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.
Encouraging early data have persuaded the Dutch RNA researcher to push straight into a pivotal trial of its Leber’s congenital amaurosis project, raising hopes for an…
A complete response letter for Waylivra has taken investors by surprise, and increases the pressure on Ionis before an FDA decision on Tegsedi.
The two companies disagree on the commercial potential of an mRNA flu vaccine, and Biontech just got Pfizer’s buy-in.