Abstract
Presentation Description :
Age-related macular degeneration (AMD) is one of the leading causes of vision loss among the elderly population in the Western world. The condition occurs due to the degeneration of the retinal pigment epithelium (RPE) monolayer that supports photoreceptors in the eye. To address this, we have developed an autologous cell replacement therapy using induced pluripotent stem cells (iPSC) for treating dry AMD patients. The process involves reprogramming patients' blood cells into iPSCs and then differentiating them into RPE cells using a protocol developed in our lab. The RPE cells are then matured on a biodegradable polylactic co-glycolic acid (PLGA) scaffold for five weeks. Quality control assays are performed to confirm the iPSC-RPE patch's purity, maturity, and functionality. Pre-clinical studies in rats and pigs have demonstrated the safety and efficacy of the iPSC-RPE patch. Transplanted immune-compromised rats showed no signs of tumor formation after nine months, confirming the safety profile. In pig eyes, we laser-injured the RPE monolayer in the visual streak and transplanted the patch after 48 hours. The patch integration was confirmed using Optical Coherence Tomography (OCT). A multi-focal electroretinogram (ERG) showed that the electric response of the retinal layers was much higher with the implant than in the lasered area without it. We started a phase I/IIa trial using an autologous iPSC-RPE patch to treat AMD. The ongoing trial will assess the iPSC-RPE patch's safety, feasibility, and integration in 12 AMD patients.
This abstract was presented at the 2024 ARVO Annual Meeting, held in Seattle, WA, May 5-9, 2024.