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

Read more on about the Lysogene trial here.  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.