Transforming Disease Treatment: Innovations in Cell and Gene Therapy
Preparing for the Future of Healthcare
Cell and gene therapies (CGT) are revolutionizing the pharmaceutical industry, transforming how we treat diseases and significantly disrupting traditional practices. Rather than using chemical compounds, CGT uses genetically engineered cells, genetic materials and engineered vectors to address the root cause of certain diseases. This process especially has promise in treating rare genetic conditions. As research in this field continues to advance, the industry is actively seeking ways to enhance flexibility in laboratories, bioprocessing and biomanufacturing design.
As part of HDR's ongoing Peer Conversations webinar series, industry leaders gathered to discuss the transformative impact of CGT. Moderated by HDR’s Yong Kang and Susan Suhar, the panel explored advancements in technology and space configurations to enable faster delivery times across discovery, development and manufacturing stages.
Navigating a Delicate Process
For as promising as CGT is, it is equally complex. Often, “you don’t know how what you’re poking at is affecting how the cells react,” Bates said. CGT is targeted to each patient’s specific condition, meaning that a lot of treatment manufacturing is carried out in small batches and requires lab workers to manually manipulate delicate cells. Consequently, the processes involved need to be highly precise, making repeatability difficult. Small differences in the process can lead to substantial variations in the result, adding to the complexity of scaling up production and distributing it to a broader base.
Crafting Spaces for CGT
Because of its unique nature, CGT manufacturing facilities have distinct design considerations compared to traditional pharmaceutical manufacturing facilities. Instead of large-scale industrial factories, CGT facilities are often composed of smaller lab spaces tailored for small-batch production processes. This bespoke approach requires designers to have an intimate understanding of the production processes to create seamless movement of personnel, product and waste throughout the space, minimizing disruptions that could affect the delicate cells.
Many CGT facilities integrate large shell spaces that are multifunctional and equipped with the necessary connections and infrastructure to adapt to various needs. Planning and pre-design can take up to three years, during which market needs can change significantly, underscoring the need for flexibility. Facilities need to accommodate clean rooms, which are a critical component of CGT production because they provide sterile environments that prevent contamination. Equipment used in clean rooms must be specifically designed to fit and operate in these smaller, colder environments. “The need is really to have buildings with flexibility or adaptability to react faster to market trends and keep clean rooms as small as possible for the needed environment,” Becker said.
Embracing Modularity
Modularity is a key strategy for facilitating small batch manufacturing in CGT. This approach allows for easy expansion and significantly reduces time to market. Equipment used in CGT production is being modified to be more effective in small batch manufacturing, such as cell culture incubators and bioreactors, which are designed to maximize efficiency within the limited space of clean rooms. This emphasis on “scaling out” rather than “scaling up” allows for more adaptable and responsive production processes. Bayer’s Berkeley, California campus incorporates modularity, including modules for therapeutic proteins, cell therapies and gene therapies within a standardized footprint. “We found if we stayed inside that, we could build it anywhere on the planet in any region of the world,” Johnston said.
Building and Maintaining a Skilled Workforce
The complex nature of CGT processes means that skilled labor is essential. Workers need to possess a combination of mechanical and technical skills, including operational technology, information technology and process control. The emphasis on small batch production further highlights the need for highly skilled personnel who can handle the intricate and delicate nature of CGT manufacturing.
Collaborations with universities are crucial in training and recruiting the next generation of CGT professionals. These partnerships equip students with the necessary skills and knowledge to thrive in the industry. Accessible facilities that provide hands-on experience and exposure are also vital for effective training programs. Thermo Fisher has customer experience centers around the world that provide hands-on training and experiences for customers and employees alike.
Creating a supportive workplace environment is key to attracting and retaining talent. This includes internal and external knowledge sharing, which fosters a culture of continuous learning and innovation. Building amenities such as mingling spaces, grab-and-go options and family programs contribute to a positive work-life balance, making the workplace more appealing to employees. Sartorius even built a kindergarten on its Göttingen, Germany campus to improve employee lifestyle.
Preparing for Tomorrow
CGT is sure to have a lasting impact on the pharmaceutical and healthcare industries, but the exact shape of that impact is yet uncertain. Manufacturers and their facilities need to be ready for the developments that are sure to come.
Flexible building design is essential to accommodate future technological needs. By pre-installing infrastructure for upcoming technologies, companies can reduce costs and improve efficiency over time, Becker explained. Designing facilities with adaptability in mind provides a framework to handle the evolving demands of the CGT industry. As robotics and artificial intelligence (AI) continue to advance, they are expected to significantly optimize production processes and improve the overall efficiency of biomanufacturing facilities.
Collaboration and technological innovation are key drivers of progress in the industry. As CGT evolves, the industry must stay adaptable and forward-thinking to meet the challenges of this rapidly changing field. Robust equipment, flexible facility designs, specialized small batch production and skilled personnel are all essential components that support the growth and development of CGT.
