A wave of clinical trial applications for new cell and gene therapies has reached the US Food and Drug Administration triggering a review of clinical guidance documents for developers. In an announcement on 15 January, Scott Gottlieb, the FDA commissioner, said new guidances are being drafted for gene replacement therapies as well as for cell-based gene therapies such as the chimeric antigen receptor (CAR) T cell drugs.

Scientists at the University of Cambridge have identified mechanisms by which mutations in the ATM gene can lead to cancer drug resistance and how this can be counteracted by changes in other genes. The findings, reported on 8 January 2019 in Nature Communications, show how cells respond to DNA damage as well as highlight potential therapeutic targets for the genetic disease, ataxia-telangiectasia (A-T).