Shanghai Vitalgen Biopharma Co., Ltd.

This phase 3 study tests a gene therapy called VGR-R01 for people with Bietti Crystalline Dystrophy, a rare inherited eye disease that causes vision loss. The trial will enroll 45 adults to see if the treatment improves eyesight compared to no treatment. The goal is to control th…

This trial tests a gene therapy called VGN-R09b in 13 children with severe AADC deficiency, a rare genetic disorder that causes severe movement problems. The therapy is injected directly into a brain region to help restore missing enzyme activity. The study checks safety and whet…

This early-stage study tests a gene therapy called VGR-R01 in 12 adults with Bietti Crystalline Dystrophy, a rare inherited eye disease that causes vision loss. The main goal is to check if the treatment is safe and tolerable. Researchers will monitor side effects and changes in …

This early-stage study tests a gene therapy called VGN-R09b in 39 people with Parkinson's disease whose motor symptoms are not well controlled by current medications. The main goal is to check safety and tolerability, while also measuring changes in movement and daily function. T…