School of Pharmaceutical Sciences, Nanjing Tech University, Nanjing 211816, China.
Key Laboratory of Advanced Drug Delivery Systems of Zhejiang Province, National Key Laboratory of Advanced Drug Delivery and Release Systems, College of Pharmaceutical Sciences, Zhejiang University, Hangzhou 310058, China.
Proc Natl Acad Sci U S A. 2024 Mar 12;121(11):e2314349121. doi: 10.1073/pnas.2314349121. Epub 2024 Mar 5.
Thrombosis, induced by abnormal coagulation or fibrinolytic systems, is the most common pathology associated with many life-threatening cardio-cerebrovascular diseases. However, first-line anticoagulant drugs suffer from rapid drug elimination and risk of hemorrhagic complications. Here, we developed an in situ formed depot of elastin-like polypeptide (ELP)-hirudin fusion protein with a prodrug-like feature for long-term antithrombotic therapy. Highly secretory expression of the fusion protein was achieved with the assistance of the Ffu312 tag. Integration of hirudin, ELP, and responsive moiety can customize fusion proteins with properties of adjustable in vivo retention and controllable recovery of drug bioactivity. After subcutaneous injection, the fusion protein can form a reservoir through temperature-induced coacervation of ELP and slowly diffuse into the blood circulation. The biological activity of hirudin is shielded due to the N-terminal modification, while the activated key proteases upon thrombus occurrence trigger the cleavage of fusion protein together with the release of hirudin, which has antithrombotic activity to counteract thrombosis. We substantiated that the optimized fusion protein produced long-term antithrombotic effects without the risk of bleeding in multiple animal thrombosis models.
血栓形成是由异常的凝血或纤溶系统引起的,是与许多危及生命的心脑血管疾病最常见的病理。然而,一线抗凝药物存在药物消除迅速和出血并发症的风险。在这里,我们开发了一种具有前药特征的弹性蛋白样多肽(ELP)-水蛭素融合蛋白的原位储库,用于长期抗血栓治疗。融合蛋白的高度分泌表达是在 Ffu312 标签的辅助下实现的。水蛭素、ELP 和响应部分的整合可以定制融合蛋白,具有可调节体内保留时间和可控制药物生物活性恢复的特性。皮下注射后,融合蛋白可通过 ELP 的温度诱导凝聚形成储库,并缓慢扩散到血液循环中。由于 N 端修饰,水蛭素的生物活性被屏蔽,而血栓形成时激活的关键蛋白酶会触发融合蛋白的切割,同时释放具有抗血栓活性的水蛭素,以对抗血栓形成。我们证实,优化后的融合蛋白在多种动物血栓模型中产生了长期的抗血栓作用,而没有出血的风险。