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.
Centessa claims a place in the antitrypsin deficiency pipeline
But investors will need more convincing; and others are more advanced, with Arrowhead and Inhibrx ones to watch.
Novo bets against Crispr for amyloidosis
Novo moves into amyloidosis not long after Intellia made a splash here.
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.
Vertex’s second try at a rare lung disorder
A small molecule for alpha-1 antitrypsin deficiency faces a key clinical test, and its chances of success aren’t good.
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.
Higher dose points Sangamo no closer to zinc finger success
Tiny signs of a therapeutic benefit are offset by safety worries, as the company pivots to higher doses and second-generation projects.
