▎WuXiEdited by Kant Content Team

ViGeneron GmbH recently announced that the companyThe IND application for the phase 1/2 clinical trial of VG801, a gene therapy under research, has been approved by the U.S. FDA for the treatment of Stargardt disease and other related conditions.ABCA4Retinal dystrophy associated with gene mutations.

VG801 utilizes ViGeneron's proprietary dual adeno-associated virus (AAV) vector REVeRT (Recombination via trans-splicing mRNA) technology, addressing the challenge of the DNA sequence encoding human ABCA4 (approximately 6.7 kb) being too large to fit into a single AAV vector.REVeRT consists of two AAV vectors, each carrying half of the ABCA4 coding sequence, producing two independent pre-mRNAs. These pre-mRNAs are trans-spliced to form a complete mRNA, which is then translated into a functional protein.VG801 also utilizes a novel vgAAV capsid, generated based on ViGeneron's proprietary vgAAV technology platform, designed to achieve widespread retinal transduction.

Previously published inNature CommunicationsOnPreclinical StudyIt shows that the REVeRT technology has been validated both in vitro and in vivo, demonstrating its ability to efficiently recombine disease-related genes and gene-editing modules larger than 5 kb.In a mouse model of Stargardt disease, researchers further demonstrated that the use of REVeRT technology successfully achievedABCA4High expression of the gene – a gene closely associated with Stargardt disease. Preliminary experimental data supports potential improvement in retinal function.

ViGeneron Co-Founder and CEO Caroline Man Xu, Ph.D., said in the press release: "VG801 delivers full functionality byABCA4"Genes have the potential to address the genetic root causes of Stargardt disease and other ABCA4-related retinal dystrophies, demonstrating our commitment to advancing next-generation gene therapies through innovative technologies to meet significant unmet medical needs."

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This Phase 1/2 multicenter, open-label, dose-escalation clinical trial will evaluate the safety, tolerability, and preliminary efficacy of VG801. As part of VG801's global clinical development strategy, the study will recruit patients globally.ViGeneronPlans to submit a Clinical Trial Application (CTA) to the European Medicines Agency (EMA) in the coming months.

Stargardt disease is one of the most common inherited retinal diseases, caused byABCA4Caused by gene mutations. Stargardt disease and other ABCA4-related retinal dystrophies lead to gradual vision loss, and currently, there are no approved treatments available to halt disease progression, highlighting an urgent need for effective therapies.

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