Gene Therapy: Maintaining Sterility At Low Temperature Storage

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 presented a challenge. 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 solution was found with West’s Ready Pack™ system, 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 the product shelf life and low temperatures.