VeonGen Therapeutics wins FDA RMAT designation for VG801

German gene therapies developer VeonGen Therapeutics today revealed that the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) 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, said VeonGen, which until recently was known as ViGeneron.