Sio Gene accepts reality

Sio Gene Therapies, the biotech famously once known as Axovant, has not had a happy time. Last October it revealed a promising early clinical update from its GM1 gangliosidosis gene therapy, but since then its valuation has halved. With the group now trading at cash drastic action was needed, and yesterday came the defenestration of its chief executive, cancelling of a Parkinson’s disease deal with Oxford Biomedica, and tightened focus on the gangliosidosis projects AXO-AAV-GM1 and AXO-AAV-GM2. At the end of 2021 Sio had $82m of cash, which will now last beyond mid-2023. The Oxford deal, covering AXO-Lenti-PD, had been signed in 2018 when the then-Axovant was worth nearly 20 times more than now and aimed to become a “world-leading innovative neurology company”. With Parkinson’s becoming a competitive area it makes sense to leave the heavy lifting to bigger players, and GM1 and GM2 gangliosidosis look like niches better suited for Sio. AXO-Lenti-PD’s dose-escalation Sunrise-PD trial was to resume this year, but the sham-controlled Explore-PD never got under way in 2021 as planned, and Oxford now needs to find another partner to take it on. The project’s earlier iteration, Prosavin, was canned after generating weak efficacy.

Selected gangliosidosis gene therapies in the clinic
Project Company Description Study
GM1 gangliosidosis
AXO-AAV-GM1 Sio Gene Therapies AAV9 vector; IV delivery NCT03952637
PBGM01  Passage Bio AAVhu68 vector; ICM delivery Imagine-1
LYS-GM101 Lysogene AAVrh10 vector; ICM delivery NCT04273269
GM2 gangliosidosis (Tay-Sachs disease, AB variant & Sandhoff disease)
TSHA-101 Taysha Gene Therapies AAV9 vector; intrathecal delivery NCT04798235
AXO-AAV-GM2 Sio Gene Therapies AAVrh.8 vector; ICM/intrathecal delivery NCT04669535
Note: ICM=intra-cisterna magna. Source: Evaluate Pharma &

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