ViGeneron GmbH announced FDA clearance for its investigational new drug (IND) application for VG801, a novel mRNA trans-splicing gene therapy designed to treat Stargardt disease and other ABCA4-linked retinal dystrophies. The clearance allows for a Phase 1/2 trial to evaluate the therapy’s safety, tolerability and initial efficacy.

VG801 utilizes ViGeneron’s REVeRT platform, which reconstitutes the full-length ABCA4 gene by combining two halves delivered via dual AAV vectors, the company said in a press release. The therapy aims to address the genetic root cause of Stargardt disease, the most common inherited retinal disease, which leads to progressive vision loss.

According to the company, this IND marks ViGeneron’s second clinical-stage program, complementing its VG901 therapy for retinitis pigmentosa. ViGeneron's novel vgAAV capsid technology is designed to enhance retinal delivery and expand therapeutic potential, the company said in the release. 

The FDA also selected VG801 for its Rare Disease Endpoint Advancement (RDEA) pilot program, enabling collaborative endpoint development and expedited regulatory feedback. A European clinical trial application is planned for 2025.

Stargardt disease currently lacks approved treatments, making VG801 a potential breakthrough for patients with biallelic ABCA4 mutations, the company said. Preclinical studies have demonstrated durable expression of the full-length ABCA4 gene and safety in animal models and human retinal organoids.