Is valrox dead?
Valrox leaves a disproportionate hole in Biomarin’s valuation, but gene therapy competitors shouldn’t celebrate just yet.
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.
EHA 2020 – Bluebird’s sickle cell plan takes shape, with a stark reminder
The plan to file Lentiglobin for sickle cell disease next year gets the green light, as a patient death is deemed unrelated to treatment.
Upcoming events – Avadel takes on Jazz and Axovant awaits early results
Data will soon show whether Avadel’s FT218 is more convenient than Jazz’s Xyrem, while Axovant will be looking for a dose response with its Parkinson’s gene therapy.
Sarepta makes history with Roche deal
A bidding war has netted Sarepta the largest-ever licensing deal for a cell or gene therapy. Can Roche make it work?
