Gene therapy for X-linked retinitis pigmentosa shows positive safety, efficacy data

October 02, 2022

1 min watch


Michaelides M. Ph1/2 AAV5-RPGR (botaretigene sparoparvovec) gene therapy trial in RPGR-associated X-linked retinitis pigmentosa (XLRP). Presented at: American Academy of Ophthalmology meeting; Sept. 30-Oct. 3, 2022; Chicago.

Michaelides reports consulting for Janssen Pharmaceuticals and MeiraGTx and holding equity in MeiraGTx.

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CHICAGO — In this Healio Video Perspective from Retina Subspecialty Day at the AAO meeting, Michel Michaelides, MD, discusses study results from a gene therapy trial for RPGR-associated X-linked retinitis pigmentosa.

“We presented excellent safety and efficacy data both in terms of retinal sensitivity and, arguably more importantly, functional vision,” Michaelides said.

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