New gene therapy hopes to tame rare metabolic disease in kids
Disease control
Recruiting now
This early-stage study tests a single-dose gene therapy called VGM-R02b in up to 12 children aged 6 or younger with glutaric acidemia type I, a rare genetic disorder that can cause brain damage. The treatment aims to replace the faulty gene and improve symptoms like movement prob…
Phase: PHASE1 • Sponsor: Shanghai Vitalgen BioPharma Co., Ltd. • Aim: Disease control
Last updated May 17, 2026 05:08 UTC