Fresh off its rebrand, VeonGen Therapeutics has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for VG801, its investigational gene therapy for Stargardt disease.
Refresh me: VeonGen is … ?
Based in Germany and a clinical-stage genetic medicine company with two gene therapies under clinical investigation.
- Its former name: ViGeneron GmbH.
Check out this coverage for info on its background and rebrand.
Now to this candidate.
VG801 is a dual adeno-associated virus (AAV) that leverages mRNA trans-splicing and relies on VeonGen’s proprietary and viral vector-based gene therapy platforms (more on those in a moment).
Its purpose: The therapeutic is currently under development and research for the treatment of patients diagnosed with biallelic ABCA4 mutations linked to Stargardt and related retinal dystrophies.
- Its mechanism of action: Is linked to those aforementioned gene therapy platforms.
Tell me about those.
- While VeonGen has developed three vector platforms, VG801 is involved with two:
- vgAAV platform
- vgRNA REVeRT (Reconstitution via mRNA Trans-splicing) platform
Their purpose: To address (and circumvent) two notable limitations associated with current AAV-based gene therapies:
- Limited capacity for DNA uptake, preventing large genes (+4.7kb) from receiving treatment
- Limited ability to cross biological barriers (in other words: to effectively pass through the body’s natural defenses such as skin or tissue)
See here for a more detailed look at the capabilities of each platform.
And how are they utilized by VG801?
The dual AAV relies on both platforms to deliver the full-length ABCA4 gene for Stargardt.
Why ABCA4? Because mutations in this gene are the culprit behind the development of Stargardt.
Duly noted. Now let’s talk FDA designations.
To date, RMAT is the second designation VG801 has received—it was previously granted Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation.
The significance of that: This made VeonGen potentially eligible for the highly coveted Priority Review Voucher—if and only if) VG801 eventually receives market approval, that is.
- See why else this could be a game-changer for the company.
Nice! So what does RMAT mean?
First: RMAT applies to regenerative medicine therapies (VG801, in this instance), which use the body’s own cells to promote healing and repair damaged tissues or organs.
Next: RMAT is just one of several designations in the FDA program exclusively designed for RMTs to speed up the development and review process of investigational drug candidates intended to treat, modify, reverse, or cure a serious or life-threatening condition.
- And looking at RMAT specifically: This is based on preliminary clinical evidence indicating a specific therapy could potentially address an unmet need for the condition.
What’s the advantage of this for VG801?
As Caroline Man Xu, PhD, VeonGen’s CEO and co-founder, noted: “Receiving RMAT designation is strong recognition of VG801’s therapeutic potential for Stargardt disease, the most common inherited retinal disorder with no approved therapies.”
And looking forward?
A couple of ongoing developments involving this candidate:
- In collaboration with the FDA, VeonGen is in the process of developing a functional endpoint through the federal agency’s Rare Disease Endpoint Advancement (RDEA) pilot program (intended to support drugs with rare disease-intended indications
- Patient dosing in a non-randomized phase 1/2 study (NCT07002398) is currently underway among patients (aged 6+) diagnosed with Stargardt due to biallelic ABCA4 mutations. Check out those details.
And when might data be available from that study?
With a June 2026 expected conclusion date, likely sometime between now and then. Stay tuned!