PPMD 2019 – Pfizer’s Duchenne gene therapy halt is good news for Sarepta
Worries over toxicity with Pfizer's PF-06939926 halts trial dosing and could leave the way clear for its DMD rival Sarepta.
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.
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.
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…
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.
Caladrius refuses to give up on Tregs for diabetes
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.
Higher dose points Sangamo no closer to zinc finger success
Tiny signs of a therapeutic benefit are offset by safety worries, as the company pivots to higher doses and second-generation projects.
Uniqure turns the screw on Spark and Pfizer
In the race to develop a haemophilia B gene therapy Uniqure now has the upper hand.
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