Promising early data with Sangamo and Pfizer’s haemophilia A gene therapy could set up a battle with Roche and Biomarin.
Rivipansel needs to help sickle cell patients leave hospital sooner and BCX7353 faces established hereditary angioedema agents.
Notable US regulatory decisions due in April feature a major pipeline hope from Abbvie, a non-opioid pain medication from Heron and Glaxo’s two-pill HIV regimen.
With rich countries like the US finding it hard to fund gene therapies, it is worth asking whether these projects will ever reach patients in developing countries. And if…
Targeting a mechanism of resistance to first-generation BTK inhibitors has caught the attention of biotechs like Arqule, which claimed a major success last week.
The first sickle cell and beta thalassaemia patients have been enrolled into a Crispr/Cas9 trial, heating up a race with Bluebird, Sanofi, Sangamo and Editas.
The Swiss group picks up Spark Therapeutics for $4.8bn, marking the biggest gene therapy buyout since Novartis’s $8.7bn move on Avexis.
In the race to develop a haemophilia B gene therapy Uniqure now has the upper hand.
Others in the FDA queue include Bausch’s psoriasis therapy Duobrii and Motif Bio’s antibiotic Iclaprim.