Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations

投稿日：2023-02-16

Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations

Summary of Key Points

Cotoretigene toliparvovec (AAV2-RPGR) is a gene therapy for X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.
A study was conducted to assess the safety and efficacy of AAV2-RPGR in XLRP patients.
The study found that AAV2-RPGR was safe and well-tolerated, and that it improved retinal sensitivity in the treated eyes.
The improvements in retinal sensitivity were sustained for up to 12 months after treatment.
These results suggest that AAV2-RPGR is a promising treatment for XLRP caused by RPGR gene variations.

Detailed Overview

X-linked retinitis pigmentosa (XLRP) is a rare, inherited retinal degenerative disorder caused by mutations in the RPGR gene. It is characterized by progressive vision loss, night blindness, and constricted visual fields. Cotoretigene toliparvovec (AAV2-RPGR) is a gene therapy that has been developed to treat XLRP caused by mutations in the RPGR gene.

To assess the safety and efficacy of AAV2-RPGR in XLRP patients, a study was conducted involving 12 patients with XLRP caused by RPGR gene variations. The patients were treated with AAV2-RPGR in one eye and a sham procedure in the other eye. The primary outcome measure was the change in retinal sensitivity from baseline to 12 months after treatment.

The results of the study showed that AAV2-RPGR was safe and well-tolerated, with no serious adverse events reported. The treated eyes showed a significant improvement in retinal sensitivity compared to the sham-treated eyes, and the improvements were sustained for up to 12 months after treatment.

These results suggest that AAV2-RPGR is a promising treatment for XLRP caused by RPGR gene variations. Further studies are needed to confirm the safety and efficacy of AAV2-RPGR in larger patient populations.

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published: 2023 Feb 9
Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations

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