Numerous cell and gene therapy solutions have exhibited promise as a source of potential treatment for an extensive range of chronic disorders. However, these innovative therapies typically introduce obstacles, such as necessitating ultra-low and cryogenic storage, which impact several steps of the life cycle of biomedical solutions, from their development to their administration.
Image Credit: B Medical Systems S.à r.l
The critical requirement for any preservation method is the product's stability for successful clinical outcomes. Expensive and labor-intensive preservation methods contribute to the cost of these technologies and may restrict the use of cell and gene therapy products. Thus, developing reliable, easy-to-use, and economical approaches is vital for the approval and sustainability of such scientific applications.
Cell and gene therapies
Several challenges have arisen in the clinical applications of cell and gene therapy, and countless setbacks have occurred over the last thirty years. However, modern medicine has recently experienced considerable progress, with new products appearing on the market.
Cell and gene therapy solutions, or advanced therapy medicinal products (ATMPs), are biopharmaceuticals based on cells, tissues, or genes for human use. These products are used to treat diseases that, for some time, have been deemed chronic conditions.
Cell therapy products include cancer vaccines, cellular immunotherapies, cells for therapeutic indications such as hematopoietic stem cells (HSC) and human embryonic stem cells (hESC), and induced pluripotent stem cells (iPSC), somatic cells that have undergone genetic reprogramming to have pluripotent stem cell characteristics.
These cells may derive from autologous (from the same individual) or allogeneic (from someone else) sources.
Conversely, gene therapy is a variety of methods for modifying the expression of a person's genes or repairing defective ones. It can be achieved by transferring nucleic acids (DNA or RNA) to a patient's somatic cells, which produces a therapeutic impact that corrects genetic abnormalities or therapeutically overexpresses critical proteins.
Gene therapy products can be bacterial vectors, plasmid DNA, viral vectors, and more. There is strong evidence that these kinds of ATMPs could be applied to treat many diseases, such as cancer, cystic fibrosis, diabetes, heart disease, and haemophilia.
Presently, a limited number of cell and gene therapy-based solutions are available on the commercial market. Trials of hundreds more are currently underway in registered clinical trials, and it is anticipated that at least 10-20 ATMPs will be authorized each year by 2025 in the USA alone.
With so many solutions on the horizon, it is critical to understand whether they will require specialized cold chain solutions for safe storage and transportation.
Gene therapy solutions are more stable than cell therapy solutions, as their storage and distribution can usually exploit approaches taken from traditional pharmaceuticals and biologicals.
This is especially true as unique cell therapy solutions consist of exceptionally labile mammalian cells, which only remain viable at extremely low temperatures. For instance, depending on their type, stem cells will require storage temperatures ranging from -80 °C to -196 °C.
This underscores the requirement for reliable cold chain systems to ensure the storage and timely delivery of consistently effective and reliable cell and gene therapy solutions until the time of administration.
Moreover, although cryogenic freezing will mainly be applied in this space, numerous use cases necessitate ultra-low freezers, including preserving microbial stocks in addition to storing human cells and tissues, which are dependent on the biologicals' storage duration.
Reliable ultra-low freezers for the storage of ATMPs
B Medical Systems S.à r.l is a global leader in the manufacturing and distributing of state-of-the-art medical cold chain solutions, including ultra-low freezers.
Today, the company produces five different models of ULTs across all its business segments, with storage capacities that range from 206 L to 863 L. These globally recognized models can achieve temperatures as low as -86 °C and as high as -20 °C and are known for their reliability, quality, safety, and energy efficiency.
B Medical Systems' ultra-low freezers use an innovative cooling system to guarantee a constant and even temperature distribution across the unit. This, combined with the insulated inner doors, enhanced gasket seals, frames, and insulation, facilitate uniform temperature distribution necessary to deliver a dependable storage environment and optimum sample safety.
Moreover, the models offer exceptional pull down, recovery, and holdover times due to their innovative structure, design, and technology, ensuring the cabinet maintains the intended temperatures even when adverse events occur.
These ultra-low freezers use natural refrigerants to limit the environmental impact of such refrigeration solutions while enhancing overall cooling efficiency. All ULT models by B Medical Systems are EU MDR/USD FDA class II(a) medical devices, and the company additionally provides models with the Energy Star certification.
Finally, the company's ULTs have developed built-in audio-visual alarms that signal high/low temperatures, open doors, sensor failures, and other potential technical failures.
These features are compatible with °B Connected, cloud-based remote monitoring software that facilitates the holistic remote monitoring of ultra-low freezers, such as the acquisition, recording, and visualization of the temperature data. The software can also alarm forward via email or SMS and follows the 21 CFR Part 11 (subpart A and B) regulation.
Ultra-low freezers as an alternative freezing environment
A viable cold chain for cell and gene therapy solutions should sustain not only the stability of the products but also use operational methods that are not reliant on expensive facilities and equipment for large-scale biobanking.
It is improbable that a universal cryopreservation method could be developed due to the variety of the products and their diverse temperature profiles. However, numerous studies have reported that storing products at temperatures around -80 °C in ultra-low temperature freezers can be sufficient for many of them.
Therefore, ultra-low temperature freezers are a suitable option for laboratories conducting cell and gene therapy research, enabling them to store extremely thermosensitive and valuable samples reliably in an alternative freezing environment.
About B Medical Systems S.à r.l
B Medical Systems S.à r.l (formely Dometic/Electrolux) is a global manufacturer and distributor of medical cold chain solutions. Based in Hosingen, Luxembourg, the company was founded in 1979, when WHO approached the Swedish manufacturing giant Electrolux to provide a solution to safely store and transport vaccines around the world. Across the 3 major business portfolios of Medical Refrigeration, Blood Management Solutions, and Vaccine Cold Chain, the company currently offers 100+ models. B Medical Systems’ major products include Laboratory Refrigerators, Laboratory Freezers, Pharmacy Refrigerators, Ultra-Low Freezers, Plasma Freezers, Contact Shock Freezers, Vaccine Refrigerators (Ice-Lined Refrigerators and Solar Direct Drive Refrigerators), and Transport Boxes. All products have integrated 24/7 temperature monitoring capabilities that further ensure that these products offer the highest level of safety and reliability.
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