West Pharmaceutical Services, Inc

Vidya Murthy

It is an exciting time to be in the cell and gene therapy industry. The astounding growth anddevelopment we have witnessed in the cell & gene therapy industry over the last decade is bringing life-changing therapies to the patients who have been so anxiously awaiting them.

At the same time, as with any new drug class, challenges that may not have been considered during the drug development phase may suddenly show up and stand in the way of overcoming that last hurdle: Packaging and delivering your precious new molecule to the patient.

You might be new to delivering drugs because your therapy is first in its class; or you might be a seasoned pharmaceutical professional that has always worked with other types of molecules. The bottom line is that a therapy that involves cells or genes is a very different beast from a small molecule, a peptide, or even a protein therapy. You need to think about its special needs early and often to safeguard it all the way from development until it reaches the patient.

Doing so will minimize your risk of not meeting timelines or staying within budget, and most importantly, it will minimize risks to the safety and efficacy of your gene therapy drug product, from the bench to the patient.

What are the risks of not considering packaging early and often?

● Compromised safety and efficacy: cell and gene therapies are highly sensitive to factors like particles and leachables, so meeting the tightest industry material and container closure integrity (CCI) specifications is absolutely critical.

● Loss of expensive product upon breakage of vials: your therapy is significantly more expensive than traditional therapies. Loss of even one vial could be very damaging to your bottom line.

● Loss of product to the container: cells and genes are more likely to interact with surfaces than other types of molecules. Not considering the type of material that your containers are made of could result in significant product loss. 

Mindfulness of Temperature Sensitivity

One common struggle for cell and gene therapies is their temperature sensitivity. Drug companies with products that can be stored at room temperature or even at 4°C do not need to worry about the dramatic physical property changes that materials like elastomers and glass undergo at extremely low temperatures, leading to loss of CCI or cracking upon thawing.

Improved particle and leachable specifications and break resistance

When looking for packaging for your cell and gene therapy, both particulates and leachables are concerns that must be considered early in the drug development phase to reduce the risk of losing precious time to market.

Improved viral vector recovery over glass vials

The material properties of the storage container should be an important consideration for the container choice of viral gene therapies.

Studiesindicate that compatibility of the storage container for a viral vector therapy can affect product quality and certaincontainer systems may serve as a preferred container for commercial viral vector storage.

Bottom Line

Cell and Gene therapies have a set of very specific containment needs that, if not considered early and throughout the development process, could lead to significant delays and product losses. Whether you are a small biotech that is new to delivering therapies, or a big pharma company looking to utilize the best containment system for a new type of molecule, you will need a partner that combines decades of experience with forward thinking innovation.