With pivotal sepofarsen data due, the group will hope to get one up on Editas.
SAR445136 could take on Crispr and Vertex’s CTX001, but plenty of other groups have the same idea.
Data on five patients treated with the Crispr therapy EDIT-101 raise doubts about efficacy and safety.
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.
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.