Show Notes:

We were fortunate to be able to interview Dr. Preti in advance of his upcoming keynote address at CBI’s Cell and Gene Therapy Production conference. The title of his talk, Global Cell Therapy Manufacturing: The Past, Present and Future for Next Gen Therapies, was the basis for our interview.

I began the interview by asking Dr. Preti to describe some of the key challenges facing the Cell Therapy industry today. He explained that key challenges include developing manufacturing that produces a high quality product with a reasonable cost of goods. The manufacturing also must be scalable to meet changing demands throughout the commercial life of the product.

He went on to describe one of the factors that makes this so problematic. He talked about why Cell Therapy products can’t be fully characterized and how this makes moving from a lab process to a commercial manufacturing process difficult. It is hard to know if specific process changes impact the final product since the product isn’t completely characterized and we don’t fully understand the mechanism of action.

Next, I asked Dr. Preti about where he thought the industry had made the most progress and the most significant milestones. He said that the most significant advancements have come in the industry’s ability to demonstrate pure potency. This pure potency can be seen in the genetically modified CAR T-cell products. These products have changed the industry and have created new clinical and commercial milestones. Also, seeing how the products work and how clearly efficacious they are has caused the regulatory bodies to move forward with accelerated approvals. These successes have caused the progress on the product side to be slightly ahead of the manufacturing side. Manufacturing milestones include the industry’s understanding of how to get these products to a level of high quality, reasonable cost of goods, scalability and sustainable manufacturing.

When looking at Cell Therapy manufacturing, Dr. Preti shared that HCATS has taken the approach of taking the manufacturing processes apart and separating them into distinct unit operations. Then evaluating each unit operation using a risk based assessment to determine potential issues and improvements. Next, there is a process of evaluating and/or building different tools and technologies to improve the process unit by unit. If all the individual elements can be controlled, then there is greater overall control to ensure that a process change in one unit won’t impact the final product. In addition, over the past ten years, the industry has engaged more engineers in process development. Thus, the industry is using more engineering and innovation to improve each unit operation and ultimately the entire process.

Next, we discussed the most critical areas where we need to make progress in Cell Therapy manufacturing. Bob explained that he thinks these changes are being made currently and it goes back to the engineering approach and creating consistent manufacturing environments for the industry. Groups like the Alliance for Regnerative Medicine (ARM), ISO, NIST and others are creating standards by which the industry can communicate and work. If we look at the biologics industry as a framework, you can see that when they began to have coordinated standards and work together, it really allowed the industry to flourish. It is also important for companies to share experiences about development work, business models, and reimbursement strategies. It is also necessary for the industry to work together to educate key groups, like regulatory agencies, investment groups and the legislature to accelerate the commercial growth of the industry. This work also includes looking to grow across all geographic areas to meet global demand.

My last question was what advancements need to be made to meet the manufacturing demands of next generation therapies? Similar to what happened in the biologics industry, manufacturing paradigms need to shift from boutique manufacturing to industrial manufacturing. This requires the industry to move from the model of making these labor intensive cocktails to creating manufacturing platforms where multiple products can be manufactured using the same platform process. This currently doesn’t exist in regenerative medicine,  we still have a product specific manufacturing paradigm. We are building a manufacturing platform where we can layer multiple products and this approach with help us achieve the high quality, consistent products in a manufacturing system that is scalable with a reasonable cost of goods.

I closed by asking Bob if he had anything else to add for listeners. He said this is the most exciting time to be in this industry. He has been in the industry for the past 30 years and he said it truly feels like this is time. Industry groups assembling all the key groups to make this a success. Big pharma is entering the space in a big way and putting big financial bets down on the industry. They are buying platforms, ideas, and entry into what the next generation of medicine, the next pillar of medicine, regenerative medicine. It is a great time to be in the field and great for patients to have access to these new therapies.

Dr. Robert “Bob” Preti is the co-founder and the visionary behind the successful growth and development strategy of PCT (on October 2, 2017 renamed Hitachi Chemical Advanced Therapeutics Solutions, LLC or “HCATS”) over much of the last two decades. Upon PCT’s acquisition by Hitachi Chemical Co., Ltd. in May 2017, Bob’s role expanded to General Manager, Regenerative Medicine Business Sector of Hitachi Chemical, responsible for development, management and oversight of the global business operations of Hitachi Chemical’s regenerative medicine business unit.  Bob built HCATS to meet a recognized need for high quality manufacturing and development services in an emerging industry. Bob is currently Chairman for the Alliance for Regenerative Medicine, where, among other activities, he previously served as Vice Chairman and in other roles. Bob holds a BS degree in biology from Fordham University, and a MS degree and Doctorate, both in biology, from New York University.