Freeline puts the brakes on haemophilia B

Freeline Therapeutics had been looking like an also-ran for some time in the race to get a haemophilia B gene therapy project to market. Yesterday the group finally faced up to reality, saying during its third-quarter results that it would stop investment in FLT180a, leaving the project stalled unless a partner can be found. This looks pretty unlikely: the latest data drop for FLT180a, from the dose-confirmation B-Lieve trial, raised questions about durability. And after numerous delays, the project is well behind the gene therapy leaders in a relatively small niche. Uniqure and CSL are expecting an FDA decision soon on etranacogene dezaparvovec; an EU verdict is likely in the first quarter of next year. Meanwhile, Pfizer is set to report pivotal data on fidanacogene elaparvovec next year – that project originated at Spark, which was acquired by Roche. Still, the path for that asset has not been smooth, with Pfizer earlier scrapping an interim analysis, which had delayed this study’s readout. For Freeline, the focus now shifts to its Fabry disease project, FLT190 – which is hardly a shoo-in – and an earlier-stage asset for Gaucher disease, FLT201.