Proqr eyes its first big test
With pivotal sepofarsen data due, the group will hope to get one up on Editas.
Ash 2021 – Sangamo and Sanofi enter the sickle cell gene editing fray
SAR445136 could take on Crispr and Vertex’s CTX001, but plenty of other groups have the same idea.
Editas falls well short of Brilliance
Data on five patients treated with the Crispr therapy EDIT-101 raise doubts about efficacy and safety.
Intellia proves its point
The first clinical evidence for in vivo gene editing, and a surge in valuation, is to be celebrated. But there is much more to do.
Proqr hopes to usherin a new ophthalmic therapy
The group’s antisense project is the only game in town for Usher syndrome, but its latest trial is not a slam-dunk.
One victory for biopharma over the coronavirus in 2020
A rollercoaster year for biopharma ended with investors firmly deciding that the pandemic was an opportunity, not a threat.
Finding the next Prevail
An $880m buyout by Lilly shows that some gene therapy companies have not yet priced themselves out of the market.
