VeonGen Therapeutics has reached alignment with the FDA at a Regenerative Medicine Advanced Therapy Type B meeting on key clinical and Chemistry, Manufacturing and Controls aspects of VG801's pivotal Phase 3 program. VG801 is the company's investigational dual-AAV gene therapy for Stargardt disease and other ABCA4-associated retinal dystrophies.
The FDA meeting minutes document alignment on the pivotal study design, including the primary endpoint, study duration and control arm. On CMC, alignment includes use of the existing clinical batch for the pivotal Phase 3 study and key aspects of the commercial manufacturing strategy.
In parallel, VeonGen is advancing the Virtual Reality Visual Test through the FDA's Rare Disease Endpoint Advancement Pilot Program to support its development as a functional vision measure in Stargardt disease. VG801 has received Phase 1/2 clinical trial authorizations in Europe and China.
The ongoing first-in-human Phase 1/2 study has dosed 15 patients across three dose cohorts, including adults and pediatric patients. VG801 has demonstrated consistent and sustained functional improvements through Month 12 across best-corrected visual acuity, VRVT and microperimetry fixation. Among the 13 patients with measurable baseline BCVA, all showed improvements from baseline to Month 6 or Month 12, with 11 achieving gains of at least 5 ETDRS letters, including 7 achieving at least 10 letters and 4 achieving at least 15 letters.
VG801 combines the company's vgAAV2 capsid with vgRNA REVeRT to enable delivery and reconstitution of a full-length functional human ABCA4 gene in retinal photoreceptor cells, offering a genotype-agnostic approach across different ABCA4 mutations.
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