Benjamin McLeod
One of the most important developments to watch over the next 12–18 months in cell and gene therapy is the evolving regulatory landscape. Markets such as China are becoming increasingly competitive, which may influence how the U.S. FDA approaches regulatory frameworks. We may also see growing momentum around right-to-try initiatives and state-level legislation, creating alternative pathways for responsible patients willing to take on some of the risks associated with emerging therapies. With more than 10,000 rare diseases worldwide, it is unrealistic to expect that each therapy can follow a traditional $2-billion development pathway. The industry will need to explore balanced regulatory approaches that maintain safety while enabling innovation and access for rare disease patients.
