Instead of tinkering with early-stage genome editing deals like this week's agreement with Bluebird, Novo Nordisk should make a decisive move in haemophilia.
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.
Tocagen’s Toca-5 trial has survived two interim reviews, and faces final readout by the end of this year.
High-profile departures at Avexis raise more questions about Novartis’s acquisition, but there were plenty of red flags before the deal was sealed.
Revelations of data manipulation might not be fatal, but they will hurt the chief exec’s attempted rebranding of Novartis as a more ethical pharma company.
Competition is looming from Novartis and Roche but the latest results from Spinraza show that Biogen still has a strong case to make for the SMA therapy.
Worries over toxicity with Pfizer's PF-06939926 halts trial dosing and could leave the way clear for its DMD rival Sarepta.
Despite concerns about a missing patient, results from a tiny trial in epidermolysis bullosa boosted Krystal Biotech’s stock by 41%.
The cystic fibrosis player does deals with Crispr and Exonics to launch itself into a highly competitive rare disease area.