FDA rejection of BioMarin gene therapy raises durability questions 20-Aug-2020 By Nick Taylor The US Food and Drug Administration (FDA) has rejected approval of BioMarin’s hemophilia A gene therapy amid durability doubts.
CSL Behring in $450m spend on hemophilia gene therapy 30-Jun-2020 By Ben Hargreaves The company buys the global rights to a gene therapy currently going through Phase III trials.
Novo Nordisk looks to bluebird for gene therapy targets 10-Oct-2019 By Ben Hargreaves Novo Nordisk partners with bluebird on a three-year research arrangement looking to discover gene therapy candidates for the treatment of genetic diseases, including hemophilia.
