VeonGen Therapeutics said it reached alignment with the US Food and Drug Administration (FDA) on elements of a planned phase 3 trial of VG801, its investigational gene therapy for Stargardt disease and other ABCA4-associated retinal dystrophies.

According to the company’s November 5 announcement, FDA meeting minutes documented agreement on the primary endpoint, study duration, and control arm, although those details were not disclosed. Discussions also addressed use of the existing clinical batch in the pivotal trial and aspects of commercial manufacturing.

The ongoing phase 1/2 study has treated 15 adults and children across 3 dose cohorts. Among 13 patients with measurable baseline best-corrected visual acuity (BCVA), all showed improvement at month 6 or month 12. Eleven gained at least 5 Early Treatment Diabetic Retinopathy Study letters, including 7 who gained at least 10 and 4 who gained at least 15.

VeonGen also reported improvements through month 12 in BCVA, virtual reality–based visual testing, and microperimetry fixation, with stable retinal structures. The company described safety findings as favorable but provided no adverse event counts or comparative efficacy results in the announcement.

VG801 uses 2 adeno-associated viral vectors and an RNA trans-splicing platform designed to reconstitute full-length, functional ABCA4 in retinal photoreceptors. The approach is intended to address multiple disease-causing variants. RP