Safety and Tolerability of RGX-314 (Investigational Product) Gene Therapy for Neovascular AMD Trial
Summary
Excessive vascular endothelial growth factor (VEGF) plays a key part in promoting neovascularization and edema in neovascular (wet) age-related macular degeneration (nAMD). VEGF inhibitors (anti-VEGF), including ranibizumab (LUCENTIS®, Genentech) and aflibercept (EYLEA®, Regeneron), have been shown to be safe and effective for treating nAMD and have demonstrated improvement in vision. However, anti-VEGF therapy is administered frequently via intravitreal injection and can be a significant burden to the patients. RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein. The long-term, stable delivery of this therapeutic protein following a 1 time gene therapy treatment for nAMD could potentially reduce the treatment burden of currently available therapies while maintaining vision with a favorable benefit:risk profile.
Timeline
- Start
- 2017-03-29
- Primary completion
- 2019-11-24
- Completion
- 2021-06-17
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | Surabgene Lomparvovec | Gene therapy (AAV / viral vector) | 3e+09 vg | Other |
| Subject | Surabgene Lomparvovec | Gene therapy (AAV / viral vector) | 1e+10 vg | Other |
| Subject | Surabgene Lomparvovec | Gene therapy (AAV / viral vector) | 6e+10 vg | Other |
| Subject | Surabgene Lomparvovec | Gene therapy (AAV / viral vector) | 1.6e+11 vg | Other |
| Subject | Surabgene Lomparvovec | Gene therapy (AAV / viral vector) | 2.5e+11 vg | Other |