Pictured: Entrance to Pfizer's office in Belgium/iStock, Alexandros Michailidis

图为辉瑞驻比利时办事处入口/亚历山大·米切利迪斯（AlexandrosMichailidis）伊斯托克（iStock）

Pfizer on Tuesday announced that a patient died in the Phase II DAYLIGHT study, evaluating its investigational gene therapy fordadistrogene movaparvovec in Duchenne muscular dystrophy.

辉瑞公司周二宣布，一名患者在第二阶段日光研究中死亡，评估其对杜兴氏肌营养不良症的研究性基因治疗。

The patient has “passed away suddenly,” the pharma noted in a letter shared by nonprofit patient group Parent Project Muscular Dystrophy on Tuesday. Pfizer still does not have complete information regarding the incident and is currently working with the trial’s investigator to better understand the mortality..

该制药公司在周二由非营利患者组织父母项目肌肉萎缩症共享的一封信中指出，患者“突然去世”。辉瑞仍然没有关于该事件的完整信息，目前正在与试验研究人员合作，以更好地了解死亡率。。

The patient death has affected Pfizer’s Phase III CIFFREO cross-over trial in Duchenne muscular dystrophy (DMD). According to the company, it completed initial dosing for CIFFREO in 2023 but has decided to suspend the administration of fordadistrogene movaparvovec for the study’s cross-over portion.

患者死亡影响了辉瑞公司在杜兴氏肌营养不良症（DMD）中进行的III期CIFFREO交叉试验。据该公司称，该公司于2023年完成了CIFFREO的初始给药，但已决定暂停fordadistrogene movaparvovec用于该研究的交叉部分。

The trial will continue otherwise as scheduled..

审判将按计划继续进行。。

“The safety and well-being of the patients in our clinical trials remains our top priority,” Pfizer wrote in its letter, noting that it will work with regulatory authorities and an independent external data monitoring committee to assess the incident. The company will share more information with the broader medical and patient community “as soon as we can.”.

辉瑞在信中写道：“在我们的临床试验中，患者的安全和福祉仍然是我们的首要任务”，并指出将与监管机构和独立的外部数据监测委员会合作评估这起事件。该公司将“尽快”与更广泛的医疗和患者社区共享更多信息。

“On behalf of everyone at Pfizer, we extend our sympathies to his family, friends, and those closest to his care,” the pharma wrote in its letter.

该制药公司在信中写道：“我们代表辉瑞公司的所有人，向他的家人、朋友以及与他最亲近的人表示同情。”。

Tuesday’s announcement follows a previous patient death in a fordadistrogene movaparvovec study. In December 2021, Pfizer was also forced to pause screening and dosing in a Phase Ib DMD study after an unexpected patient death. At the time, the pharma did not specify whether the patient had been treated with the investigational gene therapy before the death..

星期二的声明是在福特·迪斯特罗·莫瓦帕沃维奇（fordadistrogene movaparvovec）的一项研究中，先前一名患者死亡之后发布的。2021年12月，辉瑞（Pfizer）在意外患者死亡后，也被迫停止了Ib期DMD研究中的筛查和给药。当时，该制药公司没有具体说明患者是否在死亡前接受了研究性基因治疗。。

In April 2022, the FDA lifted its clinical hold on the Investigational New Drug application for fordadistrogene movaparvovec, allowing Pfizer to open the first U.S. sites for CIFFREO.

2022年4月，美国食品和药物管理局解除了对fordadistrogene movaparvovec研究性新药申请的临床控制，允许辉瑞在美国开设首个CIFFREO网站。

DMD is a severe and genetic disease characterized by the progressive degeneration of muscles, leading to weakness and difficulty moving. It eventually affects vital organs such as the lungs, which could ultimately result in respiratory failure.

DMD是一种严重的遗传性疾病，其特征是肌肉进行性退化，导致虚弱和运动困难。它最终会影响肺部等重要器官，最终可能导致呼吸衰竭。

The disease mainly affects young boys between the ages of two and three years of age and is is caused by mutations to the gene encoding for the dystrophin protein, which is crucial for muscle function and stability. Pfizer’s fordadistrogene movaparvovec works by delivering a functional but shortened version of the dystrophin gene.

该疾病主要影响两岁至三岁的男孩，是由肌营养不良蛋白编码基因突变引起的，肌营养不良蛋白对肌肉功能和稳定性至关重要。辉瑞公司的fordadistrogene movaparvovec通过提供功能性但缩短的肌营养不良蛋白基因来工作。

The gene therapy uses an AAV9 capsid as its delivery mechanism, which is known to be able to target muscle tissue..

基因疗法使用AAV9衣壳作为其递送机制，已知其能够靶向肌肉组织。。

Tristan Manalac is an independent science writer based in Metro Manila, Philippines. Reach out to him on LinkedIn or email him at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

特里斯坦·马纳拉克（TristanManalac）是一位独立的科学作家，总部位于菲律宾马尼拉。在LinkedIn上联系他，或发电子邮件给他tristan@tristanmanalac.com或tristan.manalac@biospace.com.