4D gets a good start in Fabry
But a serious adverse event and waning enzyme levels raise questions about the data.
Boehringer takes a different approach in cystic fibrosis gene therapy
Deals this week with a UK consortium and Oxford Biomedica see the group bet on lentiviral vectors.
Idorsia’s Fabry flop puts focus on gene therapies
But questions about gene therapy safety could benefit the enzyme-replacement therapy incumbents.
Roche takes the Tim-3 battle to Novartis
The Swiss group has likely been paying attention to industry developments as BCMA and CD40 go out, while Covid-19 and Tim-3 move up.
Clearside’s long-acting eye project shows promise
But data are early, and other groups are also developing less frequent injections for wet AMD.
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.
