FDA批准补充 治疗疗法的基因治疗试验 对失明致盲眼病的试验。 FDA approves Complement Therapeutics' gene therapy trial for blindness-causing eye disease.

补充疗法已经得到林业发展局的批准,开始对CTx001进行Opti-GAIN第一阶段/第二阶段试验。 CTx001是一种针对与年龄有关的肌肉畸形导致的地理萎缩(GA)的基因疗法,这是不可逆转的视力丧失的主要原因,影响到约150万美国人。 Complement Therapeutics has received FDA approval to launch the Opti-GAIN Phase I/II trial for CTx001, a gene therapy targeting geographic atrophy (GA) due to age-related macular degeneration, a leading cause of irreversible vision loss affecting about 1.5 million Americans. 该疗法使用亚丁诺相关病毒(AAV)提供经修改的补充受体1蛋白质,以调节免疫系统的补充路径,从而可能减缓疾病蔓延。 The therapy uses adeno-associated virus (AAV) to deliver a modified Complement Receptor 1 protein to regulate the immune system’s complement pathway, potentially slowing disease progression. 第一次人类试验预计将于2026年初开始进行,它将根据i-GAIN自然历史研究的数据,评估国际视网膜中心75名病人的安全性、可耐性和早期有效性。 The first-in-human trial, expected to begin dosing in early 2026, will evaluate safety, tolerability, and early effectiveness in 75 patients across international retinal centers, informed by data from the i-GAIN natural history study. 该公司是来自曼彻斯特大学的一家分公司,它把这一里程碑看作是朝着对目前选择有限的一个状况进行一次性治疗的可能性迈出的关键一步。 The company, a spinout from the University of Manchester, views the milestone as a key step toward a potential one-time treatment for a condition with limited current options.