The cat is out of the bag. The promise of cell and gene therapy is no longer conceptual. The FDA has already approved biologics like Kymriah, Yescarta, Zolgensma and Luxturna. The fundamental pathway has been laid out by pioneers of these new advanced therapies. Consequently, there are many new biotechs and pharma companies in the space resulting in increased demand for cell and gene therapy supplies.

The complexity of cell and gene therapy development and manufacturing is unique compared to small molecule production. Consequently, supply chain management for these therapies requires precise demand planning and forecasting, especially with regards to long lead times. Further adding to this complexity is an “inch worm” effect where suppliers must catch-up with demand and increase their own manufacturing capacity for the raw materials required in complex cell and gene therapy global manufacturing facilities. This also applies to equipment. Oftentimes, building the equipment, shipping, installing, validating, and qualifying can take several months. These pieces are not off-the-shelf and must be mapped out well in advance. In some instances, up to a year.

There is a lot of ingenuity in these products that require extensive process development and research to fine-tune the optimal batch requirements. The “recipe” for the manufacture of these can end up limiting raw material sourcing flexibility. Ideally, clients work hand in hand with The Discovery Labs manufacturing and scientific teams to create a process with the most appropriate raw materials at the earliest stages to ensure flexibility and scalability later in the process. For example, fetal bovine serum (FBS) is a critical raw material necessary as a growth medium in cell therapies and generally comes from livestock in New Zealand. If you can either avoid serum or validate another supply source you end up with more flexibility in your manufacturing processes.

In cell and gene therapy development and manufacturing we look to employ “just in time” inventory control—not too early, not too late. Just in time supply chain decreases your warehousing needs, inventory valuation, spoilage concerns and increases cash flow and results in a more efficient, lean manufacturing process. However, it is important to strike the right balance with cell and gene therapies because we are creating therapies that are personalized medicines. The global nature of the cell and gene therapy supply chain, especially during this COVID pandemic, increases these raw material challenges. If any of the raw materials originate from China or India, for example, the wait times are magnified.

Particularly during COVID, there must be strategic plans in place in the event of another global shut-down. Ensuring redundant sources, as well as stockpiling vital supplies, are part of any plan where COVID-related shortages could occur. The last thing we want to do is run out of a critical supply in the middle of a manufacturing run. Somebody’s life is literally on the line and our staff is cognizant of that fact.

Cell and gene therapy developers and their CDMO partners must employ strategic solutions to anticipate and mitigate supply chain issues as early as possible in the process. Organizations that can produce any necessary materials in house through vertical integration will hedge against shortages. For example, producing media and buffer in-house reduces the reliance on vendors, and provides redundant sources for this vital raw material. In early clinical development we add contingencies to buffer ourselves and look to validate alternative suppliers and raw materials as soon as possible. As a therapy progresses and we near commercial scale production we will have set ourselves up to employ a leaner process.

At The Center for Breakthrough Medicines (CBM), a pure-play CDMO focused on cell and gene therapies, we provide end-to-end support around both development and manufacturing. This level of service requires strong partnerships with key suppliers. Our ability to provide strong supply chain solutions is derived from a seasoned core team with some of the strongest credentials in the burgeoning cell and gene therapy marketplace.

We have partnerships with multiple vendors who can deliver on key raw materials such as media, reagents, equipment, and single use consumables. We also have strong relationships with the largest global couriers who rapidly and reliably ship sensitive patient materials, reducing vein-to-vein time.

Our staff has pioneered work on the development, manufacture and commercialization of key cell and gene therapies including Kymriah, Yescarta, Ryoncil, Algenpantucel-L, TIL–Lifileucel, JCARs, and many others. The collective supply chain and logistics knowledge acquired by the team during the development of these advanced therapies will be applied at The Discovery Labs Center for Breakthrough Medicines.