Sio Gene Therapies’ AXO-AAV-GM1 and Passage Bio’s PBGM01 – are due to yield data in mid-2021, and Lysogene is not too far behind with LYS-GM101. Within the next year there will be a chance to see how the projects stack up against each other in the clinic.

Sio, formerly known as Axovant, is in the lead for now. The group has already reported promising six-month data from the low-dose cohort of its phase I/II trial; notably, clinically meaningful increases have been seen in the enzyme beta-galactosidase, which is deficient in GM1 gangliosidosis.