Abstract
Purpose :
To evaluate the efficacy and safety of intravitreal injection of AAV-RC-C08, an adeno-associated viral vector expressing the RS1 gene, as a potential therapeutic intervention for X-linked juvenile retinoschisis.
Methods :
A cohort of XLRS patients(n=5) was enrolled in a single-arm, open-label IIT trial. Fundus examinations, visual acuity, visual fields, electroretinography (ERG), and optical coherence tomography (OCT) were employed to monitor both safety and efficacy outcomes.
Results :
Following intravitreal injection of AAV-RC-C08, one-month analysis demonstrated a mean increase in ETDRS BCVA of +4.2 letters (p<0.001) and a mean enlargement in dynamic visual field of +22.4% (p<0.001) were observed. Sixty percent of subjects experienced a decrease in CST and structural ameliorations in the macular EZ intensity, with improvement in BCVA and b-wave amplitude. No serious ocular or systemic AEs were encountered.
Conclusions :
The IIT study highlights the promising potential of intravitreal AAV-RC-C08 - RS1 as a novel treatment approach for XLRS. Further research and clinical studies are warranted to validate these preliminary results.
This abstract was presented at the 2024 ARVO Annual Meeting, held in Seattle, WA, May 5-9, 2024.