A slew of data on spinal muscular atrophy projects over the weekend shows that this tiny market is set for some big competition, with Novartis, Roche and Biogen jostling…
The RNAi specialist gets an $800m cash injection under a central nervous system tie-up with Regeneron.
Upcoming data from Roche/Ionis and Wave Life Sciences will be an important test of potentially disease-modifying candidates.
Alnylam looks likely to get approval for its second RNAi candidate, givosiran. But toxicity issues have tainted the Envision trial win.
Duchenne muscular dystrophy attracts a new entrant in pamrevlumab, while Alnylam awaits more pivotal data with givosiran.
After two other liver disease projects fade into the background Ionis delays the antisense asset AKCEA-ANGPTL3-LRx by a year.
Curevac’s decision to exit work on an mRNA approach to OTC deficiency follows a clinical hold on Translate Bio’s similar project, and raises broader concerns.
Tiny signs of a therapeutic benefit are offset by safety worries, as the company pivots to higher doses and second-generation projects.
Alnylam appears to have turned its Onpattro launch around, but is adamant that investors should have been more patient all along.