ViGeneron Gains FDA IND Clearance for VG801 mRNA Gene Therapy for Stargardt Disease

ViGeneron GmbH, a pioneering company in the field of gene therapy, has announced that the FDA has approved their Investigational New Drug (IND) application for VG801. This marks a significant step forward for the treatment of Stargardt disease and other retinal dystrophies linked to mutations in the ABCA4 gene. VG801 represents a groundbreaking approach in gene therapy, using ViGeneron's proprietary dual AAV vector technology named REVeRT. This technology addresses the challenge of the large size of the ABCA4 coding sequence, approximately 6.7 kilobases, which is too big to fit into a single AAV vector. Instead, REVeRT facilitates the reconstitution of the complete ABCA4 mRNA by utilizing two AAV vectors, each delivering half of the coding sequence. These are then trans-spliced to create a full-length mRNA, which is translated into the functional protein necessary for proper retinal function.

VG801 employs a unique vgAAV capsid developed by ViGeneron, tailored to achieve widespread transduction in the retina. As Dr. Caroline Man Xu, the Co-founder and CEO of ViGeneron, emphasized, VG801 holds considerable promise in tackling the genetic cause of Stargardt disease and other ABCA4-related retinal disorders. This latest IND clearance, along with the ongoing VG901 clinical trial for Retinitis Pigmentosa, significantly expands ViGeneron's clinical-stage pipeline, showcasing their dedication to advancing innovative gene therapies that address urgent unmet medical needs.

The upcoming Phase I/II clinical trial is designed as a multicenter, open-label study that will explore the safety, tolerability, and initial efficacy of VG801. The trial will be conducted globally, and ViGeneron is planning to submit a Clinical Trial Application to the European Medicines Agency soon. Furthermore, ViGeneron has been chosen by the FDA to participate in the Rare Disease Endpoint Advancement (RDEA) pilot program. This initiative aims to drive innovation in the development of treatments for rare diseases. Under this program, ViGeneron will work closely with the FDA to develop novel efficacy endpoints, benefiting from additional opportunities for engagement with the regulatory body.

Stargardt disease is a prevalent inherited retinal disease, affecting approximately 1 in 8,000 to 10,000 individuals worldwide. It is characterized by a gradual decline in visual acuity and can lead to legal blindness, primarily due to mutations in the ABCA4 gene. Currently, no approved treatments exist to halt the progression of this disease, underscoring the need for effective interventions. VG801 represents a novel therapeutic approach aimed at addressing this unmet need by delivering the full-length ABCA4 gene through dual AAV vectors. This strategy has shown promising results, demonstrating in vivo functionality and expression in animal models and human retinal organoids.

ViGeneron, founded in 2017 in Munich, Germany, is committed to advancing gene therapy innovations for the treatment of ophthalmic diseases. The company has developed three proprietary platforms to overcome the limitations of existing AAV-based therapies. These include the vgAAV vector platform, designed for superior transduction efficiency and less invasive administration routes; the REVeRT technology for reconstructing large genes in targeted tissues; and the AAV Transactivation platform, which uses CRISPR-Cas for regulating gene expression. Through these platforms, ViGeneron aims to transform the landscape of gene therapy, bringing hope to patients with previously untreatable conditions.