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.
Arbutus Biopharma already looked second best to Arrowhead Pharmaceuticals in the race to develop a functional cure for hepatitis B. New data have made the former’s task…
As data on Amicus Therapeutics' clinical-stage Pompe disease candidate roll in, the group locks up preclinical gene therapy assets.
The sellside’s protestations aside, results at the World Muscle meeting position risdiplam and AVXS-101 as serious challengers to Spinraza in spinal muscular atrophy.
After buying Avexis for $8.7bn to access a potentially transformative treatment for spinal muscular atrophy, data approaches from Novartis.
A genome editing tie-up between Gilead and Precision Biosciences, and promising early data with Arrowhead’s RNA interference project, are two novel approaches in hep B.
Nightstar hopes that early gene therapy data will give it a spark, while Alnylam awaits pivotal results for one of its biggest pipeline prospects, givosiran.