Expanding the Emerging Therapeutic Horizon through Standardization

Drug development is inherently costly and time-intensive, with potential attrition serving as a persistent burden on bio/pharma companies’ finances (1). As companies gain a deeper understanding of cell and molecular biology and seek to develop more complex therapeutics for patient populations, they will also need to be more innovative to ensure commercially viable success is achieved (2).

An area that has witnessed significant breakthroughs over recent years has been that of cell and gene therapies. The market for these emerging therapies is projected to grow at a compound annual rate of 22.41% between 2022 and 2030 (3), driven by an expanding pipeline and increasing regulatory approvals.

Despite the successes that have already been achieved with emerging therapy development and manufacturing, companies are still facing numerous challenges. Aspects such as demand for accelerated development timelines, advancing technologies, and the growing issue surrounding sustainability are monopolizing the priorities of bio/pharma companies. However, through new opportunities, such as allogeneic cell therapy, it is hoped that greater benefits to patients, particularly those in undertreated populations, will be achieved (4).

In speaking with Øystein Åmellem, director of Cell Therapy at Thermo Fisher Scientific, and Evan Zynda, senior staff scientist at Thermo Fisher Scientific, Pharmaceutical Technology® discussed with them how standardization is the future for emerging therapies, and tackling challenges such as scalability will get the industry to that point.

Continued investment in cell and gene therapies

PharmTech: Is there anything specific you are seeing in terms of new emerging therapy development or manufacturing in the industry as of now?

Åmellem/Zynda (Thermo Fisher Scientific): One of the biggest manufacturing trends we are currently seeing is a growing focus on standardization. The current lack of standardization within the industry can be attributed to the recent rapid growth of the cell therapy market and the associated drive to improve the efficiency and cost-effectiveness of production. This has led many companies to develop their own unique manufacturing workflows.

Although this has been beneficial on an individual basis, increased standardization has the potential to improve manufacturing across the industry. For example, it can help simplify processes and reduce production costs in the long term. Additionally, standardized approaches, particularly those that incorporate automation and utilize closed systems, can reduce the risk of manufacturing failures or contamination—helping manufacturers to consistently deliver safe and efficacious therapies.

Addressing new short- and long-term challenges

PharmTech: Are there any new challenges or hurdles in the cell therapy space that should be addressed for the short- and long-term?

Åmellem/Zynda (Thermo Fisher Scientific): A key challenge that must be addressed is scalability. For developers working in the allogeneic space, there is a need to scale up production to meet the projected future demand for these therapies while also keeping manufacturing workflows closed. Conversely, in the autologous space, there is a focus on scaling down production to rapidly produce smaller doses and prioritize optimal cell quality. In terms of addressing these needs, innovative manufacturing solutions are essential. For instance, standardized, closed-system equipment that can be flexibly used to scale production of both types of therapies, without compromising on either safety or cost-effectiveness, can have a hugely positive impact.

The benefits of utilizing allogeneic cell therapies

PharmTech: Why are companies starting to look at allogeneic cell therapies more closely?

Åmellem/Zynda (Thermo Fisher Scientific): In addition to their ability to improve time-to-treatment and treatment efficacy, allogeneic cell therapies have several strategic advantages for biopharmaceutical manufacturers. Most notably, batch sizes of allogeneic therapies are generally much larger, with the intention of freezing down several doses. This can offer considerable benefits by reducing both the production cost per dose and, in turn, the treatment cost for patients.

Industry players and allogeneic therapies

PharmTech: Are allogeneic cell therapies a bigger opportunity for industry players, such as contract development and manufacturing organizations (CDMOs) and contract research organizations (CROs), and why?

Åmellem/Zynda (Thermo Fisher Scientific): There are several reasons why allogeneic therapies are seen as an opportunity for both CROs and CDMOs. For example, the rapid growth of this therapeutic area has led to skills and knowledge gaps within the industry, particularly around regulatory requirements, which can be filled by working with an experienced partner. Additionally, the scaled-up production volumes employed for allogeneic therapies mean that maximizing manufacturing capacity is an essential consideration; however, building this capacity in-house can be both financially and logistically challenging. Thus, choosing to outsource production to a CDMO can be the most effective choice, especially for smaller companies.

Other up-and-coming therapy developments

PharmTech: What do you hope to see for the future of allogeneic cell therapies/any other emerging therapy development or manufacturing?

Åmellem/Zynda (Thermo Fisher Scientific): Further advancements in cell culture media could be highly beneficial. For example, optimizing media for different manufacturing workflow stages could lead to an improved therapeutic product. From a logistical point of view, increased digitalization of documentation such as batch records could help to dramatically simplify workflows and reduce manual workloads, lowering manufacturing costs. This would have the additional benefit of improving overall process control, which is key for optimizing consistency during large-scale cell therapy production.

PharmTech: Beyond cell therapies and gene therapies, what other categories of emerging therapies are being developed, and can we expect to see approvals for those in the near future?

Åmellem/Zynda (Thermo Fisher Scientific): One of the biggest future opportunities within the cell therapy field is utilizing other immune cells beyond T cells as a therapeutic product. Natural killer cells have attracted considerable attention as they are seen as a more feasible option for producing allogeneic therapies. There is also a growing interest in using cell-based immunotherapies to treat solid tumors. Although there are still several challenges, including overcoming the radically different tumor microenvironment, clinical success within this area has the potential to lead to a major shift in cancer treatment.

References

• Agrawal, G.; Keane H.; Prabhakaran, M.; Steinmann, M. The Pursuit of Excellence in New-Drug Development. McKinsey & Company, Nov. 1, 2019. https://www.mckinsey.com/industries/life-sciences/our-insights/the-pursuit-of-excellence-in-new-drug-development
• McKinsey & Company. How AI Can Accelerate R&D for Cell and Gene Therapies. www.mckinsey.com, Nov. 16, 2022.
• https://www.mckinsey.com/industries/life-sciences/our-insights/how-ai-can-accelerate-r-and-d-for-cell-and-gene-therapies
• Precedence Research. Cell and Gene Therapy Market (by Therapy Type: Cell Therapy and Gene Therapy; by Indication: Cardiovascular Disease, Cancer, Genetic Disorders, Infectious Diseases, Neurological Disorders; by Delivery Method: In-Vivo and Ex-vivo; by End-Users: Hospitals, Cancer Care Centers, Wound Care Centers, Others)—Global Industry Analysis, Size, Share, Growth, Trends, Regional Outlook, and Forecast 2022–2030. Market Report, November 2022.
• Fontanillo, M.; Paulick, K.; Poda, P.; Silberzahn, T. Out of the Shadows: A Brighter Future for Pharma Technical Development. McKinsey & Company, Sept. 16, 2022. https://www.mckinsey.com/industries/life-sciences/our-insights/out-of-the-shadows-a-brighter-future-for-pharma-technical-development