The first sickle cell and beta thalassaemia patients have been enrolled into a Crispr/Cas9 trial, heating up a race with Bluebird, Sanofi, Sangamo and Editas.
Tiny signs of a therapeutic benefit are offset by safety worries, as the company pivots to higher doses and second-generation projects.
A deal between J&J and Meiragtx in eye disease shows growing interest from big pharma as gene therapy's commercial issues come into focus.
Reaction to data from Sangamo's ongoing genome-editing trial shows that small numbers can equal big risks.
Beam Therapeutics is developing a new method of Crispr genome editing, but it could get caught up in the same old patent problems.