Cell and gene therapies are transforming the lives of patients with various rare genetic diseases as well as more common conditions such as cancer. With hundreds of therapeutic candidates in the pipeline, millions of additional patient lives could be transformed in the years to come. However, that can only happen if the evolution of biopharmaceutical manufacturing keeps pace with the rapid scientific progress being gained within the industry. Significant shifts will need to occur if cell and gene therapies are to meet their full, life-changing potential, and standardization is one of the key elements the industry needs to meet this requirement.
There are multiple ways the industry can bring supply in line with demand. Additional capacity, new technologies, process development, scaling up and scaling out can all support increased supply. However, the rapid pace of scientific progress has overwhelmed supply infrastructure, creating pressures to scale manufacturing, increase capacity and meet a wide range of deliverable requirements.
At the end of 2015, there were 631 active regenerative medicine clinical trials.1 By late 2020, the number of ongoing clinical trials had risen to 1,109.2 The rapid expansion of the clinical development pipeline is set to translate into a surge in approvals of cell and gene therapies. From 2025 onward, the Food and Drug Administration expects to approve 10 to 20 cell and gene therapy products per year.3 The European Medicines Agency is also braced for growth after the number of adopted advanced therapy medicinal product classifications jumped by 56% between 2018 and 2019.4
The scientific progress is quickly increasing the volume of cell and gene therapies the industry needs to produce to support clinical trials and commercial sales. A lack of manufacturing standardization is a contributing factor to the challenge of delivering the required volumes in the necessary timeframe and level of quality resulting in the current cell and gene therapy capacity only meeting an estimated 20% of total demand.5