Status: Not yet recruiting
Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial
This is a study for the administration of in utero AAV9 transfer in prenatally diagnosed Type I or Type II GM1.
Participants needed: 5
Trial details
Phase: Phase 1Biological sex: AllType: InterventionalSponsor: Tippi MackenzieUpdated: Apr 13, 2026
Eligibility criteria
In the event that parents are identified as genetic carriers of Type I or Type I... [+3]
Esophageal or bowel atresia [+18]