The cell and gene therapy sector is booming. At the end of 2019, there were 1,069 registered clinical trials in the field. By 2024, the cell and gene therapy market is estimated to reach revenues of $6.6B (€5.9B). But there is a problem. As a result of the rapid growth of gene and cell therapies, the demand for plasmids is skyrocketing. And the industry is struggling to meet it.
“Plasmids are the key building blocks needed to manufacture viral vectors, which are the most common strategy to develop gene therapies,” explains Stefano Baila, Director of Operations and Business Development at Anemocyte. “Plasmids are not only crucial for the development of gene therapies, but also for therapies that involve the genetic modification of cells, such as CAR-T. This has greatly increased the demand for plasmids and I would say that the industry was probably not ready to address this demand.”
While the industry is struggling with the lack of plasmids on the market, another challenge is quality. Those providers that can meet the high-quality requirements for plasmids have long waiting lists of up to 12 months, says Baila. For companies moving into phase 3 or commercial production, it becomes crucial that the good manufacturing practice (GMP) requirements are met.
But another result of the rapidly evolving gene and cell therapy space is the fact that the regulatory requirements are not quite up to scratch. “The regulatory framework around plasmid production is very confusing for the industry at the moment,” says Baila. “The main guidelines refer to the quality of the product, but the level of quality remains open to interpretation. More clarity would definitely help once and for all to define the exact quality levels required at different stages of drug development.”
Finding solutions for plasmid manufacturing in the cell and gene therapy sector
Consequently, plasmid providers have to be able to address all quality levels required at each stage of drug development. As the first biotech manufacturing organization (BMO) worldwide, Italian company Anemocyte has met this challenge by focusing greatly on the industry’s needs. Their keyword is flexibility.
Before starting their work on plasmid manufacturing, the team spent several months interviewing companies about their difficulties and needs regarding the bottleneck in plasmid production and regulatory issues.
“For us, it was key to understand the needs of the industry and find a possible solution,” Baila explains. “Our research resulted in a brand new facility, which is designed with adaptable manufacturing spaces that enable flexible time management. This ensures that the manufacturing process continues to roll without creating a bottleneck.”
As a next step, the Anemocyte team had to decide whether to use a classified cleanroom or just a regular lab for the manufacturing process. “We decided to keep the bar pretty high, so we are working with a cleanroom facility where we apply the GMP standard,” says Baila. “We maintain a high quality and also address the time issues that all companies seem to share as their main challenge in cell and gene therapy development.”
Customer collaboration is key
The flexibility of its manufacturing facility allows the Anemocyte team to easily adapt to its customers’ needs. “Our customers have control over what we do,” explains Marco Ferrari, CEO of Anemocyte. “They have the opportunity to be involved in the process, and decisions and actions are discussed and shared with them to ensure their product is produced at the high standard they expect.”
Moreover, Anemocyte pays attention to new technologies and innovations. “The fast evolution of the cell and gene therapy industry greatly increases the demand for new solutions,” Ferrari explains. “Staying on top of innovation is therefore mandatory today. Our approach is to stay ahead of the trends and be capable of deploying useful solutions for our customers.”
Anemocyte’s manufacturing facility is built in such a modular way that it can be replicated and adapted to meet the rising demand for plasmids. This, as Ferrari puts it, ensures that the Anemocyte team “will not be caught off guard when more companies come knocking at our door.” This is an advantage for long-term customers because the manufacturing facility can be duplicated and built according to the customers’ needs.
Solving the manufacturing bottleneck for plasmids in the future
As the world’s first BMO, Anemocyte pays specific attention to innovation and the ongoing trends in the industry. One of the emerging trends is the use of nonviral vectors for the development of gene therapies.
“Even in the nonviral approach, plasmids play a key role,” explains Baila. “A part of the nonviral strategy is the transfer of plasmids into cells via mechanical or chemical methods. So, in one way or another, plasmids will always be needed.”
“Our investment in the nonviral gene modification space is an example of how we are tackling potential future trends that are still under the radar or explored at an academic level,” adds Ferrari.
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Author: Larissa Warneck, Science Journalist at Labiotech.eu