**Gaucher Disease Gene Therapy Trial**
This study is for adults with Gaucher disease Type 1, a condition affecting how your body breaks down a certain fatty substance. Currently, patients in the study must have been on regular enzyme replacement therapy (ERT) or substrate reduction therapy (SRT) for at least 2 years. The study will test a new treatment called FLT201 to see if it's safe and works well when ERT/SRT is stopped.
Key Points:
- The study is a Phase 3 trial, meaning it's in the later stages of research and involves multiple locations.
- Participants must be at least 18 years old, have Gaucher disease Type 1, and have stable blood levels.
- Those who have had certain transplants, or who are pregnant, are not eligible.
This study aims to provide a new treatment option for Gaucher disease Type 1 through gene therapy, which involves using genes to treat or prevent disease. Participants will help researchers understand if FLT201 can replace current therapies safely and effectively.