After a patient develops inhibitors questions emerge about a new approach to treating haemophilia A.
Worries about a stricter US FDA come to nothing, as last year’s approval total beats 2019’s.
Waning factor VIII levels mean the durability question is still unanswered.
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 2021 will see rare diseases dominate for biotech, with Sarepta revealing key data on its gene therapy play.
And bleeds seen with the “transformative” project were played down.