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Phase 1/2 trial of gene therapy treatment for X-linked retinitis pigmentosa begins

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A phase 1/2 clinical trial of a gene therapy treatment for X-linked retinitis pigmentosa has begun enrolling and dosing subjects, NightstaRx announced in a press release.

It is the first kind of trial like this for X-linked retinitis pigmentosa. The gene therapy, AAV-XLRPGR, uses adeno-associated virus to deliver a codon-optimized copy of the retinitis pigmentosa GTPase regulator gene into the cells of the eye, the release said.

“The current trial is a multicenter open-label, dose-escalation study designed to enroll at least 24 male patients at ophthalmology centers of excellence, such as Oxford and Manchester,” Aniz Girach, MD, chief medical officer of NightstaRx, said in the release. “Each patient will receive a single subretinal injection of AAV-XLRPGR gene therapy. The primary goal of the study is to assess safety and tolerability of AAV-XLRPGR over a 12-month period.”