Cell and gene therapy - current legal issues

Two CAR T cell therapies have already been approved for use by the European Medicines Agency (EMA) and the US Food & Drug Administration (FDA). Both Kymriah, produced by Novartis, and Kite/Gilead’s Yescarta use modified T cells to treat blood cancers. Gene therapies based on AAV technology are also moving into the clinic, with Novartis’ Luxturna now approved for rare inherited eye conditions in both the US and Europe. 

Cell therapies are predicted by some to achieve a market of $8.21bn by 2025. Kymriah received funding approval from the NHS less than 10 days after its marketing authorisation – one of the swiftest approvals in NHS history.

Developing these advanced therapies presents a unique set of challenges, both practical and legal. We review the issues in a series of three articles, dealing with the regulatory framework, the patent landscape and commercial partnerships.