VeonGen Therapeutics debuts with FDA recognition and bold plans for inherited eye diseases.
ViGeneron (Munich, Germany) announced its rebranding as VeonGen Therapeutics, stepping into its next chapter as a clinical-stage gene therapy company focused on treating serious inherited diseases. The rebrand reflects the company’s progress and ongoing commitment to advancing genetic therapies in ophthalmology and beyond.
VeonGen announces the rebranding on its new LinkedIn page.
Leading the way is VG801, VeonGen’s investigational therapy for Stargardt disease and other ABCA4-related retinal dystrophies. The program recently received Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA), and a first-in-human Phase I/II clinical trial is already underway with patient dosing in progress.
READ MORE: FDA Grants Breakthrough Therapy Designation to Tinlarebant for Stargardt
VG801 is being studied in over 600 patients across 47 U.S. sites. The therapy combines two FDA-approved compounds—carbachol and brimonidine tartrate—to create a “pinhole effect” that improves near and distance vision by reducing pupil size and allowing only focused light rays to enter the eye.
To strengthen its impact, VeonGen is also working with the FDA’s Rare Disease Endpoint Advancement (RDEA) pilot program to develop new functional endpoints for VG801 in an aim to accelerate meaningful clinical outcomes in trials.
“Rebranding to VeonGen Therapeutics aligns with our clinical focus and the momentum behind our lead programs,” said Dr. Caroline Man Xu, co-founder and chief executive officer of VeonGen Therapeutics. “We are encouraged by the FDA’s RPDD designation, which highlights the potential of VG801 to address a significant unmet need.”
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More in the pipeline: VG901 and proprietary platforms
VG801 isn’t alone. VeonGen’s second clinical program, VG901, is being developed to treat retinitis pigmentosa caused by CNGA1 mutations, using an intravitreally delivered AAV gene therapy approach.
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Both programs are powered by VeonGen’s proprietary platforms:
- vgRNA REVeRT: (Reconstitution via mRNA Trans-splicing): Tackling the AAV cargo limit challenge head-on, this platform allows delivery of large genes—those exceeding the 4.7 kb limit—by enabling their reconstitution at the mRNA level. This approach broadens therapeutic possibilities, especially for conditions caused by large-gene mutations.
- vgAAV: This platform features engineered AAV capsids optimized for enhanced transduction efficiency. It’s designed to bypass biological barriers, enabling safer, less invasive delivery routes such as intravitreal and even systemic administration—expanding the therapeutic reach of AAV-based therapies.
- AAV Transactivation: A powerful tool that harnesses CRISPR/Cas-mediated transcriptional regulation, this platform enables precise in vivo activation or repression of disease-relevant genes. It opens the door to applications beyond traditional gene supplementation, including potential breakthroughs in genetic diagnostics and regulation-based therapies.
VeonGen is also exploring broader applications in cardiovascular, CNS and additional genetic conditions, expanding the reach of its platforms beyond ophthalmology.
Editor’s Note:
- For more information, see the official press release from VeonGen Therapeutics.
- This content is intended exclusively for healthcare professionals. It is not intended for the general public. Products or therapies discussed may not be registered or approved in all jurisdictions, including Singapore.