Newly-renamed and rebranded VeonGen Therapeutics announced clinical progress for its Stargardt disease gene therapy candidate VG801—including a new FDA designation and plans for a first-in-human clinical trial.
Lots to unpack! But first: What was the pre-rebranded name?
Its original name: ViGeneron GmbH.
The reason for the change: “This rebranding reflects our journey—from a platform innovator to a clinical-stage company with two gene therapies in the clinic,” according to Caroline Man Xu, PhD, co-founder & CEO
Tell me more about this company.
The German-based, clinical-stage genetic medicine company was founded in 2017 as a spin-off of the Ludwig-Maximilians-University Munich.
- Its clinical focus: Two inherited retinal disease (IRD) gene therapy programs—and one currently unnamed—built on three proprietary and viral vector-based gene therapy platforms.
And those programs?
- VG901, an intravitreally-delivered, adeno-associated virus (AAV) gene therapy
- Note: This is based on the vgAAV platform
- Received FDA Rare Pediatric Disease Designation (RPDD) for retinitis pigmentosa (RP) in January 2025 (and Orphan Drug designation before that)
- Note: This is based on the vgAAV platform
- VG801, a dual AAV that leverages mRNA trans-splicing
- Note: This is based on the vgRNA REVeRT and vgAAV platforms
Explain the basis for these gene therapy platforms.
To start: All three platforms are based on adeno-associated virus (AAV) vectors and designed to resolve two major limitations of current AAV-based gene therapies:
- Limited DNA uptake capacity (prevents treatment of large genes)
- Limited ability to cross biological barriers (requires local delivery and, as a result, restricts administration routes)
Let’s look specifically at the two platforms VG801 is involved with.
- The vgAAV platform includes engineered AAV capsids with advanced transduction efficiency as well as the capability to circumvent biological obstacles—allowing fewer invasive delivery routes, like intravitreal (IV) and systemic administration
- The vgRNA REVeRT (Reconstitution via mRNA Trans-splicing) platform permits large gene (+4.7 kb) delivery and reconstruction at the mRNA level in tissues identified via a selected AAV capsid
Now talk about the candidate.
Take note: VG801 is intended to treat patients with biallelic ABCA4 mutations linked to Stargardt and related retinal dystrophies.
What it does: This dual AAV relies on the vgRNA REVeRT and vgAAV platforms to deliver the full-length ABCA4 gene for Stargardt.
- As a refresh: Click here for some background on how mutations in the ABCA4 gene lead to Stargardt.
So! What’s the latest FDA news on this candidate?
VeonGen announced that the federal agency granted Rare Pediatric Disease Designation (RPDD) to VG801 for the treatment of ABCA4 mutation-associated retinal dystrophies (such as Stargardt).
- Why this is critical: Such a designation means VeonGen may become eligible for a Priority Review Voucher (PRV) following a possible market approval of VG801.
- Specifically: This PRV would also shorten the FDA review process and be transferable to another asset in its pipeline (including VG901 or that other undisclosed program)
Nice! And what comes next?
The company shared that VG801 is currently under clinical investigation in a first-in-human phase 1/2 clinical trial, with patient dosing underway.
- Along with this: VeonGen is also developing a functional endpoint in collaboration with the FDA through the Rare Disease Endpoint Advancement (RDEA) pilot program.
What we know about the phase 1/2 study setup:
- The design: A single-arm, open-label, non-randomized, single dose-escalation study (NCT07002398)
- The participants: An estimated 15 patients (aged 6+) diagnosed with Stargardt due to biallelic ABCA4 mutations (see the criteria)
- The setup: Patients are receiving a single, subretinal injection of VG801 in three varying doses (low, medium, and high)
And what's being measured?
Starting with the primary outcome:
- Adverse events (AEs) and serious AEs
And the secondary outcomes:
- Best-corrected visual acuity (BCVA)
- Optical coherence tomography (OCT)
- Fundus autofluorescence
- Microperimetry
- Novel virtual reality mobility test (exploratory)
Note: All outcomes except for BCVA (measured at screening to Month 12) are being measured from baseline to Month 12).
Lastly, when is data expected?
The study is planned to conclude in June 2026, so keep any eye out for interim data in the meantime.