Cell and gene therapies (CGTs), a diverse category of medications encompassing cell-based immunotherapies, cell therapies, gene therapies, and tissue-engineered products, have witnessed unprecedented funding, clinical research, and new product launches in recent years, fueled by the potential of CGTs to offer transformative benefits to patients who often lack alternative treatment options.

According to research by nova one advisor, the global cell and gene therapy market size was valued at $18.13 billion in 2023 and is anticipated to reach around $97.33 billion by 2033, growing at a CAGR of 18.3% from 2024 to 2033.

However, despite the increasing activity, the health system infrastructure needs and the complexities of the journey often required by patients to receive these therapies pose significant barriers to the future success and sustainability of these treatment modalities. Findings from IQVIA indicate that significant uncertainty still exists about the future trajectory and sustainability of the cell and gene therapy sector.

One major challenge to the CGT market is the difficult and expensive manufacturing process, which includes certain steps that are different from other types of medicines, vaccines and treatments.

In this exclusive interview with Contract Pharma, Jon Ellis, an innovator with 20 years of experience in cell therapy technology and process development and the CEO of Trenchant BioSystems—a CGT manufacturing platform developer that has designed a platform to drastically transform the CGT manufacturing landscape—shares his insights on the current state of cell and gene therapy manufacturing, the sector’s biggest challenges, and its future.

Contract Pharma: In what ways have CGT therapies changed the clinical outcomes of patients with autoimmune diseases and cancers?

Jon Ellis: The development of CGT therapies has been one of the most exciting developments in modern medicine. Patients who would have otherwise succumbed to their disease are alive and in remission at a rate very rarely seen in clinical trials. The subsequent regulatory approval of these therapies has changed the lives of many patients, but sadly, nowhere near enough. Without improving accessibility and making CGTs more affordable, patients will not receive life-saving therapies.

CP: How has the cell and gene therapy manufacturing landscape changed over the past five years?

JE: There have been some developments to increase automation in manufacturing, but there have not been the necessary advancements to make a significant difference to manufacturing. There are still far too many batch failures. New technologies in development, including the Trenchant platform, will hopefully finally be able to make a meaningful change to the way CGTs are made to enable more patients to be treated. Without automation and increased robustness, we will not be able to scale the industry to meet demand.

CP: Where do you envision the CGT market going in the future?

JE: There hopefully will be a wider range of clinical indications, including the most insidious solid tumors. Autologous manufacturing moving to a distributed or point-of-care model will reduce the vein-to-vein time and simplify logistics.

CP: What are the current factors driving growth in the CGT sector?

JE: FDA approvals using TILs for melanoma and encouraging early clinical data in autoimmune disease are driving growth. The three modalities of autologous, allogeneic and in-vivo offer specific benefits, and it is likely that all three will continue to grow and find their specific niche.

CP: What are some of the biggest challenges you’ve observed in the production of CGTs?

JE: The use of legacy platform technologies that require skilled operators to use and do not connect fluidically or digitally to each other, leading to batch records that can run to hundreds of pages. Such technologies often suffer cell loss at each manufacturing step or fail to achieve the number of cells required for the prescribed therapeutic dose.

CP: What specific challenges in current CGT manufacturing does your platform uniquely address? What are the key technological innovations that underpin your platform?

JE: Trenchant has redesigned CAR-T manufacturing processes and increased efficiency to drastically reduce many of the steps currently required in the manufacturing processes. We developed our platform as a fully automated, configurable, integrated cell therapy manufacturing platform.

Key features include:

• Automation: The Trenchant platform is a truly hands-off manufacturing process for all cell handling and operations including sampling, reducing the requirement for highly trained operators.
• Aseptic fluid transfer: A module for aseptic transfer of fluids and gases within the platform without the use of conventional tube welding technologies reducing manufacturing cost and risk.
• Cell selection: A proprietary cell selection technology for the isolation of specific cell populations such as T or NK cells. Secondary selection is also possible to further purify to a more specific cell population which could be utilized to ensure a more consistent therapeutic phenotype between patients.
• Digital Control System: Capture and management of all manufacturing data to populate the batch record and compliance with critical process parameters.

CP: What are the biggest challenges you anticipate facing in the coming years, and how do you plan to overcome them?

JE: The biggest challenges relate to the unknowns that will occur with the treatment of additional disease indications. Ensuring the industry can scale to meet patient need, therapies can be produced at the appropriate scale in terms of cells per dose and number of doses, ensuring equity of patient access, and reducing the time to treatment are all problems that need to be overcome but they are the same problems we have already been dealing with for a number of years—the majority of which can be addressed with improvements to manufacturing.