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.
Ash 2020 – durability worries hit Sangamo and Pfizer’s haemophilia A gene therapy
Waning factor VIII levels with giroctocogene fitelparvovec once again raise the spectre of Biomarin’s valrox.
Finding the next Vertex
Other groups with large disconnects between sellside and investor expectations include Beigene, Alnylam and Acceleron.
Has 2020 been a big year for regulatory knockbacks?
An analysis of FDA complete response letters does not reveal a big uptick – but the year is not over yet.
Are the stars waning for Pfizer’s haemophilia A gene therapy?
Could Pfizer/Sangamo’s project be facing the same durability issues as Biomarin’s Roctavian?
Is valrox dead?
Valrox leaves a disproportionate hole in Biomarin’s valuation, but gene therapy competitors shouldn’t celebrate just yet.
