Jury still out on Sarepta’s next generation
The group’s lead next-gen exon skipper beats a low bar set by Exondys, but adverse events loom large.
Dangling PD-1s emerge strong from three-day grilling
A US advisory panel has largely endorsed three anti-PD-(L)1 drugs, with voting apparently driven by the availability of alternatives.
Go or no go? Apellis and Heron head for the finish line
May will see US FDA decisions for Apellis, Heron and Bristol Myers Squibb, while Provention Bio is set for a panel meeting.
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.
Epidermolysis bullosa gene therapies wait in the wings
Amryt marches ahead, but gene therapies from Krystal, Abeona and Castle Creek are heading towards data readouts.
