Krystal Biotech shares surged after positive data with its epidermolysis bullosa project, KB103, in just two patients. Now it needs to repeat the trick.
Krystal Biotech shares have surged 53% so far this week on gene therapy data in two patients, but rivals are waiting in the wings.
Early data are due for Krystal Biotech’s epidermolysis bullosa project and Uniqure’s haemophilia B candidate.
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 looming interim analysis could make Amryt a player in epidermolysis bullosa – and boost its deal-making ambitions.