SAR445136 could take on Crispr and Vertex’s CTX001, but plenty of other groups have the same idea.
But investors will need more convincing; and others are more advanced, with Arrowhead and Inhibrx ones to watch.
Novo moves into amyloidosis not long after Intellia made a splash here.
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.
A small molecule for alpha-1 antitrypsin deficiency faces a key clinical test, and its chances of success aren’t good.
A rollercoaster year for biopharma ended with investors firmly deciding that the pandemic was an opportunity, not a threat.
Tiny signs of a therapeutic benefit are offset by safety worries, as the company pivots to higher doses and second-generation projects.