Article: The Commercialization of Cell and Gene Therapies: Supplying Patients in Need

By Gerhard Bauer; Simon Ellison

In light of recent advances in cell and gene therapy, and two gene-modified T-cell products gaining approval for the commercial market in the United States and the European Union, many are hoping these novel therapies will soon be available for the many patients in need of treatment globally. 

For this to happen, parts of the manufacturing and distribution processes involved in getting these products to patients, are in need of optimization. This is because, without a properly organized and executed shipping and distribution chain, living medicines will not reach the patients in the correct condition, and within the timeframe required for optimal administration.

Current shipping modalities for an autologous cell and gene therapy product, such as a CART-cell therapy, divide into the below three stages:

1. Incoming product: This is the product collected from the patient in the clinical setting, that is then sent to the manufacturing facility remote from the clinical site

2. Outgoing product: This is the product sent from the manufacturing facility to the patient in the clinical setting, remote from the manufacturing site

3. Quality control (QC) samples: These are the in-process and final product samples, sent from the manufacturing facility to a specialist-testing laboratory

For incoming and outgoing product, shipping temperatures should be between 2-8°C or below -150°C, and QC samples should at 2-8°C, -70°C or below -150°C. However, there are currently no uniform regulations in place for how to transport such samples.

Therefore, companies need to demonstrate to regulatory agencies that their shipping procedures and materials can maintain the integrity, safety and efficacy of products in their control, which requires validation studies. To accomplish the appropriate shipping of these products, they also need reliable transport containers. These containers have to be rugged, and the temperature of the product in them needs constant monitoring to avoid excursions.

Commercial airlines are also, often used as shipping carriers. Transcontinental flights are commonly involved and prone to cancelation, for reasons including the weather. Therefore, backup routes need to be pre-determined.

Moreover, to ensure future CGT products can treat patients at a global level, all organizations involved in the manufacturing and delivering of them must develop methods for increasing their capacity and streamlining shipping processes, while keeping them reliable and in an affordable price range.

Cell and gene therapies used in the treatment of cancers and other devastating diseases are highly promising – which we can recognize in the repeated cures for leukemia achieved in patients that did not have options previously. However, a lot of work and coordination will be required to bring such therapies to all patients in need, on a global scale.

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About the Authors

Gerhard Bauer

Director GMP Facility, Professor of Hematology / Oncology 
University of California Davis
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Simon Ellison

Cell and Gene Therapy Service Director from Sep ’17 – April ’20
World Courier
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