Novo Nordisk took an early step towards genome editing with yesterday’s Bluebird tie-up, and isn’t ruling out a move into conventional gene therapies.
Orphan status for its haemophilia cell therapy and buy-in from Lilly on its diabetes project are good signs for Sigilon, but research is still very early.
Sangamo is slowly building a case for SB-525 over Biomarin’s valrox.
Worries over toxicity with Pfizer's PF-06939926 halts trial dosing and could leave the way clear for its DMD rival Sarepta.
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.
Promising early data with Sangamo and Pfizer’s haemophilia A gene therapy could set up a battle with Roche and Biomarin.
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…
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.