Bluebird Confident Beti-Cel Will Fare Better In US Than Europe
The company built on its case for FDA approval with new beta-thalassemia data at ASH, but investor jitters linger after the pullout of Zynteglo from Europe amid reimbursement woes for the gene therapy.
You may also be interested in...
The French non-profit, whose IP is incorporated in Zolgensma, is taking its Crigler-Najjar gene therapy into a pivotal study on its own for now. It hopes to be ready for an EU filing in two years.
US FDA’s user fee goal calendar for 2022 already contains 45 applications for novel agents featuring a wide array of biologics, from cell therapy in cancer and hematology to bispecifics and new immuno-oncology targets that could move the field beyond PD-1/L1.
In a gloomy start to the year for the biotech sector, Vertex’s strong showing in cystic fibrosis and promising pipeline could lift the mood.