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ProQR宣布寡核苷酸药物QR-421a进入临床试验阶段以治疗Usher综合征

2018-12-06 MedSci MedSci原创

QR-421a寡核苷酸药物Usher综合征
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ProQR是一家致力于通过的变革性RNA药物治疗遗传性罕见疾病的生物技术公司，近日宣布美国食品和药物管理局（FDA）已批准其寡核苷酸药物QR-421a的研究性新药申请（IND）。

ProQR是一家致力于通过的变革性RNA药物治疗遗传性罕见疾病的生物技术公司，近日宣布美国食品和药物管理局（FDA）已批准其寡核苷酸药物QR-421a的研究性新药申请（IND）。QR-421a是一种基于RNA的寡核苷酸药物，旨在治疗USH2A基因13号外显子突变引起的Usher综合征和非综合征性视网膜色素变性（RP）。ProQR首席执行官Daniel A. de Boer说：“QR-421a在斑马鱼模型中表现出了很好的活性，我们希望QR-421a能够对Usher综合症患者产生积极影响”。ProQR计划在未来几个月开始招募患者参加I/II期STELLAR试验，初步数据预计在2019年中期获得。

Usher综合症又称遗传性耳聋-色素性视网膜炎综合征，视网膜色素变性-感音神经性耳聋综合征，聋哑伴视网膜色素变性综合征等。是以先天性感音神经性聋、渐进性视网膜色素变性而致视野缩小、视力障碍为主要表现的一种常染色体隐性遗传性疾病，具有遗传异质性。


原始出处：

http://www.firstwordpharma.com/node/1609088#axzz5Yod0v3he

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    2019-02-23 heli0118

    #寡核苷酸#

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    2019-03-12 xsm918

    #核苷酸#

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    2018-12-08 zhouqu_8

    #综合征#

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