News|Articles|September 4, 2025
VeonGen Snags FDA RMAT Designation for Stargardt Gene Therapy VG801
Author(s)Noah Stansfield
VG801 is intended to provide a functional copy of ABCA4 with the use of the company’s proprietary vgRNA REVeRT platform.
Advertisement
VeonGen Therapeutics has received regenerative medicine advanced therapy (RMAT) designation from the FDA for VG801, an investigational dual adeno-associated virus (AAV) vector-based gene therapy intended to treat Stargardt disease and other retinal dystrophies associated with ABCA4 mutations.
VG801 is intended to provide a functional copy of ABCA4 with the use of the company’s proprietary vgRNA REVeRT platform, and thus is expected to be able to treat Stargardt cases and other retinal dystrophies caused by any mutation in that gene. It utilizes an engineered capsid referred to as vgAAV that is meant to deliver the therapy to photoreceptor cells efficiently.
Notably, VG801 previously received FDA rare pediatric disease and orphan drug designations and the agency selected VeonGen to help develop a new functional end point for Stargardt via its Rare Disease Endpoint Advancement (RDEA) pilot program. The therapy is currently being evaluated in an open-label, nonrandomized phase 1/2 clinical trial (NCT07002398), which launched on December 23, 2024, and is expected to enroll an estimated 15 patients in total.
“Receiving RMAT designation is strong recognition of VG801’s therapeutic potential for Stargardt disease, the most common inherited retinal disorder with no approved therapies,” Caroline Man Xu, PhD, the cofounder and chief executive officer of VeonGen Therapeutics, said in a statement.1 “This recognition highlights the promise of our novel vgRNA REVeRT and vgAAV platforms and provides an opportunity to accelerate VG801’s development and deliver a much-needed therapy to patients as quickly and efficiently as possible.”
VeonGen is also developing VG901, an investigational AAV vector-based gene therapy being evaluated for the treatment of retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene in a phase 1b clinical trial (NCT06291935).2 Notably, in June 2025, at the time the company received the rare pediatric disease designation for VG801, it announced that it was rebranding from its previous name “ViGeneron” to its current name, VeonGen Therapeutics.
“This rebranding reflects our journey – from a platform innovator to a clinical -stage company with 2 gene therapies in the clinic,” Xu said in a statement at the time.1 “With VG801 and VG901 progressing in clinical trials and our platforms demonstrating robust translational potential, we are well positioned to expand the frontier of genetic medicine in ophthalmology and beyond. The FDA rare pediatric disease designation for VG801 not only highlights the strength of our scientific approach but also reinforces our focus on accelerating the development and delivery of transformative therapies for patients in urgent need.”
VG901 previously received rare pediatric disease designation from the FDA in January 2025 .3 In addition to the new designation, the company also announced that the data safety monitoring board (DSMB) for the phase 1b trial evaluating VG901 had given the green light for dose escalation. VG901 previously received orphan drug designation from the FDA in April 2024, around the same time that the first patient in the trial was dosed.4
“The DSMB has unanimously recommended proceeding with dose escalation in the ongoing VG901 phase 1b clinical trial,” DSMB chair Bart P. Leroy, MD, PhD, the head of the Department of Ophthalmology at the Center for Medical Genetics at Ghent University Hospital, said in a January 2025 statement.3 “No dose-limiting adverse events related to VG901 have been reported in the first-dose cohort to date. This marks a critical step toward advancing to the higher dose and represents an important milestone in its clinical development.”
REFERENCES
1. VeonGen Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for VG801 Gene Therapy for Stargardt Disease. News release. VeonGen Therapeutics. August 21, 2025. Accessed September 4, 2025. https://veongen.com/press/veongen-therapeutics-receives-fda-regenerative-medicine-advanced-therapy-rmat-designation-for-vg801-gene-therapy-for-stargardt-disease/
2.Vigeneron rebrands as veongen therapeutics and announces FDA rare pediatric disease designation and clinical progress for lead gene therapy VG801 in stargardt disease. News release. VeonGen Therapeutics. June, 2025. Accessed September 4, 2025. https://veongen.com/press/vigeneron-announces-fda-rare-pediatric-disease-designation-for-vg901-and-dsmb-approval-to-advance-dose-escalation-in-phase-1b-retinitis-pigmentosa-trial-2/
3. ViGeneron announces FDA rare pediatric disease designation for VG901 and DSMB approval to advance dose escalation in phase 1b retinitis pigmentosa trial. News release.ViGeneron GmbH. January 8, 2025. Accessed September 4, 2025. https://vigeneron.com/press/vigeneron-announces-fda-rare-pediatric-disease-designation-for-vg901-and-dsmb-approval-to-advance-dose-escalation-in-phase-1b-retinitis-pigmentosa-trial/
4. ViGeneron announces first patient dosed in phase 1b clinical trial of VG901 for the intravitreal treatment of retinitis pigmentosa. News release. ViGeneron GmbH. April 10, 2024. Accessed September 4, 2025. https://vigeneron.com/press/vigeneron-announces-first-patient-dosed-in-phase-1b-clinical-trial-of-vg901-for-the-intravitreal-treatment-of-retinitis-pigmentosa/
Newsletter
Stay at the forefront of cutting-edge science with CGT—your direct line to expert insights, breakthrough data, and real-time coverage of the latest advancements in cell and gene therapy.
Advertisement
Related Articles
VeonGen Snags FDA RMAT Designation for Stargardt Gene Therapy VG801September 4th 2025
CGTLive®’s Weekly Rewind – August 29, 2025August 29th 2025
- CGTLive®’s Weekly Rewind – August 22, 2025
August 22nd 2025
Advertisement
Advertisement
