Novo moves into amyloidosis not long after Intellia made a splash here.
After a patient develops inhibitors questions emerge about a new approach to treating haemophilia A.
Animal toxicity findings with ARO-ENaC come not long after Ionis’s similar setback.
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.
Novartis makes progress with its most valuable pipeline asset, but others also hope to make a mark in IgA nephropathy.
Full data from the Helios-A trial support vutrisiran’s profile as a more convenient Onpattro, though that’s probably as good as it gets.