First-ever prenatal gene therapy trial aims to stop deadly brain disease before birth
Disease control
Not yet recruiting
This early-stage study tests a one-time gene therapy given directly to the fetus before birth for GM1 gangliosidosis, a severe genetic disorder that damages the brain and body. The treatment uses a harmless virus to deliver a working copy of the missing enzyme. The main goal is t…
Phase: PHASE1 • Sponsor: Tippi Mackenzie • Aim: Disease control
Last updated May 11, 2026 20:53 UTC