MeiraGTx (NASDAQ:MGTX) reported that AAV-RPE65, its investigational gene therapy for the treatment of retinal dystrophy, met the primary endpoint of safety and tolerability in a Phase 1/2 study, as well as demonstrated significant improvement in vision at six months.
Retinal dystrophy caused by a RPE65 deficiency is a rare genetic disorder that results in the degeneration of the entire retina over time, significantly impairing vision and usually resulting in blindness. AAV-RPE65 is designed to deliver a normal copy of the RPE65 gene via subretinal injection to restore the eye’s photoreceptor function.
“This trial demonstrated that AAV-RPE65 has the potential to restore vision in a severely debilitating disease. These highly encouraging early data reflect the promise of gene therapy to change the lives of patients with blinding inherited retinal diseases,” Dr. Michel Michaelides, MeiraGTx’s head of clinical ophthalmology, said in a statement.
Having selected the dose for its planned pivotal study, MeiraGTx intends to meet with regulatory authorities in the second half of 2019 to define AAV-RPE65’s regulatory approval pathway. Full data from the Phase 1/2 trial are expected later in 2019.
