Valrox leaves a disproportionate hole in Biomarin’s valuation, but gene therapy competitors shouldn’t celebrate just yet.
As durability continues to fog the future for Biomarin’s gene therapy, Pfizer and Sangamo keep up the pressure.
The plan to file Lentiglobin for sickle cell disease next year gets the green light, as a patient death is deemed unrelated to treatment.
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.
A bidding war has netted Sarepta the largest-ever licensing deal for a cell or gene therapy. Can Roche make it work?