miR-155抑制剂治疗T细胞淋巴瘤,获得美国FDA的孤儿药物指定

2020-07-24 MedSci原创 MedSci原创

microRNA-155的过表达与多种T细胞淋巴瘤和其他血液癌的预后不良有关。

生物制药公司miRagen Therapeutics宣布,美国食品与药品管理局(FDA)授予其cobomarsen孤儿药称号,用于治疗T细胞淋巴瘤

See the source image

Cobomarsen是miRagen开发的miR-155抑制剂,在多种血液癌细胞中均发现miR-155的含量异常增高。目前Cobomarsen正在两项临床研究中进行开发,包括皮肤T细胞淋巴瘤(CTCL)的2期临床试验和成人T-细胞白血病/淋巴瘤(ATLL)的1期临床试验。

miRagen总裁兼首席执行官William S表示:“这是cobomarsen发展的重要里程碑,我们相信cobomarsen有潜力用于治疗其他miR-155升高的癌症类型患者的潜力。 ”

T细胞淋巴瘤(如ATLL和CTCL等)在美国,合起来占不到非霍奇金淋巴瘤的15%。T细胞淋巴瘤的类型很多,但都很少见。microRNA-155的过表达与多种T细胞淋巴瘤和其他血液癌的预后不良有关。

原始出处:

https://www.firstwordpharma.com/node/1743234?tsid=4

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    2021-02-22 smallant2002
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    2020-07-24 146a476fm02暂无昵称

    学习

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