Sigilon hold hurts ahead of key haemophilia meeting
After a patient develops inhibitors questions emerge about a new approach to treating haemophilia A.
Haemophilia B delay hits Uniqure
One-year data look encouraging, but etranadez will now not be filed until 2022.
Nightstar becomes nightmare, but Biogen has bigger fish to fry
A second clinical failure means that Biogen threw away $877m, but this sum will today be seen as little more than a rounding error.
Adverum halt raises more gene therapy questions
A serious adverse event puts ADVM-022’s future in doubt, and it could hurt other eye disease gene therapy players too.
The Huntington’s pipeline takes a blow
The failure of Roche and Ionis’s late-stage candidate tominersen casts doubt on Wave’s approach, too.
Gene therapy data shake Solid
Better-looking safety might not be enough to get Solid back in the Duchenne muscular dystrophy game.
