Sigilon hold hurts ahead of key haemophilia meeting
After a patient develops inhibitors questions emerge about a new approach to treating haemophilia A.
2020 drug approvals rise despite Covid-19
Worries about a stricter US FDA come to nothing, as last year’s approval total beats 2019’s.
Pivotal valrox data give Biomarin déjà vu
Waning factor VIII levels mean the durability question is still unanswered.
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.
Key upcoming clinical catalysts for biotech
Early 2021 will see rare diseases dominate for biotech, with Sarepta revealing key data on its gene therapy play.
Ash 2020 – hints of durability emerge for Uniqure's gene therapy
And bleeds seen with the “transformative” project were played down.
