Overcoming the Challenge of Maintaining Sterility at Low Temperature Storage

Gene therapies are developed by modifying a person’s genes and are often targeted to treat complex and chronic diseases. Due to advancements in science and research, gene therapy is positioned to transform treatment options for patients who are living with these incurable diseases. There is a growing pipeline of gene therapies globally, with 352 global trials underway at the end of 2019, up 12% from 2017.^1.

As advancements in gene therapies continue to grow, West is committed to providing packaging solutions for these complex treatments. Learn more about a biotechnology company, with a recently approved gene therapy, that partnered with West to select a packaging solution that would allow them to meet their needs for storage at low temperatures and still get to market quickly.

The Challenge

Bringing the first gene therapy for pediatric Spinal Muscular Atrophy to market in a leak and break resistant container which maintains sterility over product shelf life, through freeze and defrost cycles. Container Closure Integrity (CCI) must be proven and consistent for storage at -80°C, and the solution must be ready to scale-up quickly due to orphan designation and Fast Track status of the program.;

The Considerations

When planning gene therapy drug development, it is critical to ensure known challenges are mitigated; therefore, a chosen packaging system needs to be proven, reliable and able to be scaled-up, at short notice. The supply chain for gene therapies requires the drug to be frozen, then stored at -80°C before being thawed for injection into the patient. It is critical that packaging components used in this complex supply chain are compatible with both the drug and specialized conditions required for storage and distribution. Having secured Fast Track designation from the FDA, the customer was able to bring its gene therapy to market quickly, allowing patients with the highest need to receive treatment in record time. The packaging system selected helped to facilitate the speed with which the drug moved from development to commercialization. The chosen system provided a range of container sizes with quality components, in ready-to-use format, proven to work as a system and available in small quantities.

The Solution

West’s Ready Pack^™ system was selected, which includes a Daikyo Crystal Zenith^® vial, NovaPure^® stopper and Flip-Off^® CCS seal. These high-quality components combine to create a proven system which ensures CCI over theproduct shelf life and low temperatures. In 2019 the FDA approved the first gene therapy treatment for pediatric Spinal Muscular Atrophy using West’s container system.

Customers can purchase the small quantity Ready Pack system directly from West’s online store. Buying this offering online allows for quick delivery and enables customers to meet the short timelines often associated with orphan drug designation and Fast Track approval. Daikyo Crystal Zenith vials and associated West components were the right choice for this customer, as vial sizes from 2-5mL supported expedited scale-up activities from lab to the patient. The Ready Pack system offers all these benefits without the unwanted cost of excess stock and is the perfect choice for bringing a gene therapy to market.

Ready Pack^™, NovaPure^® and Flip-Off^® are trademarks and registered trademarks of West Pharmaceutical Services, Inc., in the United States and other jurisdictions.
Crystal Zenith^® is a registered trademark of Daikyo Seiko, Ltd.
Crystal Zenith^® technology is licensed from Daikyo Seiko, Ltd.