How to keep pace with evolving regulatory standards for gene and cell therapies

Clive Glover, director, strategy, Pall Corporation, provides an Expert View on what recent developments mean for the industry and the importance of safety in gene therapy manufacturing.

While the concept behind gene therapies is simple and elegant, it has taken decades for it to be realized. But since the approval of the first five gene or gene-modified cell therapies in the USA – that is, Zolgensma (onasemnogene abeparvovec), Luxturna (voretigene neparvovec), Kymriah (tisagenlecleucel), Yescarta (axicabtagene ciloleucel), and Tecartus (brexucabtagene autoleucel) – this new therapeutic modality has been growing at an almost exponential rate.

While there were no issues raised by regulatory bodies regarding the efficacy and safety profiles of these early-in-class medicines, more recent applications have encountered challenges. High-profile requests for more data, like those received by Sarepta Therapeutics (Nasdaq: SRPT) and BioMarin Pharmaceutical (Nasdaq: BMRN) from the US Food and Drug Administration (FDA), and sobering issues, such as the third patient death in the high-dose arm of the Audentes’ ASPIRO trial, have rattled the industry. These requests, along with the FDA’s refusal to even review the biologics license application for Bristol Myers Squibb (NYSE: BMY) and bluebird bio’s (Nasdaq: BLUE) idecabtagene vicleucel CAR-T therapy, indicate that the regulatory burden and scrutiny on the regenerative medicine industry is likely to increase and set to do so by quite a margin. And this is as it should be.