Manufacturing in pharmaceuticals must evolve to accommodate increased research and usage of cell gene therapy. This can be done via acquisitions and mergers or through the expansion of manufacturing capabilities within a single company. Cell and gene therapy use genetic material, or DNA, to manipulate a patient’s cells to treat an inherited or acquired disease. While cell and gene therapy or CGT will not overtake established biotechnologies like monoclonal antibodies, immunoglobulins, or Messenger RNA vaccines anytime soon, it will likely emerge as a meaningful and profitable industry subsector very shortly. There are over 1000 clinical trials for GCT presently, and the market for CGT production is predicted to reach 13.8 billion by 2026. Therefore, pharmaceutical manufacturing will have to find a way to accommodate the production of both established biotechnologies and emerging CGT. This change may also represent new competition within the pharma industry as smaller manufacturers may be more able to pivot to meet emerging demands.  

 Cell and gene therapy is incompatible with traditional pharmaceutical bulk manufacturing methods used for conventional pharmaceuticals. Traditionally, manufacturing methods in pharma focus on creating high-quality made-to-order bulk products sold in mass quantities to many different patients. Bulk manufacturing volume is based on market demand and generally takes over a year to complete. Cell and gene therapy manufacturing differs significantly as it requires genetic information from an intended patient before it can be manufactured. This means that each treatment is made to order, and demand cannot be easily predicted. It should also be noted that CGTs are created within days or weeks after biological information is extracted from the intended patient rather than months or years.  

 To keep up with or even participate in cell and gene therapies, manufacturers must find ways to accommodate the market demands of both conventional biotechnological production and CGT. This likely means developing facilities that utilize smaller production runs that are flexible and can meet requests within a days’ notice. This change creates many logistical challenges; for instance, CGT’s creation relies on parts of the process occurring in hospitals or clinics, within cold chain transit, as well as manufacturing facilities. Having part of the process be outsourced challenges quality control. This has led to calls for CGT manufacturing systems to be portable, small, and implantable in hospitals. Manufacturing systems created for mobile production and implantability in hospitals must record batch data and maintain standards. These systems should also manage patient variabilities, ¨ such as choosing between different consumable sets or the timing and sequencing of events at the processing stage¨. Near patient, model manufacturing creates new safety requirements such as high aseptic handling procedures during transportation and consistent, standardized testing. 

 CGT production is changing the landscape of Life Sciences manufacturing. Cell and gene therapies have created a need for new operational capabilities, supply chain integrity, and distribution channels. Therefore, pharmaceutical manufacturers should take advantage of available information on small batch production, data informatics technologies and accelerate the adoption of novel production methods. Life Sciences manufacturers can realize the full range of benefits in their digital investments by connecting these systems to the organization and its business operations. Pharma companies who do not learn how to shift into this market will be left behind as other companies rush to fill the void. 

References:

1. Cell And Gene Therapy Flips Life Sciences Manufacturing On Its Head
2. Medicines in Development for Cell Therapy and Gene Therapy

STRAMMER