Gene Therapy Shows Early Potential in XLRP

July 25, 2020
Kevin Kunzmann

New phase 1/2 data shows AAV-RGPR may benefit a previously untreated population of inherited retina disease boys and young men.

At the American Society of Retina Specialists (ASRS) 2020 Virtual Sessions this weekend, phase 1/2 data from a novel assessment of investigative gene therapy AAV-RGPR showed early safety and tolerability among patients with X-linked retinitis pigmentosa (XLRP).

The findings, from a team of Janssen and Meira GTx investigators, could have significant implications for the XLRP treatment field. Currently, no drugs are approved by the US Food and Drug Administration (FDA) for the disease which causes debilitating vision in boys and young men.

Additionally, the new findings add fodder to the greater discussion of gene therapy in ophthalmology.

In alignment with the new ASRS 2020 data, AAV-RGPR primary investigator Michel Michaelides, MD, joined DocTalk for a discussion on the early trial findings, the burden of disease among patients with XLRP, and what promises are held for gene therapy in ophthalmology.


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