After a patient develops inhibitors questions emerge about a new approach to treating haemophilia A.
One-year data look encouraging, but etranadez will now not be filed until 2022.
A second clinical failure means that Biogen threw away $877m, but this sum will today be seen as little more than a rounding error.
A serious adverse event puts ADVM-022’s future in doubt, and it could hurt other eye disease gene therapy players too.
The failure of Roche and Ionis’s late-stage candidate tominersen casts doubt on Wave’s approach, too.
Better-looking safety might not be enough to get Solid back in the Duchenne muscular dystrophy game.