由加泰罗尼亚生物工程研究所(IBEC)领导的研究团队开发了一类名为prosthe6的光激活小分子药物，能够帮助失明小鼠和斑马鱼恢复光感知和视觉导向行为1。该药物通过眼内注射或眼滴剂应用的方式给药，无需基因疗法、植入装置或特殊光源，为退行性失明患者提供了一条简便的治疗途径1。这项研究成果已发表在《美国化学学会期刊》上，并已获得专利保护1。
prosthe6的作用机制在于靶向ON-双极细胞中的mGlu6蛋白，从而有效替代丢失光感受器的功能1。其中两个最有前景的化合物prosthe6-12和prosthe6-15已通过眼内注射和眼滴剂应用均产生了效果，且恢复的视觉行为可在室内照度或阴天户外照度水平下出现，不需要高强度光源1。全球约200万人受到黄斑变性(AMD)和色素性视网膜炎(RP)等眼病影响，视力丧失每年造成的全球经济负担超过4000亿美元1。
研究团队负责人Pau Gorostiza表示："这些分子无法治愈失明，但在恢复视力方面效果显著，且采用简单、患者友好的方法"1。该研究基于10多年的研究基础，团队正与即将成立的衍生公司Eyelumina合作推进临床试验1。
Researchers at the Catalan Institute of Bioengineering (IBEC) have developed a class of light-activated small-molecule drugs called prosthe6 that successfully restored light perception and visually guided behavior in blind mice and zebrafish larvae 1. The compounds work by targeting the mGlu6 protein in ON-bipolar cells, effectively replacing the lost function of damaged photoreceptors 1. Two of the most promising variants, prosthe6-12 and prosthe6-15, demonstrated effectiveness when applied either through intraocular injection or as eye drops 1.
The treatment approach offers significant advantages over existing interventions, as it requires neither gene therapy, implanted devices, nor specialized high-intensity light sources 1. The restored visual responses occurred at indoor lighting levels or under overcast outdoor conditions, making the therapy practical for everyday use 1. According to Pau Gorostiza, the lead researcher, "These molecules cannot cure blindness, but they are very effective at recovering sight and use a simple, patient-friendly approach" 1.
The findings address a substantial medical need, as approximately two million people globally suffer from degenerative eye diseases such as age-related macular degeneration and retinitis pigmentosa, with vision loss imposing an annual economic burden exceeding $400 billion worldwide 1. The research, building on more than a decade of foundational work and protected by patents, was published in the Journal of the American Chemical Society 1. The research team is now collaborating with a newly established spinoff company called Eyelumina to advance the therapy toward clinical trials 1.
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