FDA Clears ViGeneron's IND for Novel mRNA Gene Therapy to Treat Stargardt Disease and Other Retinal Dystrophies
ViGeneron announced that the FDA has cleared the investigational new drug (IND) application for the phase 1/2 study of VG801, a gene therapy candidate to treat Stargardt disease and other retinal dystrophies associated with mutations in the ABCA4 gene.
VG801 utilizes ViGeneron’s proprietary dual AAV vector technology REVeRT (REconstitution Via mRNA Trans-splicing), as the human ABCA4 coding sequence, approximately 6.7kb in size, is too large to be packaged into a single AAV vector (Nature Communications 2023, PMID: 37852949). REVeRT enables the reconstitution of the full-length ABCA4 messenger ribonucleic acid (mRNA) through transduction with dual AAV vectors. Each vector carries one half of the ABCA4 coding sequence, producing two separate pre-mRNAs that are trans-spliced to form a full-length mRNA, which is subsequently translated into the functional protein. VG801 utilizes a novel vgAAV capsid, derived from ViGeneron’s proprietary vgAAV technology and designed to enable widespread retinal transduction (EMBO Mol Med 2021, PMID: 33616280).
“By delivering full-length functional ABCA4 gene, VG801 has the therapeutic potential to address the genetic root cause of Stargardt disease and other ABCA4-linked retinal dystrophies,” Caroline Man Xu, PhD, ViGeneron’s Co-founder and CEO, said in a company news release. "The VG801 IND clearance, together with our ongoing VG901 clinical trial for retinitis pigmentosa, expands our clinical-stage pipeline to two programs and underscores our commitment to advancing next-generation gene therapies with novel technologies to address significant unmet medical needs.”
The phase 1/2 multicenter, open-label, dose-escalation clinical trial will evaluate the safety, tolerability and preliminary efficacy of VG801. The study will recruit patients globally as part of a global regulatory strategy for VG801’s clinical development, with a Clinical Trial Application (CTA) submission to the European Medicines Agency (EMA) planned in the coming months.
ViGeneron also announced that the FDA has selected its proposal for the Rare Disease Endpoint Advancement (RDEA) pilot program. The RDEA program is designed to foster innovation and advance rare disease drug development programs. As part of the RDEA program, ViGeneron will collaborate with the FDA throughout the efficacy endpoint development process and gain additional opportunities to meet with the FDA. Notably, the FDA accepts no more than three RDEA programs per year for fiscal years 2024 to 2027.