VeonGen and FDA Align on Phase 3 Study for VG801 in Stargardt Disease

VeonGen Therapeutics announced its alignment with the FDA on the phase 3 development of VG801, a gene therapy targeting Stargardt disease. This milestone clarifies regulatory aspects for the pivotal study design and manufacturing strategy. VG801 has already shown promising results in its phase 1/2 trials, demonstrating functional vision improvements in patients over 12 months.

In a meeting with the FDA, VeonGen secured agreement on the study's primary endpoint, duration, and control measures. Importantly, they confirmed the use of their existing clinical batch for upcoming trials. Additionally, VG801’s functional improvement was noted in best-corrected visual acuity and other vision tests, with 11 of 13 patients showing significant letter gains from baseline visual acuity tests.

VeonGen’s dual-AAV approach combines novel capsid and vgRNA technologies to address the genetic causes of Stargardt disease. VG801 aims to offer a broad therapeutic approach, agnostic to ABCA4 mutations, providing potential vision preservation or improvement for patients.