Cheryl Guttman Krader, BS, Pharm

Novel gene therapy demonstrates improved visual acuity and retinal thickness.

According to Jeffrey Heier, MD, RGX-314 administered by subretinal injection was well tolerated. In addition, it was associated with dose-dependent increase in ocular protein expression and evidence of clinical benefit.

Speakers at AAO 2019 event describe journey leading to approval of the first gene therapy for a genetic disease.

Phase III studies of a gene therapy for Leber Hereditary Optic Neuropathy have generated some unexpected positive findings. There is biologic plausibility to explain the data.