Issue: January / February 2022

Cell and gene therapies (C&GT) are a fast growing and promising class of medicines that offer the possibility of curing conditions with a genetic basis that have few or no other therapeutic options for patients. From a turbulent start in the 1990s, with some notable setbacks in clinical trials, the promise of these therapies has now become a reality, with numerous market approvals in recent years, a robust clinical pipeline and strong industry and investor interest. The sector is both immature, from the regulatory and CMC perspectives, and growing fast. Numerous challenges will need to be surmounted in the coming years to ensure that these therapies can reliably reach patients, but the future looks very promising.