EXOSOMES SCIENTIFIC SIGNATURE SERIES: PROGRAM SCHEDULE
Saturday, June 1, 2024
09:00–09:15
Opening Remarks
Sai Kiang Lim, PhD
A*STAR Institute of Molecular Biology
Singapore
Elani Wiest, PhD
Mayo Clinic
United States
Bernd Giebel, PhD
University of Duisburg-Essen
Germany
09:15–10:45
Translating MSC-EV Products: Lessons from the MSC Field for Clinical Implementation
Despite the immense confirmed potential, the journey of translating MSC products is marked by setbacks and disappointments. For instance, despite demonstrating efficacy in a phase III clinical trial, a particular MSC product repeatedly failed to obtain market authorization. Many of the issues and challenges identified in this context are also highly relevant for translating MSC-EVs into clinical applications. In this session, Jacques Galipeau will offer insights based on his experiences in the MSC field.
CHAIR
Bernd Giebel, PhD
University of Duisburg-Essen
Germany
Jacques Galipeau, MD
University of Wisconsin-Madison
United States
Learning Objectives:
Gain insight into critical challenges associated with the clinical adoption of MSC-EV products.
10:45–11:45
Regulatory Challenges for EVs: Perspectives, Hurdles, and Solutions
Extracellular vesicles (EV) are emerging as promising regenerative therapies in multiple disease states with ongoing clinical trials. Unlike the MSCs themselves, their native EV-products do not fall into the ATMP category. Up to now, no EV therapies are currently approved by the FDA or EMA. There are also no official regulatory guidelines for EV therapies, though much work is ongoing to develop guidelines and frameworks for regulators. In this session, panelists from regulatory agencies will discuss the regulatory approach to EVs, challenges, and possible approaches to consider for developing cGMP-compliant EV products.
CHAIR
Elani Wiest, PhD
Mayo Clinic
United States
Laura Ricles, PhD
FDA
United States
Andreas Marti, PD, PhD
Swissmedic
Switzerland
Shinichi Noda, PhD
PMDA
Japan
Learning Objectives:
- Understand the regulatory framework and challenges for EV products.
- Discuss the differences between MSC-EVs and MSCs from a regulatory perspective.
- Understand how the disease state (rare vs. standard) influences the approval of EV products.
11:45–12:30
Lunch
12:30–13:45
Transitioning MSC-EV Manufacturing from Basic Research to Clinical Applications
To translate MSC-EVs into therapeutic applications, a scalable manufacturing process compliant with regulatory protocols is essential. However, basic researchers often lack familiarity with these protocols, leading to challenges in technology transfer to GMP manufacturers. In this discussion, commercial GMP manufacturers will outline key parameters for manufacturing MSC-EVs for clinical translation.
CHAIR
Sai Kiang Lim, PhD
A*STAR Institute of Molecular Biology
Singapore
Nisa Renault, PhD
Fujifilm Cellular Dynamics, Inc.
United States
Jon Rowley, PhD
RoosterBio, Inc.
United States
Learning Objectives:
- To increase awareness of these parameters and integrate them into MSC-EV preparation during pre-clinical investigations at the earliest opportunity
- Understand how to better integrate process development personnel in manufacturing to close this gap
13:45–15:00
Funding EV Research and Clinical Studies
Despite the promise of extracellular vesicles (EVs) for diagnostic, therapeutic, and regenerative applications, obtaining funding for research and clinical studies is a major roadblock. In part, this is because there are no FDA or EMA approved EVs for clinical use. A vital challenge this raises is attracting funding for the research and clinical trials that will form the groundwork of later regulatory approvals. Funding for EV products can come from various sources including government grants, venture capital investments, private equity funding, partnerships, and public offerings. In this session, panelists will discuss challenges to obtaining funding for EV research and clinical studies, possible funding sources, and considerations regarding pros and cons of each.
CHAIR
Elani Wiest, PhD
Mayo Clinic
United States
Byong Seung Cho, MBA
ExoCoBio
South Korea
Konstantin Glebov, PhD
University of Plymouth
United Kingdom
Learning Objectives:
- Name funding hurdles posed by EVs.
- Identify approaches to acquire funding for EV research and clinical trials.
- Recognize possible advantages and disadvantages of the various funding sources for EVs.
15:00–16:00
Closing Panel:
Building a Roadmap for MSC-EVs: How do We Move Forward from Here?
Session Chairs join closing panel to summarize takeaways from each session presentations and discussions and build a roadmap for getting MSC-Evs towards a first regulatory approval with an eye to eventual adoption.
Sai Kiang Lim, PhD
A*STAR Institute of Molecular Biology
Singapore
Elani Wiest, PhD
Mayo Clinic
United States
Bernd Giebel, PhD
University of Duisburg-Essen
Germany
16:00–17:30
ISCT Scientific Signature Series Cocktail Reception