Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
RGX-314 · 1 trial · 2 indications
Participants with ocular and non-ocular AEs and SAEs through 26 weeks (24 weeks following RGX-314 administration)
| Arm | Type | Description |
|---|---|---|
| Cohort 1 | EXPERIMENTAL | 3E9 GC (genome copies)/eye of RGX-314 (E means the exponential constant) |
| Cohort 2 | EXPERIMENTAL | 1E10 GC/eye of RGX-314 |
| Cohort 3 | EXPERIMENTAL | 6E10 GC/eye of RGX-314 |
| Cohort 4 | EXPERIMENTAL | 1.6E11 GC/eye of RGX-314 |
| Cohort 5 | EXPERIMENTAL | 2.5E11 GC/eye of RGX-314 |
| Name | Type | Description |
|---|---|---|
| RGX-314 | GENETIC | RGX-314 is a recombinant adeno-associated virus (AAV) gene therapy vector carrying a coding sequence for a soluble anti-VEGF protein |
Inclusion Criteria: 1. Patients ≥ 50 and ≤ 89 years with a diagnosis of subfoveal CNV (Choroidal neovascularization) secondary to AMD in the study eye receiving prior intravitreal anti-VEGF therapy. 2. BCVA (Best Corrected Visual Acuity) between ≤20/63 and ≥20/400 (≤63 and ≥19 Early Treatment Diabe...
RGX-314 is an investigational gene therapy being developed for the treatment of neovascular age-related macular degeneration (wet AMD). It is designed to address the underlying cause of the disease by delivering a gene that encodes for an anti-angiogenic protein, potentially reducing the need for frequent eye injections.
RGX-314 targets the vascular endothelial growth factor (VEGF) pathway. It is designed to produce a protein that inhibits VEGF, thereby reducing abnormal blood vessel growth and leakage in the retina, which are hallmarks of neovascular age-related macular degeneration.
RGX-314 is being developed by REGENXBIO Inc., a biotechnology company focused on gene therapy. REGENXBIO is publicly traded on the Nasdaq under the ticker symbol RGNX.
RGX-314 is in Phase 1 clinical development. It is an investigational drug and has not been approved by the FDA. A Phase 1 trial has been completed to evaluate its safety and tolerability in patients with neovascular age-related macular degeneration.
RGX-314 has been studied in one completed Phase 1 clinical trial, identified as NCT03066258. This trial evaluated the safety and tolerability of RGX-314 in 42 participants with neovascular age-related macular degeneration in the United States.
RGX-314 is the investigational name for this gene therapy product. No alternative names have been reported for this drug in the available information.