VeonGen’s Stargardt Disease Gene Therapy Gets FDA RMAT Status

The clinical-stage genetic medicine company VeonGen Therapeutics announced on Aug. 21, 2025 that FDA has granted regenerative medicine advanced therapy (RMAT) designation to VG801, VeonGen’s lead investigational gene therapy for Stargardt disease and other retinal dystrophies associated with mutations in the ABCA4 gene (1).

Frequency and symptoms of Stargardt

VG801 also holds rare pediatric disease and orphan drug designations in addition to RMAT, and VeonGen has been selected to work with FDA under the Rare Disease Endpoint Advancement pilot program; the purpose of that collaboration is to develop a novel, functional endpoint for Stargardt disease (1).

According to VeonGen, Stargardt disease is characterized by progressive central vision loss, often beginning in childhood or adolescence, which may gradually lead to loss of visual acuity and, in some cases, legal blindness (1). It affects approximately 1 out of every 8000 to 10,000 individuals worldwide, making it the most prevalent of all inherited retinal disorders.

There are no approved treatments for Stargardt disease at this time. VG801, a dual adeno-associated virus (AAV) gene therapy, is designed to restore full-length, functional ABCA4 expression and is currently being evaluated in a Phase I/II clinical trial (1).

“Receiving RMAT designation is strong recognition of VG801’s therapeutic potential for Stargardt disease, the most common inherited retinal disorder with no approved therapies,” said Caroline Man Xu, PhD, co-founder and CEO of VeonGen Therapeutics, in a company press release (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.”

First major news following rebrand

In June 2025, VeonGen Therapeutics announced its evolution into a clinical-stage company by rebranding from its former name, ViGeneron (2). VG801 was identified at that time as leading the company’s pipeline, noting that the therapy was the first novel messenger RNA trans-splicing gene therapy to receive investigational new drug (IND) clearance from FDA.

Another VeonGen investigational gene therapy, VG901, was reported to be continuing clinical development as well at the time of the rebrand. This treatment is designed for patients with retinitis pigmentosa caused by mutations in the CNGA1 gene and was granted rare pediatric disease status by FDA in January 2025 (2).

Other Stargardt treatments in pipeline

Another IND application for a therapy for Stargardt disease was cleared by FDA in July 2025, AAVantgarde Bio’s AAVB-039 (3). Similarly to VeonGen’s VG801, AAVantgarde said AAVB-039 has the potential to benefit people with ABCA4 mutations, targeting the underlying genetic cause of Stargardt disease by restoring the full-length ABCA4 protein. The gene therapy uses a proprietary dual AAV intein platform to deliver large genes.

In April 2025, as part of an announcement of various investments being made, Entos Pharmaceuticals said it would be creating a state-of-the-art R&D center and good manufacturing practice biomanufacturing facility in Edmonton, Alberta, Canada (4). At that time, an Entos treatment for Stargardt disease was mentioned among the therapeutic pipeline that the company said this new facility would help advance.

References

1. VeonGen Therapeutics. VeonGen Therapeutics Receives FDA Regenerative Medicine Advanced Therapy (RMAT) Designation for VG801 Gene Therapy for Stargardt Disease. Press Release. Aug. 21, 2025.
2. VeonGen Therapeutics. ViGeneron Rebrands as VeonGen Therapeutics and Announces FDA Rare Pediatric Disease Designation and Clinical Progress for Lead Gene Therapy VG801 in Stargardt Disease. Press Release. June 5, 2025.
3. AAVantgarde. AAVantgarde Receives FDA IND Clearance to Progress Stargardt Disease program AAVB-039. Press Release. July 15, 2025.
4. Mirasol, F. Entos Collaborates with Circio to Develop In-Vivo Delivery of DNA Vectors. BioPharmInternational.com, April 10, 2025.