One-year data look encouraging, but etranadez will now not be filed until 2022.
The whole haemophilia gene therapy field will be hoping that a case of liver cancer in etranacogene dezaparvovec’s pivotal trial is not treatment related.
Early data show that Freeline’s project can get factor IX levels into the normal range, but the group is still looking for the “Goldilocks” zone.
But a licensing deal with CSL could have underappreciated benefits, even if expectations were for something much bigger.
Despite the recent flurry of gene therapy deals there is still a fairly extensive list of affordable companies out there.
Syncona might have hoped for a bigger payday with the prostate imaging specialist, but it has plenty more irons in the fire.
In the race to develop a haemophilia B gene therapy Uniqure now has the upper hand.