The group’s antisense project is the only game in town for Usher syndrome, but its latest trial is not a slam-dunk.
A rollercoaster year for biopharma ended with investors firmly deciding that the pandemic was an opportunity, not a threat.
An $880m buyout by Lilly shows that some gene therapy companies have not yet priced themselves out of the market.
Investors are piling into biotech IPOs, but the track record of previous years' new issues shows how hard it is to pick winners.
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.