"We deliver the difference for patients."

Dr. Koliwer, the market for cell and gene therapies (CGT) has been growing rapidly for years. What makes this form of therapy so exceptional?

Dr. Judith Koliwer: The underlying idea of one of the most successful CGT approaches, CAR-T therapy, is to take cells from diseased people and genetically modify them in such a way that - when injected back into the patient’s body - they can precisely find and fight cancer cells. This personalized form of therapy opens up an entirely new range of treatment and healing options for patients. This is already saving many lives for which, until recently, there was little hope.

At what stage do you currently see CGT?

After a major breakthrough in 2017 - the first approval of the CAR-T therapy I just mentioned - and several other significant advances since then, we are now in the midst of a phase of exponential implementation. CGTs, or ATMPs (Advanced Therapy Medicinal Products), are undoubtedly the fastest growing market within the pharmaceutical industry right now. 

Last year alone, both the number of approvals and the sales volume almost quadrupled worldwide. There are numerous good and well-tested products on the market, especially for the treatment of certain types of cancer such as leukemia, hereditary diseases like cystic fibrosis, or non-hereditary diseases including Alzheimer's or Parkinson's disease. 

However, with the growth of the industry massive challenges are arising: For example, are we able to produce these highly complex and personalized therapies in large numbers? How do we manage the extensive documentation requirements and the huge amounts of data? And how do we learn quickly and effectively from previous therapy experiences?

So how exactly are innovative software solutions like Körber's Werum PAS-X going to help us meet these challenges?

As said, the production processes in cell and gene therapy are of high complexity – from cell sampling via the tailor-made manufacturing procedure to the actual patient treatment. Herein lies the source of numerous errors that are potentially life-threatening – particularly when the assignment of therapeutic agent and patient is carried out manually.

Digital Manufacturing Execution Systems such as Werum PAS-X MES greatly simplify both the establishment and documentation of this Chain of Identity. Hence, they can help to ensure that the coherent cells arrive at the right patients. Additionally, they support the Chain of Custody by recording and monitoring all events, the devices used and the participants involved throughout the entire process. 

Our customers can digitize the entire production process, collect all the data they need and obtain all the relevant information in real time - whether it's managing equipment, inventory and materials, documenting the process with Electronic Batch Recording (EBR) or administering manufacturing recipes with Master Batch Records (MBR). 

And finally: When all data is available digitally, you can react quickly and flexibly to new conditions, as this was the case during the COVID-19 pandemic, for example. Also, the legal situation is progressing for these new therapies, as the approval criteria of the responsible U.S. authority, the FDA, and the EU authority, the EMA, are also adjusted as new findings emerge.

The high cost per therapy is considered the main obstacle to a wider application of CGT. Can PAS-X software solutions pave the way for CGT to become a mainstream therapy?

Taking into account, that there are often six-figure sums involved per therapy, the pressure is obviously on to both maximize the chances of success and reduce the cost of the treatment itself. One important aspect of this is easing the burden on staff. The CGT market is growing much faster than companies can train and hire suitable personnel. 

It is therefore all the more crucial to relieve the existing experts in such a way that they have as much time as possible for the actual therapy work and offer the companies opportunities for upscaling. Digitization of processes can play an essential role here, for example by automating calculations within the process and using known algorithms for prescription decisions. In addition, the documentation effort can be significantly reduced by reading information from barcodes instead of manual documentation. Another important aspect is the standardization of processes - a major challenge, as the source material can be highly variable. 

With digital systems, flexibility within the process can be precisely defined: Is it possible to have different paths at which points? When and where should the system provide information on the basis of which experts can make decisions? Where is the process fixed and can only be changed within the framework of a documented deviation? By capturing digital data, this approach can then be used to further optimization and iterative standardization of the process.

Can PAS-X also support the evaluation of the digital data?

Without a doubt. Digital data can be merged, visualized, and analyzed using PAS-X Data Access and PAS-X Savvy from a variety of sources, including those outside PAS-X. This allows large amounts of data to be evaluated including learning results from previous therapies in order to optimize the process. There are so many parameters to consider and process steps to evaluate, so digital systems surely are a great relief. We increase the success of therapy in this way, too – and that's what's driving us. We can really deliver the difference for patients.

Can you name any customers that Körber is already working with – and how does the collaboration work?

Körber is the market leader for digital manufacturing execution systems for pharmaceutical, biotech and cell & gene therapy and works for leading organizations around the world. The internationally active cell therapy manufacturer Takeda, for example, uses PAS-X MES for data and technology transfer at its site in Cambridge. And China's vaccine manufacturer Walvax Biotechnology also relies fully on PAS-X at its digital factory in Yuxi. On top of that, our collaboration with start-ups is growing rapidly. 

All our customers have a high degree of expertise at all levels. We intensively share ideas and learn from each other on an ongoing basis – whether bilaterally, at industry conferences, or on business partner platforms. At Körber, we are specifically developing our expertise in supply chain platforms through strategic partnerships with companies such as Hypertrust Patient Data Care, TrakCel, and Vineti. 

We aim to increase CGT production performance and transparency with standardized interfaces for manufacturing and logistics solutions, as well as create comprehensive ecosystems for digitized manufacturing planning that integrate all levels and players.

How will CGT industry evolve in the coming years?

Market dynamics will continue to grow - focusing increasingly on allogeneic forms of therapy. Until now, autologous therapies have been leading the way, based on the modification of the body's own cells. In the allogeneic variant, patients are treated with cells specifically tailored to their needs from healthy donors or cell banks. A single donor can thus supply an entire group of patients. While this opens up new opportunities in terms of scaling and mass efficacy, it also poses new risks, such as side effects and data processing. As you can see, a lot is happening. In the fight against widespread diseases, we are just beginning to fully exploit our technological potential.

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