Spotlight – Cystic fibrosis developers take a deep breath
The next couple of years should show whether inhaled genetic projects have potential.
Abeona takes on Amryt – and more importantly, Krystal
Three new epidermolysis bullosa therapies are approaching the US, and expectations vary wildly.
Syncona sets its sights on a gene therapy turnaround
But, with Applied Genetic Technologies Corporation, can lightning strike a third time?
Krystal shines in epidermolysis bullosa
Look out Amryt: Krystal’s topical gene therapy is gaining ground.
Centessa claims a place in the antitrypsin deficiency pipeline
But investors will need more convincing; and others are more advanced, with Arrowhead and Inhibrx ones to watch.
Boehringer takes a different approach in cystic fibrosis gene therapy
Deals this week with a UK consortium and Oxford Biomedica see the group bet on lentiviral vectors.
Biotech’s key fourth-quarter data
Deciphera, Allakos and Uniqure are all set for important data disclosures.
Epidermolysis bullosa gene therapies wait in the wings
Amryt marches ahead, but gene therapies from Krystal, Abeona and Castle Creek are heading towards data readouts.