From The Editor | August 17, 2021

Inside Cell & Gene Live: The Therapeutic Potential of Exosomes


By Erin Harris, Editor-In-Chief, Cell & Gene
Follow Me On Twitter @ErinHarris_1

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Our most recent Cell & Gene Live, The Therapeutic Potential of Exosomes, featured three expert panelists who shared their insights on the future of exosome development. Dr. Maria Mitrani, Co-Founder and Chief Scientific Officer at Organicell; Benny Sorensen, Scientific Advisory Board member at Codiak BioScience; and Nicolas Rousseau, Co-Founder and COO at EVerZom spent the hour detailing the current state of exosome research, the existing challenges associated with manufacturing and scalability, as well as potential and feasible solutions.

The Background on Exosomes

Exosomes are nano-sized biovesicles released into surrounding body fluids upon fusion of multivesicular bodies and the plasma membrane. They were shown to carry cell-specific cargos of proteins, lipids, and genetic materials, and can be selectively taken up by neighboring or distant cells far from their release, reprogramming the recipient cells upon their bioactive compounds. Therefore, the regulated formation of exosomes, specific makeup of their cargo, cell-targeting specificity are of immense biological interest considering extremely high potential of exosomes as non-invasive diagnostic biomarkers, as well as therapeutic nanocarriers.

In a previous article with Sorensen on the promise of engineered exosomes for drug delivery, he explained that exosome-based therapeutics have the potential to be a new class of medicines with the potential to transform the treatment of not just cancer, but of a wide spectrum of diseases. Codiak leverages the inherent biology, function, and tolerability profile of exosomes to engineer non-viral drug delivery vehicles that are designed to carry and protect potent drug molecules, provide selective delivery, and elicit the desired pharmacology at the desired tissue and cellular sites. Specific to cancer, this means pursuing oncologic targets and pathways that have so far proven elusive to drug development, such as STING, IL-12, STAT6 and others. 

During Cell & Gene Live, Sorensen explained that in the past five years, the industry has developed a deeper understanding of the biological role of exosomes — in particularly in overall immune surveillance. “A key role of the function of exosomes is to communicate with and orchestrate immune-system responses,” he says. “And these are proteins that have been able to build a highly versatile engineering platform. In a very modular fashion, exosomes with bespoke pharmacological properties, add multiple different types of drugs. I believe that at a key inflection point in the journey of transforming exposome biology into medicines.”

Characterization Hurdles

Nicolas Rousseau provided a great deal of information throughout the live event and specifically about characterization of exosomes. “When characterization is critical is on your process development because you want to make sure once you are scaling up this process — moving to a 10-liter, 100-liter — you want to keep the same consistency of product. You need to have a well-characterized product from the beginning. If you are transferring to a CMO or if you know the partner, you want to make sure that you do not have any deviation of a few new products. Spend time on the characterization and purification.”

Is Scalability Even Possible?

When asked to describe and detail the challenges with scalability, Dr. Mitrani explained that while scalability is important, even more important is reproducibility — that every single drug has the same pattern. She explains that when referring to biologics, from a worldwide regulatory perspective, she and her peers are looking for percentages to be as close as possible in the final product. “Being able to demonstrate that throughout all your batches, at the end of the day, that every single clinical trial and all of the patients that have been enrolled are using that same dose of a final product.” Ensuring consistent trials is critical.

The entire Cell & Gene Live event is chock-full of detailed information about the present and future states of exosome development, as each expert panelist provides detailed information and data on their experience with exosomes. Be sure to visit Cell & Gene to view previous Cell & Gene Live events, The Future of CRISPR-Engineered Cell Therapies and Can Innovation Drive Down Cell Therapy Manufacturing Costs?