making cell & gene therapy available

Gene therapy substitutes or corrects DNA to help the body recover function or prevent malfunctions caused by diseases, or viruses. Cell therapies, on the other hand, use specially grown or adapted human cells to replace impoverished tissue or execute healing functions. Popular types of cell therapy incorporate stem cell transplantation, which can create bone marrow, and blood transfusion.

Of the over 50 total drugs approved by the FDA in 2021, only two were cell or gene therapies. Although this is a small amount of the total approved drugs in 2021, it means the number of approved cell and gene therapies grew by 10%.

The cell and gene therapy industry has experienced explosive growth over the past decade. The market is projected to grow rapidly, reaching sales forecasts of EUR 27.9 billion by 2026. More than 1,000 therapies are currently in development, and 50-75 are expected to be approved in the U.S. by 2030. Both CMOs and biomanufacturers plan to significantly add cell and gene therapy manufacturing capacity over the next five years.

However, the industry faces serious operational challenges in addressing demand efficiently, such as a high percentage of non-conforming products, unmet needs, talent gaps and extraordinarily high commodity costs. These challenges are intensified by the highly hyper-competitive nature of the industry and the need to accelerate the pace of progress required to achieve breakthrough indications and a rapid approval pathway to improve patient access. The root causes of these challenges include highly qualified manual processes, and lack of data collection and integration. It is critical to select and implement the right technology to address the complex challenges of CGT manufacturing.

Many cell and gene therapeutics manufacturers are expanding their facilities to increase production capacity and meet market demand, but this tactic is short-term and unsustainable in the long term. This approach results in increased costs, and data management problems. Manufacturers have also begun to investigate new technologies to optimize existing and new facilities. Current CGT equipment and technology operate in silos with little integration, making process optimization or scalability hard. The industry is investing in next-generation technologies that promise to mitigate manufacturing bottlenecks by eliminating human error and enabling process scalability and reliability like large-scale automated platforms. The goal is to deliver manufacturing that can work alongside people to provide more therapeutics in a solid and reliable manner.

Pfizer is currently investing $800 million to establish manufacturing sites to support the manufacturing of gene therapies from preclinical research to commercial-scale production. These plants indicate the significant investment that will be required to scale up cell and gene therapies for market distribution.

Most CGT manufacturers use single/multi-stage automation solutions, which include customized machines that perform one or a series of steps in the manufacturing process. However, manufacturers want to move to fully automated and agile systems to maximize productivity, further reduce cost?and improve batch variability. Let's look at two types of E2E manufacturing systems on the market: those from a single vendor and operations agnostic systems.?

The robotics solution for cell & gene pharma which is closed in single vendor ecosystem is usually specific to autologous T-cell therapies, and has quite limited flexibility. Many in the industry have attempted to address this stiffness by testing closed-loop machines that can increase throughput, provide greater flexibility in process design and therapeutic modality, and reduce manufacturing footprint. By using a completely proprietary system, manufacturers take on more risk the less vendors they use.

An operations agnostic robotics system that can be used in the production of cell and gene therapies allows manufacturers to mix and match unit operations as they see fit. The system offers increased process and supplier flexibility and reduced labor costs. Designing manufacturing plants to take into account the physical movements of the robot, equipment exchange on demand, and integrating data from each unit operation presents new challenge. It is critical to integrate smart automation and modularity early in the process to improve product consistency, support rapid scaling, and reduce post-market process changes.

Investing in robotics alone is not enough to scale up cell and gene therapy production. Robust data to support manufacturers is also essential. Real-time data monitoring is crutial for maximum process optimization and efficiency. The implementation and transition to automation requires careful planning and technology transfer to minimize the impact on current processes and equipment.?

Emerging robotics, modular and automation systems provide solutions for CGT manufacturing, but it is necessary to consider the data infrastructure and impact of technology transfer. Full control and continuous development of the underlying technology platform is of utmost strategic importance.

The biggest manufacturing challenge, is not a lack of laboratory space or capacity. As new therapies are approved, an important factor determining their success will be whether they are allogeneic (based on donor-derived cells) or autologous (based on patient-derived cells). Allogeneic cell-based therapies fit the more traditional model of having a treatment available for distribution when needed, while autologous therapies require modification of cells from the patients themselves to return as a treatment. This means that each dose must be custom-made, which brings significant difficulty and cost to treatment design, production and the entire supply chain.

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