Axovant is the latest company to benefit from excitement over gene therapy data in small numbers of patients.
Three gene therapy contenders, Abeona, Fibrocell and Krystal, are taking different approaches in the quest to cure epidermolysis bullosa.
Sage Therapeutics looks to keep up momentum with SAGE-217 in bipolar depression, while Marinus tries to catch up with an oral postpartum depression project.
Krystal Biotech shares surged after positive data with its epidermolysis bullosa project, KB103, in just two patients. Now it needs to repeat the trick.
Duchenne muscular dystrophy attracts a new entrant in pamrevlumab, while Alnylam awaits more pivotal data with givosiran.
After two other liver disease projects fade into the background Ionis delays the antisense asset AKCEA-ANGPTL3-LRx by a year.
After a trial failure last week, Caladrius Biosciences has not abandoned hope for its T regulatory cell therapy in type 1 diabetes, but funding might become a problem.
As the final cohort in the Phoenix trial reads out positively for bardoxolone the stage is set for pivotal results and a decision by Abbvie.
Promising retinitis pigmentosa results have given Reneuron a boost, but the company still faces a pivotal trial.