Development has ended for BIIB105 in ALS after it failed to clear a crucial biomarker and show clinical benefit. Also, Biogen opted not to advance ION582 for Angelman syndrome, so Ionis will go it alone.

Supporting ‘n of 1’ gene therapy development, the playbook from a Foundation for the National Institutes of Health-backed consortium aims to help investigators who are not traditional product sponsors prepare for meetings with US FDA and IND submissions.

Ultragenyx’s antisense oligonucleotide for treating Angelman syndrome and Sangamo’s gene therapy for Fabry disease are the latest products to make it onto the European Medicines Agency’s priority medicines scheme.

From extricating Daiichi Sankyo from the Ranbaxy acquisition to enabling the rapid development of blockbuster Enhertu, George Nakayama has had a winding career path through multiple key strategic positions and decisions. He shares his experience and insights, and a good dose of humor, in this exclusive interview.