Evaluate Vantage's coverage of the virtual Ash 2020 congress.
And bleeds seen with the “transformative” project were played down.
Waning factor VIII levels with giroctocogene fitelparvovec once again raise the spectre of Biomarin’s valrox.
Among the upcoming haematology meeting’s non-oncology presentations Uniqure’s haemophilia B gene therapy scores a valuable late-breaker.
Could Pfizer/Sangamo’s project be facing the same durability issues as Biomarin’s Roctavian?
Valrox leaves a disproportionate hole in Biomarin’s valuation, but gene therapy competitors shouldn’t celebrate just yet.
But a licensing deal with CSL could have underappreciated benefits, even if expectations were for something much bigger.
As durability continues to fog the future for Biomarin’s gene therapy, Pfizer and Sangamo keep up the pressure.