Shanghai Vitalgen Biopharma Co., Ltd.

This early-stage trial tests a new gene therapy called VGN-R08b for children with type III Gaucher disease, a rare genetic disorder affecting the brain and body. The treatment is given as a single injection into the brain's fluid spaces. The study will monitor safety and measure …

This study is for people with Bietti's crystalline dystrophy, a rare genetic eye disease that causes vision loss. They have already received the gene therapy VGR-R01 in one eye. Now, researchers want to test if giving the same treatment to the other eye is safe and can improve vi…

This early-phase trial tests a gene therapy called VGN-R08b in 17 people with Parkinson's disease who have a GBA1 gene mutation. The therapy is injected directly into the brain's fluid spaces to try to improve symptoms. The main goals are to check safety and see if it works.