In vivo editing data advances lift hopes for better access

Displacing cell therapies with genetic medicines has become a major goal among a set of gene editing companies, as some pivot from ex vivo to in vivo editing of stem cells and others begin creating CAR T cells inside patients. If successful, the next-generation products could solve major access barriers that have prevented patients from benefitting from marketed CAR Ts and genetically modified stem cell therapies.

Last week delivered a pair of milestones for in vivo stem cell editing. Editas Medicine Inc. (NASDAQ:EDIT) and Tessera Therapeutics Inc. each presented preclinical data suggesting that it may be possible to modify gene expression in the difficult-to-access hematopoietic stem and progenitor cell (HSPC) type, without taking the cells out of the body. Editas also announced it was shifting the focus of its sickle cell development program from ex vivo to in vivo editors...