Ash 2022 – Orchard gets a Sanfilippo boost
Promising but early data with OTL-201 could see the group compete against the likes of Ultragenyx and Lysogene.
Ash 2022 preview – Argenx’s expansion plans come into focus
Vyvgart gets a prestigious press slot for its next potential use, while Sangamo’s haemophilia A gene therapy mimics Biomarin’s valrox again.
Syncona sets its sights on a gene therapy turnaround
But, with Applied Genetic Technologies Corporation, can lightning strike a third time?
Zynteglo gets the nod, and the hard work begins
Bluebird’s gene therapy is to be launched in the US priced $2.8m per patient, but can the company tread the cautious path to profitability?
Roxadustat shows the latest regulatory fault line split
Fibrogen’s controversial anaemia pill becomes the latest example of markedly different regulatory stances taken in Europe and the US.
Bluebird split looks premature
Today’s safety scare with Lentiglobin calls into question the logic behind Bluebird’s planned business separation.
