Cell and Gene Therapy Manufacturing and Development Trends

Precedence Research reports that the global cell and gene therapy market size was about $15.54 billion in 2022 and is projected to reach approximately $82.24 billion by 2032. To understand this growth, GEN spoke to two experts at Cytiva—Clive Glover, PhD, vice president, viral vectors, and Martin Westberg, vice president, cell therapy.

GEN: What are some of the larger trends you are predicting for the cell and gene therapy industry for 2024?

Glover: There is a great deal of excitement in the gene therapy industry. We are seeing a rise in gene editing therapeutics with the approval of the world’s first CRISPR-based gene editing therapy. (The therapy, Casgevy, aims to cure sickle cell disease and transfusion-dependent b-thalassemia.) Despite this and other recent scientific advancements, there continue to be challenges around cost and access. We must develop improved manufacturing practices for viral vector–based products if we want to help reduce the costs of gene therapies. The industry also needs to work with payers to improve access and distribution of these novel therapeutics.

In early 2023, Peter Marks, MD, head of the FDA’s Center for Biologics Evaluation and Research (CBER) outlined a four-point plan to help safely accelerate approval processes. One way is to use new research efforts to improve manufacturing. As an industry, we must collaborate with academic researchers, early-stage firms, and commercial-stage biotechnology companies to ensure we are developing the right technologies and solutions that will accelerate the development, approval, and adoption of these life-changing therapies.

Westberg: Over the last several years, some autologous chimeric antigen receptor (CAR) T-cell therapies have moved from third-line treatments to second-line treatments. It’s further proof of the science and benefits of the therapeutics. We will continue seeing increased levels of scrutiny and reviews of long-term data which are all part of the standard regulatory review process. The push to develop allogeneic cell therapies and cell therapies for the treatment of solid tumors will continue in 2024 and beyond. Simultaneously, we must continue working toward standardizing and industrializing the manufacturing process. There is a lot of work to be done, but the solutions and technologies that are being researched and developed have the potential to transform global health.

GEN: What are the greatest challenges facing your customers?

Westberg: A key challenge for the entire cell therapy industry is scale-up and industrialization of processes. While automating processes that are overly dependent on manual labor is an important part of this, scaleup requires bespoke equipment, software, and reagents. Cell therapies are highly complex therapeutics with an equally complex manufacturing process. Standardizing and industrializing processes will likely help alleviate some of the pressure around cost and access.

Glover: The field must also continue working to understand the body’s response to in vivo viral vectors. Over the last several years, there have been several adverse events. We are starting to better understand how the body reacts and how we modify treatment regimens to achieve better outcomes, but there is still quite a bit of work to be done.

GEN: What is the greatest opportunity for cell and gene therapy drug developers?

Westberg: For cell therapies, there are three big opportunities in the near future. The first is for approved therapies to be prescribed earlier in the treatment plan, the second is to develop a CAR T-cell therapy for solid tumor cancers, and the third is to develop an allogeneic “off the shelf” therapy. Much work is being done globally for the development of a solid tumor therapy and for an allogeneic therapy. We are hopeful that the research being done today will lead to approved therapies in the next few years. In the meantime, we must continue working to automate processes so that therapies can be manufactured at scale.

Glover: Gene therapies have enormous potential to revolutionize global healthcare. For the first time, we are able to treat the cause of the disease and not the symptoms. Upgrading improved manufacturing techniques that will enable drug developers to commercially manufacture these therapies is an enormous challenge and opportunity. To do that successfully, we must leverage the many years of knowledge gained from manufacturing monoclonal antibodies and recombinant proteins. There are many lessons that can be applied. One area that we are very focused on at Cytiva is improving the biology with stable cell lines and high-density cell culture for adeno-associated virus manufacturing.

GEN: Historically, the biopharma industry has been slow to adopt digital solutions. How will greater adoption of automation and digital solutions impact the larger cell and gene therapy industry?

Glover: Automation is effective only if you have a well-defined and -characterized process. This is where the focus should be for gene therapy. Once we have done that, many automation solutions already developed for monoclonal antibody processes can be used.

Making sure the process is stable and robust requires stable cell lines and high-density cell culture. You can’t have a robust manufacturing process without robust materials supporting that process.

Westberg: Increased adoption of automation and digital solutions is necessary to accelerate the manufacture and adoption of cell therapies. It comes back to automating parts of the workflow that are overly dependent on manual labor and having bespoke equipment, software, and reagents. It requires collaboration across the entire ecosystem. No one company or academic institution is going to solve this problem. We must work together to develop the tools and technologies that will accelerate the industry.

GEN: What lessons from the pandemic can be applied to the cell and gene therapy industry?

Glover: We learned that when the scientific community, governments, academic researchers, and the life sciences industry come together we can solve problems faster. We know advancing and accelerating the development of cell and gene therapies is going to require more regulatory flexibility. We saw how well that worked with the development of COVID-19 vaccines, both mRNA- and viral vector–based vaccines. Now, the industry is building out strong mRNA capabilities, and there are many clinical trials in development for mRNA-based therapies.

However, despite this success during the COVID-19 pandemic, the industry is less resilient than it was two years ago. According to Cytiva’s Biopharma Resilience Index, a survey of more than 1,200 biopharma and pharma executives across 22 countries, collaboration has fallen in the last two years. The three most challenging R&D partners to find are 1) patient group associations, 2) companies from other industries, and 3) other pharma/biopharma companies.

Additionally, more than half of respondents believe regulatory agencies are not good at ensuring availability of specialized pathways for cell and gene therapies. If we truly want to move the industry forward and deliver for patients who need medicines the most, we must collaborate and be more flexible when it comes to regulatory pathways.

GEN: How can the industry better harness the power of collaboration?

Westberg: We know that one organization or company can’t do it alone. We must rely on the greater power of the industry itself. One example involving allogeneic cell therapies is the Cytiva–Bayer collaboration. By leveraging Cytiva’s expertise in developing tools and technologies for the manufacture of therapies, and Bayer’s deep capabilities in drug development, we are working to create a modular end-to-end manufacturing platform for allogeneic cell therapies. With the science moving so quickly, we are working to make sure that the manufacturing processes keep pace with innovation.

Another great example of collaboration was with AstraZeneca, Oxford University, and Pall Life Sciences. (Pall is now part of Cytiva.) Oxford University and AstraZeneca relied on the expertise of Pall to help develop a scalable manufacturing process in just eight weeks instead of five years. When the organizations delivered an effective COVID-19 vaccine at record speed, it was a real testament to their ability to harness their respective strengths.

As I think ahead to 2024 and beyond, I believe we will need to see more industry collaborations to truly move the cell and gene therapy industry forward.

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