When Robert Preti, PhD, co-founded PCT back in 1999, he already had extensive experience in stem cell processing and other roles from his time with the New York Blood Center. In the two decades since PCT’s founding, Dr. Preti and the staff at PCT, now Hitachi Chemical Advanced Therapeutics Solutions, have grown their expertise in the field of cell therapy.
Recently, the International Society for Cell & Gene Therapy (ISCT) conducted an interview with Dr. Preti to discuss the past, present, and future of cell and gene therapy. This interview was highlighted in ISCT’s Talking with Giants segment.
Here are a few highlights from the interview:
How Has Cell Therapy Changed over the Years?
As Dr. Preti stated in the interview: “When I started in the field in 1986, there really was no cell and gene therapy industry at all.” At the time, the regulatory frameworks, investors, and infrastructure needed to support the regenerative medicine industry were either nonexistent or incredibly limited.
However, in the years since PCT’s founding—and especially in the last few years—the cell and gene therapy industry has made great strides. For example, in 2017, the U.S. Food and Drug Administration (FDA) approved three separate cell and gene therapy products (Kymriah, Yescarta, and Luxturna). These approvals demonstrate an increasing acceptance of and investment in gene and cell therapy products.
What Does the Future Hold for Cell and Gene Therapy?
It is a given that cell and gene therapy developers/manufacturers will continue to look for ways to improve the cost of goods, quality, scalability, and sustainability of their products. However, one key issue that some developers may overlook is the need for a consistent and reliable payment model.
As Dr. Preti pointed out in the interview: “Right now, as each company gets close to commercialization, they’re working out different, creative payment models with the payers… but those models are going to have to be much more sophisticated for larger scale distribution.” Without a stable and robust payment model, cell and gene therapy products may struggle to achieve and maintain sustainability. So, it’s easy to forecast that there may be some changes to the way companies handling cell and gene therapy approach their payment systems.
Finally, Dr. Preti forecasted that, with the continued globalization of the economy, “We’re going to have to find a common, harmonized regulatory pathway around the world so that companies developing in one part of the world can consider their products being developed in other parts of the world.”
The Biggest Challenges Facing Cell and Gene Therapy Development
In the interview, Dr. Preti noted that even small changes “in the collection, manufacturing, storage or transportation of the cells all have the potential to change the potency and safety profile of the product.” This, in turn, leads to comparability risks that affect a cell therapy product’s ability to achieve regulatory approval.
Another major challenge is ensuring that therapeutic cells can be delivered consistently while meeting all quality, safety, cost, and sustainability goals. Limitations of transportation networks and cell collection/modification/storage processes makes increasing deliverability of cell therapy products difficult.
Advice for New Cell and Gene Therapy Developers/Manufacturers
Dr. Preti ends the interview with a piece of advice for anyone looking to join the regenerative medicine industry: “Keep the patient front and center and the mission stays clear.” At the end of the day, it is the patients that cell therapy products are meant to help. If the product cannot be delivered to the patient in a safe, consistent, and affordable way, the commercial viability of the product suffers.
The easier cell and gene therapy manufacturers can make it for patients to benefit from their products, the more viable the treatment will be.
Download the full article from ISCT at the link below, or contact us to learn more about cell therapy manufacturing and development issues.
