Biogen Inc. has made a lot of early initiatives in gene therapy but nothing concrete has come out of the effort. The most notable setback has been a delay getting a gene therapy for spinal muscular atrophy (SMA) into the clinic, while rival Novartis AG has gotten its SMA gene therapy AVXS-101 all the way to the FDA. Novartis acquired its gene therapy with the $8.7bn acquisition of AveXis Inc. last April, a deal some Biogen investors wished at the time that the big biotech had done instead, since AVXS-101 will be a threat to Biogen's own Spinraza (nusinersin). (Also see "Biogen's SMA Ambitions Run Up Against A New Deep-Pocketed Rival" - Scrip, 9 April, 2018.)
Biogen had no update on the gene therapy program for investors at the J.P. Morgan conference, noting only that it...