VG801 is an innovative gene therapy designed to deliver a full-length, functional ABCA4 gene to address the genetic root cause of Stargardt disease, which affects approximately 1 in 8,000 to 10,000 people worldwide and currently has no approved treatments.

Stargardt disease is the most common inherited retinal disorder, characterized by progressive central vision loss starting in childhood or adolescence, and affects about 1 in 8,000 to 10,000 individuals globally.

VG801 utilizes VeonGen’s proprietary vgRNA REVeRT platform and engineered vgAAV capsids to target all forms of Stargardt disease by restoring ABCA4 gene function in photoreceptor cells.

Currently in Phase I/II clinical trials, VG801 has patient dosing underway and aims to restore vision by targeting the underlying genetic mutation.

VeonGen has received FDA's Regenerative Medicine Advanced Therapy (RMAT) designation for VG801, which accelerates development through enhanced FDA engagement, streamlined processes, and potential priority review.

VG801 has also earned designations such as Rare Pediatric Disease and Orphan Drug status, and VeonGen is collaborating with the FDA under the Rare Disease Endpoint Advancement pilot program.

VeonGen’s broader pipeline includes therapies for retinitis pigmentosa and applications in cardiovascular and CNS diseases, leveraging proprietary gene therapy platforms.

Based in Munich, Germany, VeonGen focuses on next-generation gene therapies for high unmet medical needs, with additional candidates like VG901 for retinitis pigmentosa.

The therapy aims to restore ABCA4 expression in photoreceptor cells to treat all forms of ABCA4-mutated Stargardt disease, the most common inherited retinal disorder causing progressive vision loss.