智通财经APP获悉，日前，美国FDA批准Alnylam公司(ALNY.US)的siRNA疗法Amvuttra(vutrisiran)扩展适应症，治疗ATTR-CM成人患者。Vutrisiran是一款皮下给药的siRNA疗法，基于Alnylam的增强稳定化学(ESC)-GalNAc偶联递送平台设计，增加了效力和代谢稳定性。在3期临床试验中，它将患者的全因死亡率和复发性心血管事件的风险降低了28%(HR=0.72，95% CI：0.56-0.93，P=0.01)。新闻稿指出，vutrisiran是可同时治疗ATTR淀粉样变性患者的多发性神经病(polyneuropathy)和心肌病的首款FDA批准疗法。
GalNAc技术是推进siRNA疗法肝脏递送的突破性技术。它通过将与肝脏细胞表面高度表达的唾液酸糖蛋白受体(ASGPR)结合的GalNAc分子和siRNA分子偶联，促进siRNA分子被肝细胞吸收，增强siRNA分子的肝脏特异性递送。
基于GalNAc偶联的递送技术问世以来，已经在siRNA药物开发领域得到广泛应用。
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