Deals this week with a UK consortium and Oxford Biomedica see the group bet on lentiviral vectors.
The failure of the phase 2 Moonsong outpatient trial prompts a phase 3 rethink and a one-year delay.
After the Valor defeat, the group and its partner Biogen will hope that going earlier could improve the amyotrophic lateral sclerosis project’s chances.
The group’s ambitions are startling, but it will need cash and collaborators to realise them.
Private drug developers have already raised more money this year than in 2020.
But questions about gene therapy safety could benefit the enzyme-replacement therapy incumbents.
Two opportunistic buys today, of Adamas and Flexion, included a contingent value rights component.