Krystal plays down dropout to claim a mid-stage win
Despite concerns about a missing patient, results from a tiny trial in epidermolysis bullosa boosted Krystal Biotech’s stock by 41%.
Vertex’s second transformation: Duchenne time
The cystic fibrosis player does deals with Crispr and Exonics to launch itself into a highly competitive rare disease area.
Upcoming events – Protagonist and Pfizer target rare diseases
Protagonist Therapeutics and Pfizer will soon get an idea of their respective chances in beta-thalassaemia and Duchenne muscular dystrophy.
Biomarin suffers minimal bleeding on valrox readout
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.
Zolgensma heads up spate of US approvals
The US FDA granted seven approvals in two days last week, including an eagerly awaited thumbs-up for Novartis’s gene therapy Zolgensma.
Biomarin hopes long-term valrox data can stem the bleeding
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.
A tale of two eyes as Meiragtx sees a clearer path forward than Gensight
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.
Mustang Bio sets its sights on 2021 approval
Its gene therapy is three years away from market, but Mustang is already thinking about outcomes-based pricing.
AAN 2019 – Roche confirms its place in spinal muscular atrophy
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…
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