ViGeneron GmbH, a clinical-stage gene therapy company, has achieved two significant milestones in the development of its novel gene therapy candidate, VG901, for the treatment of retinitis pigmentosa (RP) caused by mutations in the CNGA1 gene. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to VG901, and the independent Data Safety Monitoring Board (DSMB) has unanimously approved dose escalation in the ongoing Phase 1b clinical trial.
The FDA's RPDD acknowledges the critical need for treatments addressing rare pediatric conditions like retinitis pigmentosa caused by CNGA1 mutations. This designation qualifies ViGeneron to potentially secure a Priority Review Voucher (PRV) upon VG901's marketing approval. A PRV enables accelerated FDA review of any drug candidate in the company’s pipeline, reducing the review period to six months, and is transferable for use or sale.
Advancing VG901 for CNGA1-Associated Retinitis Pigmentosa
VG901 is a first-in-class gene therapy designed to deliver the functional CNGA1 gene to retinal photoreceptor cells via intravitreal injection, utilizing ViGeneron's novel next-generation vgAAV capsid. The ongoing Phase 1b trial (NCT06291935) is an open-label, single-arm, dose-escalation study assessing the safety, tolerability, and preliminary efficacy of a one-time intravitreal administration of VG901.
"This RPDD recognition from the FDA highlights the significant unmet medical need in retinitis pigmentosa and underscores VG901's therapeutic potential as the first-in-class and only clinical-stage therapy targeting retinitis pigmentosa associated with mutations in the CNGA1 gene," said Dr. Caroline Man Xu, ViGeneron’s Co-founder and CEO. "In addition to the previously granted FDA Orphan Drug Designation for VG901, the RPDD designation further supports our efforts to accelerate the development of VG901."
The DSMB, composed of independent experts, conducts a rigorous evaluation of trial data after each cohort of patients. "The DSMB has unanimously recommended proceeding with dose escalation in the ongoing VG901 Phase 1b clinical trial," commented Dr. Bart P Leroy, Ophthalmologist & Clinical Geneticist, Head of Department of Ophthalmology, Ghent University Hospital, Belgium, and the DSMB Chair for this trial. "No dose-limiting adverse events related to VG901 have been reported in the first-dose cohort to date. This marks a critical step toward advancing to the higher dose and represents an important milestone in its clinical development."
Addressing Retinitis Pigmentosa
Retinitis pigmentosa (RP) is a group of related eye disorders that cause progressive vision loss. It is the most common type of inherited retinal disease (IRD), estimated to affect 1 in 3,500 to 1 in 4,000 people in the United States and Europe, respectively. Mutations in the CNGA1 gene, encoding a subunit of CNG channels in rod photoreceptors, are reported to cause approximately 2%–8% of autosomal recessive retinitis pigmentosa (arRP).
Advancing Novel Therapies for Stargardt Disease
ViGeneron also announced that the FDA has cleared the Investigational New Drug (IND) application for the Phase I/II study of VG801, a potentially transformative gene therapy 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.
ViGeneron also proudly 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 novel efficacy endpoint development process and gain additional opportunities to meet with the FDA.
