Posted by Michael Wonder on 21 Aug 2025
VeonGen Therapeutics receives FDA regenerative medicine advanced therapy designation for VG801 gene therapy for Stargardt disease
21 August 2025 - VeonGen Therapeutics today announced that the US FDA has granted regenerative medicine advanced therapy designation to VG801, its lead investigational gene therapy for Stargardt disease and other ABCA4 mutation–associated retinal dystrophies.
By delivering a full length, functional ABCA4 gene, VG801 has the potential to address the underlying genetic root cause of Stargardt disease across all ABCA4 mutations.
