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.
Ash 2020 preview – late-breaker puts Uniqure in pole position
Among the upcoming haematology meeting’s non-oncology presentations Uniqure’s haemophilia B gene therapy scores a valuable late-breaker.
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.
No buyout for Uniqure
But a licensing deal with CSL could have underappreciated benefits, even if expectations were for something much bigger.
All still to play for in haemophilia gene therapy race
As durability continues to fog the future for Biomarin’s gene therapy, Pfizer and Sangamo keep up the pressure.
