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.
The tiny German biotech needs strong signals to emerge from an approaching read out of its chemokine-targeting lead project.
The buttoned-up biotech, recently valued at $7bn, has finally unveiled plans to go public. But investor appetite for new issues has taken a knock. Does Moderna’s move…
Krystal Biotech shares have surged 53% so far this week on gene therapy data in two patients, but rivals are waiting in the wings.