Update (October 2026): Lysogene, Sio Gene Therapies (formerly Axovant) and Passage Bio no longer exist. Lysogene’s GM1 program ended. The Sio trial continues as an investigator-led study at the NIH, and PBGM01 is now sponsored by Gemma Biotherapeutics / RareTx and is closed to new enrollment. For the trials open today, see our GM1 clinical trials guide.
Cure GM1 is pleased to share the tremendous news that both Passage Bio and Lysogene have received regulatory approvals to initiate AAV gene therapy clinical trials for infantile and late infantile GM1 gangliosidosis in 2021! Please note that recruitment and enrollment have not yet begun.
Read the Press Releases
- 1/11/21 Lysogene Receives MHRA and Research Ethics Committee Approvals to Initiate the Gene Therapy Clinical Trial in the UK with LYS-GM101 for the Treatment of GM1 Gangliosidosis
- 1/4/20 Passage Bio Receives FDA Clearance of IND Application for Lead Gene Therapy Candidate PBGM01 for Treatment of Infantile GM1 Gangliosidosis
- 12/10/20 Passage Bio Receives MHRA Clinical Trial Authorization for PBGM01 for Treatment of GM1 Gangliosidosis
Read more on clinicaltrials.gov about the Lysogene trial NCT04273269. Cure GM1’s web site is also up to date with all the current clinical trials and natural history studies.
The Sio Gene Therapies intravenous AAV9 trial continues at the National Institutes of Health for Type 2 and Type 1.