With more and more cell therapies entering the market, Dr Maitry Ganatra, global market development director at Thermo Fisher Scientific guides readers on how to comply with regulatory standards to ensure quality and safety, and ultimately make it to commercialisation.
Market
In recent years, sophisticated cell therapies have emerged to treat a broad range of cardiovascular, neurological and autoimmune diseases, among others. The complexity and variability of these therapies present unique challenges for the transition from the research phase to commercialisation. Thorough consideration of current good manufacturing practice (cGMP) requirements and regulatory compliance early in the process, even during the research stage, can help alleviate the challenges involved in scaling up. Additionally, applying quality by design (QbD) principles to the production process can ensure high quality and safety of new therapeutics, making commercialisation easier.
Fundamental considerations when transitioning from research to commercialisation of cell therapies
Commercialisation of cell and gene therapies, whether allogeneic or autologous, can be challenging. In allogeneic cell therapy, where a single cell type is mass produced for treating multiple patients, the challenge is that of scaling up a thousand-fold from the research phase, while still maintaining the necessary physiological conditions. When processes get scaled up, laboratory instruments, such as centrifuges and CO2 incubators used during the research phase, may not be compatible with the requirements of the cGMP environment. Typically, when equipment designed for research purposes are used in a cGMP compliant facility without consideration of validation and documentation requirements, this can pose a huge issue during commercialisation.