Complications in gene therapy for inherited retinal diseases highlighted at ASRS 2023

Although gene augmentation therapy using transvitreal subretinal injections (TSI) shows promise in treating inherited retinal diseases (IRDs), it is associated with a significant rate of complications, according to a study presented at ASRS 2023 which found that complications were relatively common, affecting approximately one-third of the eyes treated.

The complications observed during the study included issues related to the surgical procedure, the vector used in the therapy, and steroid-induced intraocular pressure elevation. Some complications were unique to specific IRDs.

Notably, chorioretinal (CR) atrophy was more prevalent among eyes treated for RPE65, and it correlated with longer follow-up duration.

Despite the complications, the study indicated that most of them were mild to moderate in severity, treatable, and did not have a significant impact on the final visual acuity outcome. However, it was observed that intact retinal anatomy, particularly preoperative foveal ellipsoid zone (EZ), played a role in facilitating faster visual acuity recovery and better tolerance against visual deficits resulting from complications.

The findings underscore the importance of closely monitoring patients undergoing gene augmentation therapy and promptly addressing any complications that arise. Further research and larger studies will be necessary to validate these findings and refine the techniques used in gene augmentation therapy for IRDs to optimize patient outcomes.

Reference

Khoo C. Complications of Transvitreal Subretinal Injections for Gene Therapy Vectors in Inherited Retinal Diseases. Presented at: ASRS 41st Annual Meeting.