A novel gene-editing medicine is now authorized in the United Kingdom for treating two inherited blood disorders, a landmark decision that marks the first approval anywhere in the world for a therapy based on the CRISPR technology.

英国现已授权一种新型基因编辑药物治疗两种遗传性血液疾病，这是一项具有里程碑意义的决定，标志着世界上任何地方首次批准基于CRISPR技术的治疗。

The Medicines and Healthcare products Regulatory Agency (MHRA) decision covers the treatment of patients 12 and older who have sickle cell disease or transfusion-dependent beta thalassemia. The therapy, known in development as exagamglogene autotemcel, or exa-cel, will be commercialized by Vertex Pharmaceuticals and CRISPR Therapeutics under the brand name Casgevy.

药品和保健品监管机构（MHRA）的决定涵盖了患有镰状细胞病或输血依赖性β地中海贫血的12岁及以上患者的治疗。该疗法在开发中称为exagamglogene autotemcel或exa-cel，将由Vertex Pharmaceuticals和CRISPR Therapeutics以商品名Casgevy商业化。

.

.

Both sickle cell disease and beta thalassemia stem from errors in the genes that code for hemoglobin, the oxygen-carrying proteins found on red blood cells. In sickle cell disease, the abnormal hemoglobin causes red blood cells to take on a crescent shape that causes ongoing complications such as severe pain, life threatening infections, and anemia.

镰状细胞病和β地中海贫血都源于编码血红蛋白的基因错误，血红蛋白是红细胞上发现的携氧蛋白。在镰状细胞病中，异常的血红蛋白导致红细胞呈新月形，导致持续的并发症，例如剧烈疼痛，危及生命的感染和贫血。

The few drugs available for sickle cell disease are chronic therapies..

少数可用于镰状细胞病的药物是慢性疗法。。

The low levels of hemoglobin in beta thalassemia can lead to severe anemia. Patients require frequent blood transfusions, which introduces other complication risks. Stem cell transplants offer a potentially curative treatment option for both sickle cell disease and beta thalassemia. But the procedure requires a matched donor and it comes with the risk of rejection..

β地中海贫血中血红蛋白水平低可导致严重贫血。患者需要频繁输血，这会带来其他并发症风险。干细胞移植为镰状细胞病和β地中海贫血提供了潜在的治疗选择。但是这个程序需要一个匹配的捐助者，并且存在拒绝的风险。。

Casgevy is made by collecting a patient’s stem cells from the bone marrow. In a lab, CRISPR is used to edit a gene in those cells to produce high levels of fetal hemoglobin. Before these modified stem cells are administered, a conditioning regimen is required to kill the remaining cells in a patient that are making faulty hemoglobin.

Casgevy是通过从骨髓中收集患者的干细胞制成的。在实验室中，CRISPR用于编辑这些细胞中的基因以产生高水平的胎儿血红蛋白。在施用这些修饰的干细胞之前，需要预处理方案来杀死患者中产生有缺陷的血红蛋白的剩余细胞。

Infused into the patient, the modified stem cells make their way to the bone marrow. Patients may need to remain hospitalized for at least a month while those cells take up residence in the bone marrow and start producing red blood cells that incorporate fetal hemoglobin. Casgevy is intended to be a one-time treatment..

注入患者体内，修饰的干细胞进入骨髓。患者可能需要保持住院至少一个月，而这些细胞占据骨髓并开始产生含有胎儿血红蛋白的红细胞。Casgevy旨在成为一次性治疗。。

The MHRA decision is based on results from a trial in sickle cell disease that enrolled 45 patients. Of those, only 29 have been in the study long enough for the primary efficacy interim analysis. All but one of those patients were free of severe pain crises for at least 12 months after treatment.

MHRA的决定是基于45名患者的镰状细胞病试验结果。其中，只有29人在研究中的时间足以进行主要疗效中期分析。治疗后至少12个月，除一名患者外，所有患者均无严重疼痛危机。

In the beta thalassemia trial, 42 of 54 patients have been in the clinical trial long enough to be eligible for the primary efficacy interim analysis. Of those eligible patients, 39 did not need a red blood cell transfusion for at least 12 months after treatment with Casgevy. The remaining three patients showed a more than 70% reduction in the need for transfusions..

在β地中海贫血试验中，54名患者中有42名已经进行了足够长的临床试验，有资格进行主要疗效中期分析。在这些符合条件的患者中，39例在用Casgevy治疗后至少12个月不需要输注红细胞。其余三名患者的输血需求减少了70%以上。。

Side effects reported in the trials included nausea, fatigue, fever, and a high risk of infections. Those problems are similar to those for other autologous stem cell transplant procedures. No serious adverse effects were reported from the studies. The MHRA decision is a conditional marketing authorization valid for one year.

试验报告的副作用包括恶心，疲劳，发烧和高感染风险。这些问题与其他自体干细胞移植手术相似。研究报告没有严重的不良反应。MHRA决定是有效期为一年的有条件上市许可。

Vertex and CRISPR Therapeutics are required to conduct additional post-marketing clinical research. The authorization for Casgevy may be renewed annually as long as ongoing review of real-world data and post-marketing studies continue to support use of the therapy..

Vertex和CRISPR Therapeutics需要进行额外的上市后临床研究。只要对现实世界数据和上市后研究的持续审查继续支持该疗法的使用，Casgevy的授权可以每年更新。。

“Today is a historic day in science and medicine: this authorization of Casgevy in Great Britain is the first regulatory authorization of a CRISPR-based therapy in the world,” Vertex CEO Reshma Kewalramani said in a prepared statement.

Vertex首席执行官Reshma Kewalramani在准备好的声明中说：“今天是科学和医学的一个历史悠久的日子：英国Casgevy的授权是世界上第一个基于CRISPR疗法的监管授权。

According to Vertex and CRISPR Therapeutics, the U.K. has an estimated 2,000 patients eligible for treatment with Casgevy. The MHRA said more details about the product’s label will be published on its website within a week.

根据Vertex和CRISPR Therapeutics的说法，英国估计有2000名患者有资格接受Casgevy治疗。MHRA表示，有关产品标签的更多详细信息将在一周内发布在其网站上。

U.K. approval of Casgevy comes ahead of upcoming regulatory decisions in the U.S. Last month, an FDA advisory committee gave its backing to the CRISPR-editing therapy. Casgevy faces a Dec. 8 target date for an FDA decision in sickle cell disease. In beta thalassemia, the therapy should receive an FDA decision by March 30, 2024..

U、 K.Casgevy的批准在美国即将做出监管决定之前。上个月，FDA咨询委员会支持CRISPR编辑疗法。Casgevy面临FDA决定镰状细胞病的12月8日目标日期。在β地中海贫血中，治疗应在2024年3月30日之前获得FDA的决定。。

In a note sent to investors, William Blair analyst Tim Lugo said Casgevy’s approval is an important milestone for people who have both blood disorders. He added that the regulatory decision is key validation for CRISPR Therapeutics’ deep pipeline of therapies based on CRISPR gene-editing. Using a model that assumes a price of $1.6 million for beta thalassemia and $1.5 million for sickle cell disease in Europe, William Blair projects peak sales of Casgevy in both indications topping $1.6 billion in Europe in the early 2030s..

在发给投资者的一份说明中，威廉·布莱尔分析师蒂姆·卢戈（Tim Lugo）表示，卡斯格维（Casgevy）的批准对于同时患有血液疾病的人来说是一个重要的里程碑。他补充说，监管决策是CRISPR Therapeutics基于CRISPR基因编辑的深层治疗流程的关键验证。威廉·布莱尔（William Blair）使用一种假设β地中海贫血的价格为160万美元，欧洲镰状细胞病的价格为150万美元的模型，预测这两种适应症在20世纪30年代初的Casgevy销售额都将超过16亿美元。。

A one-time treatment for beta thalassemia is already available from Bluebird Bio. Last year, Bluebird won FDA approval for Zynteglo, marking the first gene therapy approval in the rare blood disorder. That therapy carries a $2.8 million wholesale price. A different Bluebird gene therapy developed for sickle cell disease is expected to receive an FDA decision by Dec.

蓝鸟生物已经有了一次性的β地中海贫血治疗方法。去年，蓝鸟获得了Zynteglo的FDA批准，标志着罕见血液病的第一个基因治疗批准。该疗法的批发价格为280万美元。为镰状细胞病开发的另一种蓝鸟基因疗法预计将在12月前获得FDA的决定。

20..

20..

Image: Meletios Verras, Getty Images

图片：Meletios Verras，Getty图片

Promoted

促进

Deploying a Digital Flywheel to Enable a Hyperpersonal Care Experience

部署数字轮以实现超级个人护理体验

Webinar will spotlight expert panelists from Praia Health and Providence, interviewed by AVIA, to discuss the benefits of adopting a hyperpersonal care model for patients, challenges that health systems face providing this care, and how they can overcome them.

网络研讨会将聚焦来自Praia Health和Providence的专家小组成员，由AVIA采访，讨论为患者采用超个人护理模式的好处，卫生系统提供这种护理所面临的挑战，以及他们如何克服这些问题。

AVIA and MedCity News

AVIA和MedCity新闻

Promoted

促进

OmniLife Health’s Clinical Workflow Solution Focuses on Complex Care

OmniLife Health的临床工作流程解决方案专注于复杂护理

OmniLife Health’s initial focus with its complex care coordination platform is organ transplant care.

OmniLife Health最初关注其复杂的护理协调平台是器官移植护理。

Stephanie Baum

斯蒂芬妮·鲍姆

Deploying a Digital Flywheel to Enable a Hyperpersonal Care Experience

部署数字轮以实现超级个人护理体验

Webinar will spotlight expert panelists from Praia Health and Providence, interviewed by AVIA, to discuss the benefits of adopting a hyperpersonal care model for patients, challenges that health systems face providing this care, and how they can overcome them.

网络研讨会将聚焦来自Praia Health和Providence的专家小组成员，由AVIA采访，讨论为患者采用超个人护理模式的好处，卫生系统提供这种护理所面临的挑战，以及他们如何克服这些问题。

AVIA and MedCity News

AVIA和MedCity新闻

OmniLife Health’s Clinical Workflow Solution Focuses on Complex Care

OmniLife Health的临床工作流程解决方案专注于复杂护理

OmniLife Health’s initial focus with its complex care coordination platform is organ transplant care.

OmniLife Health最初关注其复杂的护理协调平台是器官移植护理。

Stephanie Baum

斯蒂芬妮·鲍姆