Another gene therapy safety scare hits
The clinical hold for Biomarin’s phenylketonuria project comes on the heels of an FDA adcom to discuss gene therapy toxicity.
AC Immune claims a tau win in Alzheimer’s
But mixed data and a previous failure provide reasons to be cautious.
MPS 2021 – Denali disappoints in Hunter syndrome
The group will need longer-term data to justify its huge valuation.
Sigilon hold hurts ahead of key haemophilia meeting
After a patient develops inhibitors questions emerge about a new approach to treating haemophilia A.
Haemophilia B delay hits Uniqure
One-year data look encouraging, but etranadez will now not be filed until 2022.
Uniqure’s safety scare shakes haemophilia gene therapy
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.
