Vial Containment Systems For Gene Therapies: Series Overview

By Margaret A. Craig, Manager, Scientific Communications Rui Li, Ph.D., Senior Scientist, R&D, Cryobiology, and T. Page McAndrew, Ph.D., Director, Scientific Communications

Gene therapy aims to treat or cure specific cancers, genetic conditions, and infectious diseases by correcting their underlying genetic problems. This involves modifying living cells by replacing or inactivating disease-causing genes, or by introducing new or modified genes. Viral vectors—viruses adapted to efficiently deliver genetic material into cells via a process called transduction—are a primary method for administering these therapies.

This chapter discusses basics of gene therapies:

• Function
• Anticipated growth rates
• Companies involved and estimates of therapies in pipeline
• Examples of commercial products and indications
• Need for ultra-low temperature storage and compatible vial containment systems