From The Editor | May 18, 2023

The Latest On Cell & Gene Live And Cell & Gene: The Podcast


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

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April and May have been busy months for Cell & Gene: The Podcast and Cell & Gene Live. In case you missed it, here’s a look at just some of what we’ve covered including computational biology’s impact on gene therapy, the future of exosome science, and best practices for in-house CGT manufacturing.

Computational Biology

I invited Kriya Therapeutics’ CEO and Co-Founder Dr. Shankar Ramaswamy to be my guest on an episode of Cell & Gene: The Podcast to discuss how computational biology and bioinformatics play a role in gene therapy development. He discusses how to use computational biology to design better products, and he explains how Kriya used algorithmically-enhanced product engineering to design therapies. For example, recent applications of deep-learning algorithms in bioinformatics include the prediction of DNA variants causing sequence specificities of RNA- and DNA-binding proteins, protein secondary structures, and more.

Last year, I talked with Ken Drazan, M.D., Co-Founder, Chairman, and CEO at ArsenalBio to learn more about how the company explores the use of computational biology to destroy cancer cells in solid tumors. Drazen explained that Arsenal uses a data-driven approach to reprogram T cells into cancer medicines is foundational to ArsenalBio. Large-scale screens of thousands of designs are executed to build rich data models that are sufficiently powered. Computational biologist-built machine learning-based pipelines to process data across multiple modalities and started to build predictive models that associate genetic perturbations and design components to the most effective CAR T cells in the treatment of solid tumors. ArsenalBio had also built a computing infrastructure and data lake to power the high throughput and high dimensional screening capabilities. Drazan also explained that the data lake integrates metadata from experimental design, raw data from instruments, and processed data from computational and machine learning pipelines. Cloud-based computing infrastructure provides the flexibility and elasticity to process and manage these assets efficiently.

Exosome Science

Exosomes are small membrane vesicles that are secreted by a multitude of cell types, which play a role in cell-to-cell communication. They can serve as clinically valuable tools for early diagnosis, prognosis, and potentially targeted treatment. Dr. Linda Marbán, Ph.D., CEO of Capricor Therapeutics, was my guest on a recent episode of Cell & Gene: The Podcast. Capricor is a clinical-stage biotech focused on the development of cell and exosome-based therapeutics for the treatment and prevention of serious diseases. Dr. Marbán explained the current state and the near-term future of exosomes as well as Capricor’s exosome program and the company’s proprietary allogeneic cardiosphere-derived cells (CDCs) and engineered exosomes, both of which are in various stages of preclinical development.

In-House CGT Manufacturing

In case you missed it, our most recent Cell & Gene Live focused on in-house manufacturing. “Building An In-House Cell & Gene Manufacturing Facility: Best Practices & Lessons Learned” delivered 60-minutes of data-driven information to the audience with Will Junker, Head of Vector Manufacturing Quality at Kite Pharma and Mitch Louer, SVP of Technical Operations at Kriya Therapeutics serving as expert panelists. Kriya has a 51,000 square foot manufacturing facility located in Research Triangle Park, North Carolina to support cGMP production of its expanding pipeline of gene therapies. And Kite Pharma, which is responsible for both YESCARTA and TECARTUS, operates cell therapy manufacturing facilities in Oceanside and El Segundo, CA and Frederick, MD, as well as its European cell therapy manufacturing facility outside of Amsterdam.

During the hour, we discussed the why behind each company’s decision to build versus buy, timelines and milestones, total cost, building the team, regulatory requirements, and more. For example, from a regulatory perspective, the FDA oversees GMP certification for drug development in the U.S. by enforcing current GMP, or cGMP, guidelines. GMP, including cGMP, provides a regulatory framework that ensures the quality of products as well as the proper design, control, and monitoring of manufacturing processes and GMP facilities. Both Junker and Louer detailed why a thorough understanding of the regulatory landscape is necessary when building your own facility. And because an empty GMP facility costs a great deal of money, we talked specifically about how to balance the cost and labor model.

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