Editor’s note: This is a developing news story. Please check back soon for updates.
The FDA granted VG801 rare pediatric disease designation in Stargardt disease, according to a press release from VeonGen Therapeutics.
VeonGen Therapeutics, formerly known as ViGeneron, is investigating the novel dual AAV gene therapy in a first-in-human phase 1/2 clinical trial, with dosing underway. Additionally, it is working with the FDA to develop a functional endpoint as part of the Rare Disease Endpoint Advancement Pilot Program, as Healio previously reported.
VG801 and VG901, a clinical program in