Dive Brief:

概要：

A patient who received an experimental gene therapy developed by Rocket Pharmaceuticals has died following treatment, the company

一名接受 Rocket Pharmaceuticals 开发的实验性基因疗法的患者在治疗后死亡，该公司

disclosed Tuesday

周二披露

.

。

According to Rocket, the patient experienced what’s known as capillary leak syndrome, where plasma and proteins seep from blood vessels into surrounding tissue, and later died from an acute systemic infection.

据罗克特称，该患者经历了所谓的毛细血管渗漏综合征，即血浆和蛋白质从血管渗入周围组织，随后死于急性全身感染。

The Food and Drug Administration ordered the study’s halt Friday, while Rocket works with clinical trial monitors and other experts to investigate the cause. The company said it is focusing on a “novel immune suppression agent” it recently added to a pre-treatment preparatory regimen.

美国食品药品管理局周五下令暂停这项研究，与此同时，Rocket 正在与临床试验监察员和其他专家合作调查原因。该公司表示，他们正在关注一种“新型免疫抑制剂”，这种药物是他们最近添加到预处理方案中的。

Dive Insight:

深度洞察：

Rocket had proactively modified its pre-treatment regimen in attempt to reduce the risk of an immune response known as “complement activation,” which it previously saw evidence of in the Phase 2 trial.

Rocket 已主动修改了其预处理方案，试图降低称为“补体激活”的免疫反应风险，此前在二期试验中曾发现过这一反应的证据。

Two patients received the immune suppression agent, company CEO Gaurav Shah explained on a conference call Tuesday morning. Rocket was able to reduce the treatment course the second patient received after seeing early signs of capillary leak syndrome in the first.

公司首席执行官高拉夫·沙阿在周二上午的电话会议上解释说，两名患者接受了免疫抑制剂。在第一名患者出现毛细血管渗漏综合征的早期迹象后，火箭公司能够减少第二名患者的治疗过程。

The company is now exploring whether the additional drug it used — a so-called C3 inhibitor — “may have influenced immune responses in an unexpected or paradoxical way,” said Shah, who noted that the only difference in protocol for these two patients was the change in preparatory regimen.

该公司现在正在研究其所使用的额外药物——一种所谓的C3抑制剂——“是否可能以某种意外或矛盾的方式影响了免疫反应”，Shah指出，这两名患者的方案唯一不同之处在于准备方案的改变。

Shah said Rocket is confident there remains a path forward for its gene therapy, which is designed to treat a rare inherited disorder known as

沙阿表示，Rocket 公司对其基因疗法仍充满信心，认为仍有前进的道路，该疗法旨在治疗一种称为罕见遗传病的疾病。

Danon disease

丹农病

. The condition results in the accumulation of sugars and certain cellular components in cardiac muscle, leading to heart failure and early death.

这种情况会导致糖分和某些细胞成分在心肌中积累，从而引发心力衰竭和早逝。

“This is a deeply tragic loss. We are committed to fully understanding the circumstances surrounding it objectively and neutrally,” said Shah. “We are also immensely grateful to the family for their contribution to this important clinical research and their commitment to help advance science for the broader Danon community.”.

“这是一个非常悲惨的损失。我们承诺客观和中立地全面了解与其相关的具体情况，”沙阿说。“我们也非常感谢这家人对该项重要临床研究的贡献，以及他们帮助推动丹侬病群体科学发展所作的承诺。”

Now, however, Rocket must prove to the FDA, researchers and its investors that its treatment, dubbed RP-A501, remains safe. It’s a position

然而，现在 Rocket 必须向 FDA、研究人员及其投资者证明其名为 RP-A501 的治疗方案仍然安全。这是一个

many

许多

other

其他

gene

基因

therapy

治疗

developers

开发者

have found themselves in after unexpected patient deaths in trials.

在试验中发生意外患者死亡后，他们发现自己处于（某种境地）。

So far, investors appear skeptical Rocket will have an easy answer. Shares in the company slumped by more than 60% in Tuesday morning trading, erasing hundreds of millions of dollars from the company’s market capitalization.

到目前为止，投资者似乎对罗克特会有简单的解决方案持怀疑态度。该公司股价在周二早盘交易中暴跌超过60%，使公司市值蒸发了数亿美元。

Rocket emphasized that the introduction of the new immune agent was limited only to the Danon program, and not used in the company’s

洛克特强调，新的免疫剂的引入仅限于丹农计划，而并未用于公司的

other pipeline projects

其他管道项目

. The company said it can’t predict when it may be able to resume the Phase 2 trial, which was expected to enroll 12 patients.

公司表示无法预测何时能够恢复预计将招募12名患者的第二阶段试验。

RP-A501 is built around a version of the gene, called LAMP2B, that’s mutated in people with Danon. The replacement DNA is encased in an engineered viral shell known as an adenovirus-associated serotype 9 capsid. The therapy is infused, and is meant to be a one-time treatment.

RP-A501围绕一种名为LAMP2B的基因版本构建，该基因在Danon病患者中发生突变。替代DNA被包裹在一种工程化的病毒外壳中，称为腺相关病毒血清型9衣壳。这种疗法通过输注进行，旨在成为一次性治疗。