GM1 Gangliosidosis Imagine-1 (PBGM01) Clinical Trial

Information for parents and caregivers

Imagine-1 trial key

  • Name: Imagine-1
  • Treatment: PBGM01
  • Goal: Study the effectiveness and safety of a new gene therapy, PBGM01, in children with GM1 gangliosidosis
  • Ages eligible: 1 to 24 months

Passage Bio is studying a potential new therapy called PBGM01 for the treatment of GM1 gangliosidosis

gene therapy

PBGM01 is a novel gene therapy intended to help replace the gene that doesn't work in children with GM1 gangliosidosis, called the GLB1 gene


This clinical trial, called Imagine-1, is studying if PBGM01 can help children with Early Onset Infantile and Late Onset Infantile GM1 gangliosidosis


Children with GM1 gangliosidosis who are aged 1 to 24 months will be included in this study

Contact Passage Bio to learn more:

We are here to help. We’re happy to answer questions you have about GM1 gangliosidosis, the treatment we are studying, and the clinical trial.

Please fill out the form to the right below to contact Passage Bio. If you are interested in trial participation, please include the information listed below:

  • Date of birth of child (month/date/year)
  • Diagnosis of child
  • Language preference