Sigilon hold hurts ahead of key haemophilia meeting
After a patient develops inhibitors questions emerge about a new approach to treating haemophilia A.
Bayer’s gene therapy juggling act
Two arm’s-length acquisitions and two licensing deals have given Bayer a cell and gene therapy unit; now to bring it all together.
Uniqure’s safety scare shakes haemophilia gene therapy
The whole haemophilia gene therapy field will be hoping that a case of liver cancer in etranacogene dezaparvovec’s pivotal trial is not treatment related.
Recycling pays off for Mereo
The company has spent five years buying in discontinued projects, and at last has managed to sell one of these on at a nice profit.
Ovid crashes out in Angelman syndrome
The failure of OV101 puts the spotlight on Ultragenyx and Roche.
Ash 2020 preview – late-breaker puts Uniqure in pole position
Among the upcoming haematology meeting’s non-oncology presentations Uniqure’s haemophilia B gene therapy scores a valuable late-breaker.
Finding the next Vertex
Other groups with large disconnects between sellside and investor expectations include Beigene, Alnylam and Acceleron.
Filgotinib setback vindicates Abbvie’s opt-out
A shock US rejection delays approval by at least two years and makes Abbvie, which had given up rights five years ago, look smart.
