Small volumes of ATMPs: Safe handling of single-use bags

In recent years cell and gene therapies (CGT) have proven to be promising approaches to personalized medicine. Patients needing a cell and gene therapy as an advanced therapy medicinal product (ATMP) rely on their treatment being ready as soon as possible. For example, there are CAR T-cell therapies that have been approved for for very aggressive and hard-to-treat types of cancer.

Moreover, CGTs yield a small volume of individualized product for each single patient. Loss of the shipment or damage to the transported goods have serious consequences for patients. Not only a safe transport, but also speed, accuracy and process flexibility play an important role in manufacturing and patient safety.

Therefore, reliable, safe, and scalable solutions are needed. RoSS.KSET satisfies all those requirements. It is an integrated solution to protect and safely transport single-use bags for small volumes at low-temperature refrigeration. The secondary packaging also enables advanced fluid management and cold chain logistics by accessing modular aliquoting of small volumes into single-use containers and plate-based freezing for the best freezing performance.

Single-use bag protection for ATMP

During autologous cell therapies, each patient receives an individually manufactured product. Therefore, the drug substance is of unusually high value. Having a safe and standardized logistics process in place is essential for success.

But also allogeneic cell therapies or viral vector production for gene therapies require a safe handling of small volumes. In this case even in different scales. In most cases, the volume of ATMP remains small. But either bag sizes or bag quantities can have an influence on manufacturing facilities. 

RoSS.KSET is a solution to safely protect small volume single use bags of all types used in cell and gene therapies. It is designed to be used effortlessly by medical and biotechnological personnel: robust and closed construction, extra space for safe storage of satellite samples and vials. 

Read more: How to avoid single-use bag leakages in bioprocessing

Meeting high standards in cell and gene therapy

Meeting the high standards in cell and gene therapy requires a huge effort in process and logistics optimization and standardizing. With highly personalized drug substances, e.g. CAR T-cells, strict requirements for the associated logistics and supply-chains have to be met. These parts of the CGT approach are essential to treat patients effectively and economically. Here, loss of drug substance is a major issue. 

Beyond implementing cGMP production into manufacturing facilities, automated solutions help fulfil 21 CFR Part 11 requirements. Reported audit trails and monitoring support the way to a more efficient manufacturing process of advanced therapies.

Small scale solutions

It all starts small. Therefore small scale solutions are required in various areas of pharma applications. Here is an except of where advanced solutions for small volumes are required

• Clinical studies from Phase I to Phase III
• Clinical trials
• Cell Banking 
• Autologous and allogeneic cell therapies
• Gene Therapies, including viral vector production
• Non-viral vector production, such as LNPs.
• Plasmid DNA

It all involves the processing of small volumes of very highly valuable substances. It is essential to minimize any risk of failure, be it from inconsistencies and deviations while handling or damage of single use bags during the transport. To make ATMPs manufacturing work on a world-wide scale, innovative automated solutions to all these issues are needed.

Secure protection of small volume bags

The handling of highly valuable small volume single-use bags is challenging. At the cold temperatures needed to keep the drug substance from disintegrating, single-use bags become prone to breakages from impacts or vibrations during transportation. Even a tiny leak has serious consequences for the seriously ill prospective recipient of the CAR T-cell therapy. But also overfilling through manual filling can be a reason for leakages in single-use bags.

Single Use Support’s RoSS.KSET was specifically designed to secure single use bags while being in the supply-chain. Their 3D foam lining offers protection against shocks and vibrations, and the robust construction offers a safe shell against impacts. The materials were chosen for their thermal conductance to ensure optimal behaviour during freezing and thawing. The compact design allows for efficient storage in cryoconditions.

Fast and flexible transportation of small volume bags

Cell and gene therapies require the handling of miniscule amounts of liquids, sometimes less than 5 mL. Standardization and automation of such processes are needed to reduce error rates and contamination risks.

Single Use Support solved these challenges with a series of platform systems:

• RoSS.FILL is an automated aseptic filling platform with unprecedented filling accuracy. Its version for small volumes can fill single-use bags and medical devices with less than 5mL and unbeaten precision. It allows the automated processing of liquids with high flexibility.
• RoSS.pFTU and RoSS.LN2F are freezing and thawing platforms that are scalable and compatible with single use bags of all sizes and customized types. Both enable controlled freezing liquids in single-use bags. 

Together with the protective shell RoSS.KSET, Single Use Support's filling and freezing platform solutions are an ideal part of the logistics side of personalized medicine.

How Single Use Support's RoSS KSET helps to improve cell and gene therapies

Cell and Gene therapies, such as CAR T-cell therapy, have proven their potential to become standard treatments to hard-to-treat diseases. Among the obstacles to their commercialization are the handling and logistics of small volumes of highly valuable and irrecoverable individualized drug substances.

Single Use Support has developed end-to-end solution in transporting small but valuable therapies safely. As response to various day-to-day challenges of manufacturers, the platforms RoSS.FILL and RoSS.pFTU enable a safe and monitored aliquoting, freezing and thawing of small volumes into single use bags. The complementary shell RoSS.KSET protects the products from damage during storage and transportation.

Single Use Support strives to further improve the ATMP manufacturing and make advanced therapies an affordable treatment option for patients all over the world.