Shanghai Vitalgen Biopharma Co., Ltd.

This early-stage study tests a gene therapy called VGN-R08b in 17 people with Parkinson's disease who have a specific genetic change (GBA1 mutation). The treatment is given as an injection into the fluid around the brain. The main goals are to check safety and see if it can impro…

This early-stage study tests a new gene therapy called VGN-R08b, given as a single injection into the brain fluid, for children aged 2 to 17 with Type III Gaucher's disease. The goal is to see if it is safe and can improve enzyme activity related to the disease. Only 12 participa…

This study tests a gene therapy called VGR-R01 in the second eye of 15 people with Bietti's crystalline dystrophy, a rare inherited eye disease that causes vision loss. Participants already received the therapy in one eye in a previous study. The main goal is to check safety, and…