs release from thrombin-receptor activating peptide (TRAP)-activated PLTs.

10:40 Networking Coffee Break

11:10 Novel Methods Enabling Extracellular Vesicle-Derived Biofluid Biomarker Discovery

Pendergrast_ShannonP. Shannon Pendergrast, PhD, CSO, Ymir Genomics LLC

Ymir Genomics has developed several novel methods for the rapid and effective isolation of biofluid extracellular vesicles (EVs). Here we discuss the use of these methods in mass spectrometry and miRNA array workflows developed with the Oregon Health and Science University to discover novel urine biomarkers for liver disease. Our preliminary data establishes proof-of-concept by identifying multiple liver-selective and known liver disease biomarkers in urine EV preps.

11:40 New Biosensor Technologies for Analysis of Extracellular Vesicles

Lee_HakhoHakho Lee, PhD, Associate Professor, Center for Systems Biology, Massachusetts General Hospital

Extracellular vesicles (EVs) are a new class of lipid nanoparticles actively secreted by cells, and have emerged as a promising circulating biomarker for many diseases. The research field has grown rapidly over the past few years, garnering significant interests from diverse disciplines including chemistry, biology, engineering and medicine. This presentation will discuss new analytical approaches that have been developed in my laboratory to empower EV research.

Oxford-Nanoimaging 12:10 pm Quantitative Imaging and Phenotyping of EVs with Single Molecule Sensitivity

Pereno_ValerioValerio Pereno, PhD, Business Development, ONI

The large majority of EV research focuses on the one-dimensional characterisation of exosome-containing samples. Complex phenotyping of exosomes is a major challenge which hinders clinical translation. ONI have developed a microscope able to fluorescently image, size, track exosomes in solutions or inside cells, with the potential for better characterisation, faster translation, and predictable outcomes.

12:40 Enjoy Lunch on Your Own

1:10 Session Break


1:55 KEYNOTE PRESENTATION: Exploiting the Biology of Exosomes for Diagnosis and Therapy for Pancreatic Cancer

Kalluri_RaghuRaghu Kalluri, MD, PhD, Professor and Chair, Department of Cancer Biology, University of Texas MD Anderson Cancer Center

Exosomes are extracellular vesicles released by all cells. Although their precise role remains unknown, it is suggested exosomes mediate specific cell-cell communication and activate signaling pathways in cells. Exosomes are detected in the tumor microenvironment and evidence suggests they play a role in facilitating tumorigenesis by regulating angiogenesis, immunity and metastasis. Circulating exosomes could be used as liquid biopsies and non-invasive biomarkers for early detection and diagnosis of cancer patients.

2:25 Unique Formulated Dual Targeting Antigen Specific and miRNA-150 Delivering Exosomes Acting at the Immune Synapse

Askenase_PhilipPhilip W Askenase, MD, Professor, Medicine, Yale University

We describe an exosome-APC circuit may be applicable beyond skin immunity we study in mice. Antigen tolerized T cells make suppressive antigen-specific exosomes due to surface antibody light chains and target cell specific genes via miRNA-150. Both aspects are lent to naïve activated exosomes by in vitro incubation alone. They bind peptides in MHC on APC that make secondary suppressive exosomes, acting at the immune synapse with effector T cells, transferring new miRNA for strong prolonged inhibition, even when administered orally at the height of the in vivo response.

2:55 Networking Refreshment Break

3:15 Development of Exosome-Based Protein Replacement Therapies

Lundin_PerPer Lundin, PhD, MBA, European Patent Attorney, Co-Founder & COO, Evox Therapeutics Ltd.

Exosomes represent a unique therapeutic modality for delivery of complex biological cargo into target cells. Numerous rare genetic diseases originate in missing and/or defect transporter proteins or enzymes and these are thus excellent targets for exosome-based replacement therapies. Evox Therapeutics has built a comprehensive proprietary technology platform which the company leverages to develop a pipeline of protein replacement therapies within the rare disease space.

3:45 Extracellular Vesicles in Regenerative Medicine

Mirotsou_MariaMaria Mirotsou, PhD, Senior Director of Research, Astellas Institute of Regenerative Medicine

EVs are increasingly recognized as biomarkers for degenerative diseases and key mediators of tissue repair mechanisms. During injury, tissue specific or immune cells release EVs carrying cargo that modulates inflammation, promotes cell survival and enhance healing. Accumulating evidence also supports that stem cells exert their regenerative effects via the release of EVs. This presentation will review the importance of EVs in tissue repair and regeneration and discuss their therapeutic potential.

4:15 Cellular Sources of Extracellular Vesicles for Induced Tissue Regeneration

Larocca_DanaDana Larocca, PhD, Vice President, Discovery Research, AgeX Therapeutics, Inc.

Loss of vascular health contributes to a variety of chronic diseases of old age. Our aim is to restore aging vasculature by inducing programs found in early embryonic cells. Toward this aim, we have established hundreds of unique progenitor stem cell lines using clonal derivation from human pluripotent stem cells. We are mining this cell bank for extracellular vesicles capable of inducing regeneration of vascular tissues in aging adults.

4:45 Close of Symposia

5:00 Dinner Short Course Registration

5:30 Dinner Short Course*

*Separate registration required.

Stay on to attend Tuesday, June 18 - Wednesday, June 19

Preclinical Strategies, Models & Tools in Oncology or
Immuno-Oncology Biomarkers 1: Predictive Biomarkers and Companion Diagnostics

Wednesday, June 19 - Thursday, June 20

iPS Cells for Disease Modeling and Drug Discovery