In a landmark announcement for the CRISPR field and the sickle cell disease (SCD) community, the U.K. authorities have granted approval to Casgevy, the CRISPR-Cas9 therapy formerly known as exa-cel developed by Vertex Pharmaceuticals and CRISPR Therapeutics.

在CRISPR领域和镰状细胞病（SCD）社区的一项具有里程碑意义的公告中，英国当局已批准Casgevy，即Vertex Pharmaceuticals和CRISPR Therapeutics开发的CRISPR-Cas9疗法，以前称为exa-cel。

The U.S. Food and Drug Administration (FDA) is expected to follow suit in the next three weeks, having issued a decision date of December 8. A further decision on Bluebird bio’s related SCD therapy should come later that month. The U.K. approval was announced by the Medicines and Healthcare products Regulatory agency..

美国食品和药物管理局（FDA）预计将在未来三周内提起诉讼，发布日期为12月8日。关于蓝鸟生物相关SCD治疗的进一步决定应该在该月晚些时候做出。英国的批准由药品和保健品监管机构宣布。。

CRISPR pioneers, scientists, and commentators took to the X social media platform to voice their delight at the news.

CRISPR先驱，科学家和评论员来到X社交媒体平台，表达他们对新闻的高兴。

The CEO of CRISPR Therapeutics, Sam Kulkarni, PhD, called this a “momentous day” and said he was “proud of the team and the investigators and patients who were part of the trials. And excited for patients.” Co-founded by Nobel laureate Emmanuelle Charpentier, CRISPR Therapeutics initiated the SCD CRISPR therapy, eventually agreeing to partner with Vertex, which has since assumed the role of the lead sponsor..

CRISPR Therapeutics的首席执行官Sam Kulkarni博士称这是一个“重大的日子”，并说他“为团队以及参与试验的研究人员和患者感到骄傲，并为患者感到兴奋。”由诺贝尔奖获得者Emmanuelle Charpentier共同创立，CRISPR Therapeutics启动了SCD CRISPR治疗，最终同意与Vertex合作，此后担任首席发起人的角色。。

Patrick Hsu, PhD (Arc Institute), one of the early developers of CRISPR genome editing working with Feng Zhang, PhD, called it “a huge victory for biotechnology, patients, and humanity.”

Patrick Hsu博士（Arc研究所）是与Feng Zhang博士合作的CRISPR基因组编辑的早期开发人员之一，他称之为“生物技术，患者和人类的巨大胜利”

The Editor-in-Chief of The CRISPR Journal, Rodolphe Barrangou, PhD, (North Carolina State University), said the news was “very momentous for the field, ushering a new era of medicines and highlighting the momentum with regulatory agencies in a global context. A milestone for all.”

CRISPR期刊主编Rodolphe Barrangou博士（北卡罗来纳州立大学）表示，这一消息“对该领域来说非常重要，开创了药物新时代，突出了全球监管机构的势头。所有人的里程碑。”

It was “remarkable to see that our discoveries over 15 years ago would enable such tremendous advances for [SCD] and thalassemia!!!” said Vijay Sankaran, MD, (Harvard Medical School), who led one of the pivotal genome wide association studies in 2008, published in Science, that pinpointed BCL11A as a critical regulator of fetal hemoglobin (HbF) levels.

“值得注意的是，我们15年前的发现将为[SCD]和地中海贫血带来如此巨大的进步！”博士（哈佛医学院）Vijay Sankaran说，他领导了一项关键的全基因组关联研究。2008年发表在《科学》杂志上，该研究将BCL11A确定为胎儿血红蛋白（HbF）水平的关键调节因子。

The last sentence of that report stated: “As a stage-specific component involved in the silencing of γ-globin expression, BCL11A, therefore, emerges as a new therapeutic target for reactivation of HbF in [SCD] and thalassemia.”.

该报告的最后一句话指出：“作为参与γ-珠蛋白表达沉默的阶段特异性成分，BCL11A因此成为[SCD]和地中海贫血中HbF活化的新治疗靶标。”。

Physician-scientist Jay Bradner, MD, who recently stepped down as president of the Novartis Institutes for Biomedical Research, said the “first approval of a CRISPR therapeutic is a milestone for the technology innovators, but also for the biologists who directed the machinery.” He offered congratulations to Sankaran and his former boss, Stuart Orkin, MD, at Boston Children’s Hospital..

最近当选为诺华生物医学研究所所长的医师科学家杰伊·布拉德纳（Jay Bradner）表示，“CRISPR治疗药物的首次批准是技术创新者的里程碑，也是指导该机器的生物学家的里程碑。”他向波士顿儿童医院的Sankaran及其前任老板Stuart Orkin表示祝贺。。

“It’s a historic day for mankind, scientific advancement, and patients waiting for gene-correcting treatments!” posted Nicole Gaudelli, PhD, head of the DNA editing platform at Beam Therapeutics. “For the first time, a Cas9-based medicine is approved. I am thrilled for the scientific community and the patients we serve.

“这是人类，科学进步和等待基因纠正治疗的患者的历史悠久的一天！”Beam Therapeutics DNA编辑平台负责人Nicole Gaudelli博士发布。“这是第一次批准基于Cas9的药物，我对科学界和我们服务的患者感到兴奋。

Onward!”.

向前。

David Liu, PhD (Broad Institute), Gaudelli’s former PhD supervisor who has pioneered the development of both base and prime editing, called the news of the first CRISPR drug “a major milestone for science, for medicine, and for patients.” He offered congratulations “to the many scientists, doctors, patients and industry leaders who made this development possible.”.

Gaudelli前博士生导师David Liu博士（Broad Institute）率先开发了基础编辑和主要编辑，他称第一种CRISPR药物的消息为“科学，医学和患者的一个重要里程碑”。他祝贺“许多使这一发展成为可能的科学家，医生，患者和行业领导者”。

Janice Chen, PhD, co-founder of Mammoth Biosciences, offered her congratulations to the two biotech companies, the “entire CRISPR field and the brave patients for this historic moment! A new era of medicine has begun.”

Mammoth Biosciences的联合创始人Janice Chen博士向两家生物技术公司表示祝贺：“整个CRISPR领域和这个历史时刻的勇敢患者！医学的新时代已经开始。”

For once, Fyodor Urnov, PhD, (Innovative Genomics Institute) who coined the term “genome editing” in 2005 when he was with Sangamo and is currently leading the charge alongside Nobel laureate Jennifer Doudna, PhD, to accelerate the development of CRISPR therapies for a large number of addressable genetic disorders, was left speechless.

Fyodor Urnov博士（创新基因组学研究所）一度在2005年与Sangamo一起创造了“基因组编辑”一词，目前正在与诺贝尔奖获得者Jennifer Doudna博士一起领导，以加速CRISPR的发展。治疗大量可解决的遗传疾病，仍然无法说话。

His post read simply:.

他的帖子简单地说：。

“!!!!!!!!!!!”

“!!!!!!!!!!!”

(That’s one exclamation mark for each year since the Nobel Prize-winning CRISPR paper. Urnov was more loquacious in this recent exclusive video interview with GEN Edge.)

（这是诺贝尔奖获奖者CRISPR论文以来每年的一个感叹号.Urnov在最近与GEN Edge进行的独家视频采访中更加笨拙。）

Casgevy works by interfering with the transcriptional regulator, BCL11A, that acts like a wheel clamp to block expression of HbF after birth. The restored γ-globin beautifully compensates for the β-globin polypeptide that carries the SCD point mutation.

Casgevy的工作原理是干扰转录调节因子BCL11A，BCL11A的作用类似于轮钳，阻止出生后HbF的表达。恢复的γ-珠蛋白精美地补偿了携带SCD点突变的β-珠蛋白多肽。

The first SCD patient to be dosed in the Casgevy trial, Victoria Gray, was treated by physician Haydar Frangoul, MD, and his team at the Sarah Cannon Institute in Nashville, TN, in July 2019. Like almost all of the dozens of other SCD patients in the trial, she is now healthy and free of pain crises or the need for blood transfusions or hospitalizations..

2019年7月，在田纳西州纳什维尔的Sarah Cannon研究所，医学博士Haydar Frangoul和他的团队在Casgevy试验中服用的第一例SCD患者Victoria Gray接受了治疗。像试验中几乎所有其他数十名SCD患者一样，她现在健康，没有疼痛危机或需要输血或住院治疗。。

Gray recently spoke at an FDA Advisory Committee meeting to assess the safety of the Vertex therapy, particularly in regard to the faint possibility of “off-target” effects, in advance of the final U.S. approval decision.

格雷最近在FDA咨询委员会会议上发表演讲，在美国最终批准决定之前评估顶点疗法的安全性，特别是关于“脱靶”效应的微弱可能性。

While today’s approval is cause for celebration, reality may soon set in when Vertex announces the price of the “one-and-done” therapy, widely expected to be around $2 million. Hematologist Akshay Sharma, MD (St Jude) warned: “Now the real work begins to make this therapy truly accessible to patients who need it the most.”.

虽然今天的批准值得庆祝，但Vertex宣布“一刀切”疗法的价格时，现实可能很快就会开始，预计该疗法的价格约为200万美元。血液学家Akshay Sharma医学博士（圣裘德）警告说：“现在真正的工作开始使最需要它的患者真正能够接受这种治疗。”。

British lawyer and ethicist Julian Hitchcock, who has followed the CRISPR field closely, also raised concerns over pricing and accessibility to U.K. patients on the National Health Service (NHS): “But will we get it on the NHS? I’m dubious.”

紧随CRISPR领域的英国律师兼伦理学家朱利安·希区柯克（Julian Hitchcock）也对英国患者在国家卫生服务（NHS）上的定价和可及性提出了担忧：“但是我们会在NHS上得到它吗？我很怀疑。”