Despite concerns about a missing patient, results from a tiny trial in epidermolysis bullosa boosted Krystal Biotech’s stock by 41%.
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.
Digging into the sales forecasts for gene therapies shows just how low their penetration is expected to be. Could biopharma come to regret its high-price, low-volume…
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.
A looming interim analysis could make Amryt a player in epidermolysis bullosa – and boost its deal-making ambitions.