CGT is the acronym for Cell and Gene Therapy and describes a multitude of new, personalized therapies that are promising cure where traditional approaches have been failing, such as Crohn’s Disease, leukemia and various tumor diseases. Unlike traditional biopharmaceutical products, the CGT approach uses the patient’s own cells, which are extracted and then genetically manipulated before being reintroduced via injection.
With a short shelf-life of only up to 48 hours, the manipulated material has to be reintroduced within a maximum of two days – this poses challenges on several fronts, not least logistics for CAR-T cells. A disruption in the supply chain can render the entire compound (and all the work and money invested) useless, as can contamination caused by seemingly minor glitches during production and subsequent handling. But when it comes to life-sustaining medication, there usually are no second chances.
These issues notwithstanding, Cell and Gene Therapy is nothing short of representing a paradigm shift in healthcare and disease treatment. It is expected to, over time, revolutionize medicine. The concept itself, which sees the adaptation of cells and genes in order to treat diseases in a personalized manner, is nothing new, however, big players in the pharmaceutical industry have recently started to invest heavily in this field.
This comes in the wake of the FDA’s approval of CAR-T therapies such as Kymriah and Yescarta. First successful treatments have triggered global interest and increased international research. Currently, the worldwide market is predicted to grow from $18.9 billion in 2016 to over $66 billion by 2022 and a compound annual growth rate of 23.3% in the same time frame.
In short, personalized medicine is changing the pharmaceutical and medical sectors. The idea is to bring the right treatment to the right patient at the right time and it has shifted the role of pharma companies who are moving from simply being manufacturers to providing entire solutions. As a result, they require new structures and logistics processes to allow for seamless procedures that are required in any international cooperation.
With the cells being taken from the patient, CGTs naturally call for a logistics process that can handle much smaller volumes than established processes for blockbuster production. Subsequently, the implementation of suitable manufacturing facilities and reliable supply chains as well as logistics processes is among the main challenges arising from this shift.
Secure Logistics in biopharma have always played a major part in terms of the development and manufacturing of valuable and often highly sensitive drug substances. This is still – if not even more so – true with the rise of Cell and Gene Therapies that use a patient’s own cells and require the production of one clinical batch for each individual patient.
In cases where external donor cells are used, the shipping – often covering long distances across several countries or even continents – adds another obstacle, especially when taking into regard the short shelf-life of such products. As with blockbuster medication, a reliable and trackable shipping process is imperative in order to guarantee the product’s safe arrival at its destination.
The established systems provided for controlled and reliable filtration, filling and freeze-thaw processes are usually geared towards vast volumes. However, the amounts needed for CGT as well as drug substances that still have to go through the research or clinical trial phase need the same meticulous care and handling.
No matter if the product at hand is produced in large quantities or is a personalized drug, contamination of the finalized compound has the same dire consequences, both financially and feasibly. This is where a scalable logistics process can provide a remedy. The Austrian single-use technology company SUSupport’s with it’s single-use filtration system RoSS.FILL is a flexible platform for the monitored allocation and distribution of drug substance in single use bags that are integrated in the RoSS® system.
The liquid path is fully disposable and can accommodate a multitude of options, including sterile filtering and segregation. It can fill up to 200 liters per hour and is scalable as well as compatible with other necessary systems such as integrity testing for single use bags.
After all, the tiniest rupture in a single use bag can cause contamination of the entire content, with dire consequences, not least for the patient waiting for his personalized therapy.