Bluebird bio has its first approval for Skysona (formerly Lenti-D), its gene therapy for children with the rare disease cerebral adrenoleukodystrophy (CALD), after getting the nod from the
The UK's competition regulator has approved AstraZeneca's $39 billion acquisition of US-based drugmaker Alexion, without opting for a broader inquiry into the deal.
Diabetes giant Novo Nordisk has opened a new front in its rare disease R&D, thanks to an acquisition agreement with Prothena focusing on drugs for ATTR amyloidosis.
Orchard Therapeutics' gene therapy for rare childhood disease metachromatic leukodystrophy (MLD) Libmeldy has been rejected for NHS use by NICE in draft guidance.
bluebird bio's sickle cell disease (CD) gene therapy LentiGlobin is the latest recipient of an 'innovation passport' introduced in the UK earlier this year to speed up NHS access to promisi
The first ever clinical data with a CRISPR/Cas9 drug used to edit the genomes of cells within the body has yielded impressive results in patients with ATTR amyloidosis, a life-threatening r