Luxa生物技术Jeffrey Stern小组宣布他们开发出RPES-RPE移植治疗干性年龄相关性黄斑变性患者的安全性和耐受性：低剂量临床结果。相关论文发表在2025年9月16日出版的《细胞—干细胞》杂志上。

干性年龄相关性黄斑变性（AMD）视网膜色素上皮（RPE）细胞萎缩损害感光细胞功能，导致视力丧失。基于干细胞的RPE替代疗法旨在逆转疾病进展并恢复视力。RPESC-RPE- 4w是一种有丝分裂后成人RPE干细胞衍生的RPE （RPESC-RPE）祖细胞产品，在临床前研究中显示出一致的安全性和有效性。RPESC-RPE-4W的首次人体临床试验完成了低剂量队列1干预（NCT04627428）。6名受试者接受了5万个RPESC-RPE-4W细胞的视网膜下灌注。未观察到明显的炎症、肿瘤或与产品相关的严重不良事件。三名视力较差的A组受试者的最佳矫正视力在12个月时比基线平均提高了21.67个字母。三个视力较好的B组受试者在6个月时平均提高了3.0个字母。低剂量队列1的阳性安全性和耐受性结果使剂量增加到中剂量RPESC-RPE-4W治疗干性AMD。

附：英文原文

Title: Safety and tolerability of RPESC-RPE transplantation in patients with dry age-related macular degeneration: Low-dose clinical outcomes

Author: Rajesh C. Rao, Brigitte L. Arduini, Susan Borden, Dhruv Sareen, Clive Svendsen, Paul Lee, Charles Ryan, Shilpa Kodati, Caroline Nyaiburi, Keith Wolsieffer, Eric Oh, Shuna Park, Glenna Ford, Keith Dionne, Sally Temple, Jeffrey Stern

Issue&Volume: 2025-09-16

Abstract: Retinal pigment epithelium (RPE) cell atrophy in dry age-related macular degeneration (AMD) compromises photoreceptor cell function, leading to vision loss. Stem cell-based RPE replacement therapy aims to reverse disease progression and restore vision. RPESC-RPE-4W, a post-mitotic adult RPE stem cell-derived RPE (RPESC-RPE) progenitor cell product, exhibits consistent safety and efficacy in preclinical studies. The first-in-human clinical trial of RPESC-RPE-4W completed low-dose cohort 1 interventions (NCT04627428). Six subjects received a subretinal suspension of 50,000 RPESC-RPE-4W cells. No significant inflammation, tumor, or product-related serious adverse events were observed. Best-corrected visual acuity in the three worse-seeing group A subjects improved by an average of +21.67 letters from baseline at 12 months. Three better-seeing group B subjects improved by an average of +3.0 letters at 6 months. The positive safety and tolerability outcomes for low-dose cohort 1 enabled dose escalation to mid-dose RPESC-RPE-4W therapy for dry AMD.

DOI: 10.1016/j.stem.2025.08.012

Source: https://www.cell.com/cell-stem-cell/abstract/S1934-5909(25)00304-2