In order to bolster its gene therapy pipeline, Novartis has agreed to pay US$87.5 M in cash for Avrobio’s investigational haematopoietic stem cell (HSC) gene therapy programmes for the treatment of cystinosis. The deal is driven by Avrobio’s Phase I/II gene therapy, AVR-RD-04, which has already demonstrated promising efficacy in patients with previously treated cystinosis. After a turbulent few months for the biotech, the deal provides Avrobio with the funds to extend its operating runway and validation of its HSC gene therapies and plato® technology.