VeonGen Therapeutics, formerly ViGeneron, announced a new corporate identity and progress on its lead gene therapy candidate, VG801, which has received Rare Pediatric Disease Designation from the US Food and Drug Administration (FDA).

VG801 targets patients with biallelic mutations in the ABCA4 gene and is delivered via a dual AAV vector platform using VeonGen’s proprietary vgRNA REVeRT and vgAAV technologies. A phase 1/2 clinical trial to evaluate VG801 as a treatment for Stargardt disease, the most common inherited retinal disease, is currently recruiting.

In conjunction with the trial, VeonGen is developing a new functional endpoint under the FDA’s Rare Disease Endpoint Advancement (RDEA) program. The company has a second program, VG901, in development for retinitis pigmentosa caused by CNGA1 mutations.

In a press release, VeonGen explained that its new name reflects a shift from platform innovation to clinical development. The company is also exploring additional indications beyond ophthalmology, including cardiovascular and central nervous system diseases.