AX-0810 is a unique therapeutic approach leading to a potentially disease modifying therapy by targeting the NTCP channel which is responsible for majority of bile acid re-uptake in liver cells. NTCP: Na-taurocholate cotransporting polypeptide. Bile acids help the digestion of fats and absorption of certain vitamins.

2024年5月9日 · ProQR is developing its AX-0810 program targeting NTCP for Cholestatic Diseases and plans to advance the program to the clinic in late 2024/early 2025. ProQR also expects the following Axiomer pipeline program milestones in 2024:

2023年3月29日 · ProQR today announced AX-0810 for Cholestatic Diseases targeting NTCP and AX-1412 for Cardiovascular Disease targeting B4GALT1 as initial pipeline programs.

ProQR专有的Axiomer®RNA编辑平台已在多个临床前体外、类器官和体内模型中证明其活性；在啮齿动物和非人类灵长类动物中观察到高达50%的编辑率。

2024年5月8日 · ProQR科学家首次在ADAR RNA编辑领域报告了使用Axiomer™RNA编辑寡核苷酸在体内靶标参与证明（RNA编辑）导致NHPs中生物标志物发生有意义的变化。 该公司AX-0810计划针对NTCP治疗胆汁淤积性疾病的临床概念验证于2024年5月9日在荷兰莱顿和马萨诸塞州剑桥美国东部夏令时上午8:00举行的ASGCT年度会议管理网络研讨会（2024年5月8日）（环球通讯社）——ProQR Therapeutics NV（纳斯达克：PRQR）（ProQR），一家致力于通过转化RNA疗 …

ProQR is developing its AX-0810 program targeting NTCP for Cholestatic Diseases and plans to advance the program to the clinic in late 2024/early 2025. ProQR also expects the following Axiomer pipeline program milestones in 2024:

In October, ProQR presented at the 20th Annual Meeting of the Oligonucleotide Therapeutics Society in Montreal, highlighting its Axiomer technology platform and AX-0810, the Company’s program targeting NTCP for cholestatic diseases.

ProQR Therapeutics is advancing its Axiomer ADAR-mediated RNA editing pipeline, aiming to file a Clinical Trial Application for its lead program AX-0810, targeting NTCP for cholestatic...

2024年5月8日 · By specifically affecting the main transporter for bile acids reuptake from the portal vein circulation to the liver, called NTCP (Na-taurocholate transporting polypeptide, SLC10A1 gene), the AX...

2025年3月17日 · 人类遗传学数据显示，NTCP的一种变体可以限制胆汁酸的摄取，这进一步支持了ProQR方法的潜在疗效。 分析师还注意到，来自人源化小鼠和非人灵长类动物（NHPs）的有希望的临床前数据显示了AX-0810显著的靶点结合能力和潜在的安全性特征。