Editas turns away from ex vivo

Launching ex vivo gene or gene-edited therapies is tough, and Editas has decided that its efforts will be better spent elsewhere: the group said on Tuesday it would look for a partner for its sickle cell disease contender renizgamglogene autogedtemcel (reni-cel). Editas will now focus on its in vivo programme. It has long been hoped that, compared with ex vivo, in vivo therapies could be more convenient and cheaper, but a difficulty in blood disorders has been directing such projects to haematopoietic stem and progenitor cells (HPSCs); current delivery vehicles like lipid nanoparticles tend to head for the liver. Editas highlighted its so-called targeted LNP, and reported an editing level of 29% in HPSCs – although this has only been achieved in mice so far. One question is who might want to licence reni-cel given the costs involved with launching ex vivo products. Vertex and Crispr Therapeutics’ Casgevy and Bluebird Bio’s Lyfgenia are approved, but Bluebird recently slashed its workforce in its quest to break even. Editas, which previously abandoned in vivo eye projects to focus on reni-cel, has $318m in the bank, but a long road ahead. Shares dipped 10% on Tuesday morning.

Reni-cel (EDIT-301) development

Source: OncologyPipeline.