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Uniqure's Hemophilia B Gene Therapy Unlikely To Be Cause Of Liver Cancer Reported In One Patient

Uniqure's Hemophilia B Gene Therapy Unlikely To Be Cause Of Liver Cancer Reported In One Patient

UnQure血友病B基因疗法不太可能导致肝癌1例报告
Benzinga Real-time News ·  2021/03/29 21:26

Uniqure NV (NASDAQ: QURE) has reported results of a comprehensive investigation into the case of hepatocellular carcinoma (HCC) diagnosed in one patient in the HOPE-B pivotal trial of etranacogene dezaparvovec, its hemophilia B gene therapy candidate.

UnQure NV(纳斯达克:Qure)有报告了一项全面调查的结果在血友病B基因治疗候选药物etranacogene dezparvovec的HOPE-B关键试验中,一名患者被诊断为肝细胞癌(HCC)。

What Happened: The trial was put on clinical last year in December, following the submission of a safety report in mid-December relating to a possibly related severe adverse event associated with a preliminary diagnosis of hepatocellular carcinoma in one patient.

发生的事情:这个试验于去年12月进行临床试验。在12月中旬提交了一份安全报告后,该报告涉及一名患者初步诊断为肝细胞癌可能相关的严重不良事件。

The results of the investigation found that it is implausible the HCC was caused by etranacogene dezaparvovec.

调查结果发现,肝细胞癌不可能是由四叠纪去扎帕沃韦克引起的。

Multiple analyses conducted by an independent laboratory and reviewed by external experts in the field show that AAV vector integration in the patient's tissue sample was extremely rare and accounted for 0.027% of the cells in the sample.

由独立实验室进行并经该领域外部专家审查的多项分析表明,患者组织样本中的AAV载体整合极为罕见,占样本细胞的0.027%。

Why It Matters: The whole-genome sequencing of the tumor confirmed that the patient had several genetic mutations characteristic of HCC and independent of vector integration.

重要的原因:肿瘤的全基因组测序证实,患者有几个肝癌特有的基因突变,与载体整合无关。

Finally, gene expression analysis of the tumor and adjacent tissue suggested a precancerous state in the liver consistent with several risk factors that predispose this patient to HCC.

最后,肿瘤和邻近组织的基因表达分析表明,肝脏处于癌前状态,这与该患者易患肝癌的几个危险因素一致。

The external lab analyzed more than 220,000 cells from the tissue sample and identified 60 cells with random integration events that have no known association with the development of HCC," stated Ricardo Dolmetsch, uniQure president of research and development.

外部实验室分析了组织样本中的22万多个细胞,鉴定出60个存在随机整合事件的细胞,这些细胞与HCC的发展没有已知的联系,“uniQure研究和开发总裁里卡多·多尔梅奇(Ricardo Dolmetsch)说。

The company has shared the data with the FDA and is prepared to have further communications regarding the clinical hold status in the second quarter of 2021.

该公司已经与FDA分享了数据,并准备在2021年第二季度就临床持有状态进行进一步沟通。

All patients in uniQure's hemophilia B gene therapy program, including the 54 patients in HOPE-B, have now had abdominal ultrasounds performed one year after dosing. Each will continue to be monitored by their care teams.

UnQure血友病B基因治疗计划中的所有患者,包括HOPE-B中的54名患者,现在都在服药一年后接受了腹部超声波检查。每名患者都将继续接受其护理团队的监测。

No other cases of HCC have been reported in uniQure clinical trials conducted in more than 100 patients in hemophilia B and other indications, with some patients dosed more than ten years ago.

在对100多名血友病B患者和其他适应症患者进行的UnQure临床试验中,没有其他肝癌病例的报告,有些患者在十多年前就服用了药物。

Price Action: QURE shares are trading 9.47% higher at $32.01 in the premarket session on the last check Monday.

价格行动:在周一的最后一次检查中,Qure股价在盘前交易时段上涨9.47%,至32.01美元。

译文内容由第三方软件翻译。


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