VeonGen, FDA align on phase 3 trial for Stargardt disease gene therapy

VeonGen has reached alignment with the FDA on key clinical and CMC aspects of the pivotal phase 3 program for VG801, its investigational dual-AAV gene therapy for Stargardt disease and other ABCA4-associated retinal dystrophies.

The RMAT Type B meeting minutes document alignment on the pivotal study design, including the primary endpoint, study duration, and control arm. Regarding CMC, FDA alignment includes the 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 (VRVT) through the FDA’s rare disease endpoint advancement (RDEA) pilot program to support its development as a functional vision measure in Stargardt disease.

The ongoing first-in-human phase 1/2 study of VG801 has dosed 15 adult and pediatric patients across three dose cohorts, with consistent, sustained functional improvements through Month 12 across multiple assessments, including best-corrected visual acuity (BCVA), VRVT and microperimetry fixation.

The most common form of inherited juvenile macular degeneration, Stargardt is caused by mutations in the ABCA4 gene. The condition leads to the progressive loss of central vision and has no approved treatment. AAVantgarde Bio, SpliceBio and Ocugen are also developing gene therapies for Stargardt.