Drugs / VG801
Trials 1
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 1/2 | NCT07002398 | Dec 2024 → May 2026 overdue | ABCA4-related retinopathy, Stargardt disease | VeonGen Therapeutics GmbH | Recruiting | No outcome recorded |
News releases announcing trial results or a regulatory action · 4
| Date | Issuer | Release |
|---|---|---|
| 2026-03-25 | VeonGen Therapeutics GmbH | Results VeonGen Announces Clinical Progress of VG801 Gene Therapy in Stargardt Disease, with an Oral Presentation at ARVO 2026 veongen.com ↗
Preliminary efficacy data show consistent functional improvements in best-corrected visual acuity (BCVA) and a newly developed Virtual Reality Visual Test (VRVT), with improvements sustained through six- and twelve-month follow-up. |
| 2025-08-21 | VeonGen Therapeutics GmbH | Regulatory VeonGen Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for VG801 Gene Therapy for Stargardt Disease veongen.com ↗
VeonGen Therapeutics, a clinical-stage genetic medicine company developing next-generation gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to VG801, its lead investigational gene therapy for Stargardt disease and other ABCA4 mutation–associated retinal dystrophies. |
| 2025-06-05 | VeonGen Therapeutics GmbH | Regulatory ViGeneron Rebrands as VeonGen Therapeutics and Announces FDA Rare Pediatric Disease Designation and Clinical Progress for Lead Gene Therapy VG801 in Stargardt Disease veongen.com ↗ |
| 2024-12-18 | VeonGen Therapeutics GmbH | Regulatory ViGeneron Announces FDA Clearance of IND for Novel mRNA Trans-Splicing Gene Therapy VG801 to Treat Stargardt Disease and Other ABCA4-Linked Retinal Dystrophies veongen.com ↗
VG801 is the first FDA IND clearance for novel mRNA trans-splicing gene therapy, delivering the full-length functional ABCA4 gene to target the genetic root cause of retinal dystrophies associated with ABCA4 mutations |
All press releases naming this drug 4 releases
| Date | Issuer | Release |
|---|---|---|
| 2026-03-25 | VeonGen Therapeutics GmbH | VeonGen Announces Clinical Progress of VG801 Gene Therapy in Stargardt Disease, with an Oral Presentation at ARVO 2026 veongen.com ↗ |
| 2025-08-21 | VeonGen Therapeutics GmbH | VeonGen Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for VG801 Gene Therapy for Stargardt Disease veongen.com ↗ |
| 2025-06-05 | VeonGen Therapeutics GmbH | ViGeneron Rebrands as VeonGen Therapeutics and Announces FDA Rare Pediatric Disease Designation and Clinical Progress for Lead Gene Therapy VG801 in Stargardt Disease veongen.com ↗ |
| 2024-12-18 | VeonGen Therapeutics GmbH | ViGeneron Announces FDA Clearance of IND for Novel mRNA Trans-Splicing Gene Therapy VG801 to Treat Stargardt Disease and Other ABCA4-Linked Retinal Dystrophies veongen.com ↗ |
Evidence & citations 1 cited value
Every value below carries the sentence it was read from. 1 source stands behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | VG801 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT07002398 ↗ |