Despite concerns about a missing patient, results from a tiny trial in epidermolysis bullosa boosted Krystal Biotech’s stock by 41%.
The cystic fibrosis player does deals with Crispr and Exonics to launch itself into a highly competitive rare disease area.
Protagonist Therapeutics and Pfizer will soon get an idea of their respective chances in beta-thalassaemia and Duchenne muscular dystrophy.
Three-year data from Biomarin’s haemophilia A gene therapy fail to quash concerns about durability, but the company insists that the results are good enough to file on.
The US FDA granted seven approvals in two days last week, including an eagerly awaited thumbs-up for Novartis’s gene therapy Zolgensma.
Expectations are low for a three-year update on the group’s haemophilia A gene therapy, and Biomarin’s rivals could capitalise on any slip-up.
As Meiragtx wows investors with its gene therapy for a cause of blindness, continuing lack of clarity around Gensight’s GS010 prompts more questions than answers.
Its gene therapy is three years away from market, but Mustang is already thinking about outcomes-based pricing.
A slew of data on spinal muscular atrophy projects over the weekend shows that this tiny market is set for some big competition, with Novartis, Roche and Biogen jostling…