Roche's Mirros study should prove idasanutlin's worth on top of chemotherapy, while Proqr hopes to Usher in a rare disease cure.
Promising data with Fibrocell’s epidermolysis bullosa gene therapy send the company down as funding worries kick in.
Three gene therapy contenders, Abeona, Fibrocell and Krystal, are taking different approaches in the quest to cure epidermolysis bullosa.
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.