Shanghai Vitalgen Biopharma Co., Ltd.

This trial tests a gene therapy called VGN-R09b for children with severe AADC deficiency, a rare genetic disorder that affects movement and brain function. The therapy is injected directly into the brain. The study aims to see if it is safe and helps children achieve motor milest…

This study tests a new gene therapy called VGN-R09b in 39 people with Parkinson's disease. The goal is to see if it is safe and can help control motor symptoms like tremors and stiffness. Participants must be between 40 and 75 years old and have at least 2.5 hours of poor movemen…

This Phase 3 trial tests a gene therapy called VGR-R01 for Bietti crystalline dystrophy, a rare inherited condition that leads to progressive vision loss. The study will enroll 45 adults who receive either the gene therapy injection under the retina or a control treatment. Resear…

This early-stage trial tests a gene therapy called VGR-R01 for Bietti Crystalline Dystrophy, a rare inherited eye disease that leads to progressive vision loss. The study involves 12 adults who receive the therapy via injection into the eye. The main goal is to check safety and t…