VeonGen Therapeutics Secures FDA RMAT Designation for VG801 in Stargardt Disease

VeonGen Therapeutics has announced that its lead investigational gene therapy, VG801, has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA. VG801 targets Stargardt disease, a common inherited retinal disorder caused by ABCA4 gene mutations. This designation supports the therapy's potential to address unmet medical needs.

RMAT designation offers benefits such as enhanced engagement with the FDA and pathways for expedited development. Accumulating RMAT, Rare Pediatric Disease, and Orphan Drug designations marks a significant milestone for VG801.

Currently under a Phase I/II clinical trial, VG801 aims to restore full-length ABCA4 expression using VeonGen’s proprietary platforms, addressing the genetic cause across all forms of Stargardt disease.