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The FDA granted regenerative medicine advanced therapy designation to VG801 for the treatment of Stargardt disease, according to a press release from VeonGen Therapeutics.
VG801, a dual-AAV gene therapy, is being evaluated in a phase 1/2 clinical trial. It previously received rare pediatric disease and orphan drug designations from the FDA, according to the release.
The FDA grants regenerative medicine advanced therapy (RMAT) designation to regenerative medicine therapies, including gene therapies, that are