Two haemophilia A subjects help Sangamo erase 2019's losses
Promising early data with Sangamo and Pfizer’s haemophilia A gene therapy could set up a battle with Roche and Biomarin.
Upcoming events – Pfizer and Glycomimetics await sickle cell data and Biocryst tries again
Rivipansel needs to help sickle cell patients leave hospital sooner and BCX7353 faces established hereditary angioedema agents.
Go or no go? Abbvie, Glaxo and Heron await FDA verdicts
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.
How to pay for gene therapies in developing nations
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…
Imbruvica resistance provides early but fertile development ground
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.
Vertex and Crispr up the ante in gene therapy chase
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.
Roche buy-in is another vote of confidence in gene therapy
The Swiss group picks up Spark Therapeutics for $4.8bn, marking the biggest gene therapy buyout since Novartis’s $8.7bn move on Avexis.
Uniqure turns the screw on Spark and Pfizer
In the race to develop a haemophilia B gene therapy Uniqure now has the upper hand.
Go or no go? Sanofi’s Cablivi leads February’s approvals charge
Others in the FDA queue include Bausch’s psoriasis therapy Duobrii and Motif Bio’s antibiotic Iclaprim.
Kickstarting ideas
Independent, data-driven daily news and analysis on pharma, biotech and medtech.