Gene therapy before birth: a new hope for babies with GM1 disease
Disease control
Not yet recruiting
This early-stage trial tests giving a gene therapy to unborn babies diagnosed with GM1 gangliosidosis, a severe genetic disorder that damages the brain and body. The therapy uses a harmless virus to deliver a working copy of the missing enzyme gene. The main goal is to see if the…
Phase: PHASE1 • Sponsor: Tippi Mackenzie • Aim: Disease control
Last updated Jun 27, 2026 12:06 UTC