The American Society of Gene and Cell Therapy (ASGCT) defines cell therapy as a therapeutic modality that involves the administration of either normal or modified cells to patients for the treatment of various diseases. In this form of therapy, patients are injected with living and intact human cells that are deemed to be capable of providing therapeutic benefit.
Cell therapies are based on the premise that the patient’s own cells (autologous), or those from a healthy donor (allogeneic), can be genetically re-programmed to combat various diseases. These therapies have emerged as viable alternatives to traditional treatment options, including small molecule drugs. It is worth mentioning that the past few decades have witnessed several advances in this domain, particularly in the cell therapy manufacturing process. However, even the latest and the most promising advances in modern medicine have their own set of challenges and risks, which need to be addressed to ensure continued growth.
Steps Involved in Cell Therapy Manufacturing Process
Manufacturing biologics and cell therapies is considerably complex when compared to small molecule drugs. In order to manufacture GMP regulated cell therapies, it is necessary to assess each manufacturing step at regular intervals. The major steps involved in cell therapy manufacturing have been briefly outlined below.
This figure presents the cell therapy manufacturing steps.
The development of cell therapy products requires a robust population of cells that can be subjected to genetic alterations, without being damaged in this process. Owing to their ability to differentiate into various types of cells and their near infinite proliferative potential, stem cells are considered to be ideal candidates for cell therapies. Fundamentally, stem cells can be broadly categorized as hematopoietic and non-hematopoietic cells. Hematopoietic stem cells are derived from the bone marrow and can be differentiated into any cell type of the hematopoietic lineage. It is worth mentioning that these cells have been widely used in several research studies.
Key Challenges Faced by Cell Therapy Manufacturers
The cell therapy manufacturing process is an exhaustive and complex. Some of the key challenges faced by cell therapy manufacturers have been briefly summarized below:
- It is difficult to scale-up the manufacturing procedures of cell therapy products. As a result, the mass production of such products is still a concern. Such procedures require sterile and higher-grade clean rooms, along with regular checks on containment levels.
- Sophisticated equipment is required for the relocation, transport, and multi-site distribution of cell therapy products. Therefore, the facilities where cell-based therapies are to be developed and manufactured should be established at easily accessible locations. This is also likely to help reduce the overall cost of manufacturing.
- Setting up cGMP facilities and obtaining the necessary qualifications and certifications is not only challenging, but also requires heavy investments. Moreover, cell production facilities should be equipped with proper storage rooms, clean rooms, cryopreservation rooms and have various other cell processing capabilities as well.
- Contamination is a key concern when it comes to manufacturing cell therapy products. To overcome this, manufacturers are required to modify their current procedures in order to incorporate closed manufacturing systems. The advantages offered by closed systems, include potential for automation, lower risk of contamination, increased yield, effective use of manufacturing space, consistent product quality in a controlled environment and provision for the parallel processing of multiple products.
- There can be limited availability of trained and expertise staff as manufacturing requirements increase. In addition, the cost of manufacturing is extremely high and requires a large workforce. There is a need to have a cost-effective cell therapy manufacturing process platform in order to make the cell therapies easily accessible to the patients.
- The enrichment and isolation of mono-nuclear cells, their ex vivo expansion and the availability of raw material are amongst the other most common manufacturing challenges faced by the stakeholders in this industry.
- The assays established for traditional biologics are unlikely to be suitable for cell and gene therapy. This is primarily due to the lack of reliable evidence related to the mechanism of action of cell therapies. In addition, the assays used in early stages of cell therapy research are difficult to implement at the commercial scale owing to the inherent complexities of such procedures, coupled to the fact that they are also time consuming and expensive.
Key Drivers in the Cell Therapy Manufacturing Domain
A number of innovative methods and technologies are under development to augment the development and production of cell therapies. They are likely to serve as important drivers of the industry’s growth. Efforts to introduce automation technologies in cell therapy manufacturing process are underway, and if implemented successfully, can significantly help in the elimination of human intervention and reduce the risk of contamination. As a consequence, it is likely to result in a significant increase in product consistency, ensure the maintenance of sterility, and decrease the production time and cost. Additionally, the shift to using closed systems is expected to limit the risk of contamination, which is a major concern in cell therapy manufacturing. However, improvements in the methods used for cell purification and enrichment are expected to substantially enhance the overall quality of the cell therapy manufacturing process. Currently, the aforementioned steps are generally carried out by physical methods, or the surface marker beads separation method. It is worth mentioning that methods that require any human support are likely to become more common in the foreseen future.
Concluding Remarks
In recent years, a spate of initiatives and guidance by regulatory agencies of different countries have enabled the developers and manufacturers to enter in this domain. Based on the recently issued regulatory guidelines, cell-based products have been distinguished from other biotechnological products on the basis of several parameters. The recognition of these differences has greatly benefitted the cell therapy community to address the challenges associated with cell-based products and to add to the uniqueness of such products.
More than 2,000 cell and gene therapy candidates are currently being investigated for the treatment of a myriad of disease indications. Additionally, numerous cell-based therapies have already been approved by various regulatory authorities across the globe. In order to ensure the continued success of these novel therapies, there are several ongoing efforts to address the complexities related to their development and manufacturing. The aforementioned efforts are focused on improving cell handling and processing equipment, ancillary materials, cell counting technologies and addressing existing logistical concerns. Further, it is gradually becoming important for cell therapy developers to invest in flexible and automated technologies, in order to cater to the challenges associated with human error in the cell therapy manufacturing process. Such advancements are likely to assist companies in ensuring quality and consistency of products at affordable costs. Furthermore, variety of stakeholders are actively engaged to reduce the development timelines to drive affordability of cell and gene therapies.
Bottom Line
Keeping every trend in mind, Roots Analysis has provided complete information on developments in cell therapy manufacturing processes and market analysis in its report titled “Cell Therapy Manufacturing Market”. The research report has some of the very recent and precise activities listed for the clients to help them make better decisions. Further, it presents current and future opportunity trends associated with the cell therapy manufacturing market, till 2035.
About Author
Ronit_Sharma
Ronit Sharma is an accomplished business research and competitive intelligence professional with about seven years of experience in the pharmaceutical and healthcare industry. As a team leader at Roots Analysis, he has authored numerous multidisciplinary market research reports, and led the efforts on several bespoken consulting assignments, providing valuable insights into the latest innovations in healthcare and the digital transformation of the pharmaceutical industry. Ronit’s exceptional analytical skills and strategic thinking in the field contribute to the firm’s intellectual capital, empowering clients to make informed decisions in the dynamic pharmaceutical landscape. With a passion for staying at the forefront of industry advancements, Ronit specializes in identifying emerging opportunities for various stakeholders, leveraging his deep understanding of market trends and technological developments. He is committed to fostering an environment of excellence, creativity, and innovation within his team, encouraging collaboration and empowering team members to bring their best ideas to the table.
Ronit holds a B. Tech degree in Biotechnology from Lovely Professional University and his research and thought leadership can be found in his professional blog and social media profiles, where he shares the latest insights and engages in meaningful discussions with industry peers. To ensure the highest ethical standards, Ronit openly declares no conflicts of interest in his work, ensuring unbiased and trustworthy contributions. His insights undergo rigorous editorial and peer-review processes, establishing his credibility as a thought leader within the pharmaceutical and healthcare domain. Ronit’s exceptional analytical and strategic thinking skills, coupled with his commitment to excellence, make him a valuable asset to any organization in the pharmaceutical and healthcare industry.
