Dive Brief:

潜水简介：

Prime Medicine will soon begin the first clinical trial of a new drugmaking technique known as prime editing, following the Food and Drug Administration’s clearance of its application to start human testing.

在食品和药物管理局批准其开始人体测试的应用后，Prime Medicine不久将开始一种称为Prime editing的新制药技术的首次临床试验。

The regulatory green light, announced by Prime Monday, is an important early step for prime editing, which adapts CRISPR technology to rewrite defective genes without breaking both strands of the DNA double helix. It’s sometimes likened to a kind of genetic “word processor,” rather than the “scissors” analogy often used to describe standard CRISPR editing..

Prime周一宣布的监管绿灯是Prime编辑的重要早期步骤，它采用CRISPR技术重写缺陷基因而不破坏DNA双螺旋的两条链。它有时被比作一种基因“文字处理器”，而不是通常用于描述标准CRISPR编辑的“剪刀”类比。。

Prime plans to first test its technology against a rare inherited condition known as chronic granulomatous disease. The company anticipates enrolling adults with stable disease in a Phase 1/2 study to assess whether its prime editing candidate PM359 is safe and works as intended.

Prime计划首先针对一种罕见的遗传性慢性肉芽肿疾病测试其技术。该公司预计在1/2期研究中招募患有稳定疾病的成年人，以评估其主要编辑候选人PM359是否安全并按预期工作。

Dive Insight:

潜水洞察：

FDA trial start clearances are usually a perfunctory exercise. But they take on greater significance when a new drug technology is being tested in humans for the first time.

FDA的试验启动许可通常是敷衍了事。但是，当一种新的药物技术首次在人体中进行测试时，它们就具有更大的意义。

The regulator has moved cautiously to green light studies of gene editing therapies, previously issuing halts to CRISPR Therapeutics, Beam Therapeutics and Verve Therapeutics when those companies sought to start their first trials.

监管机构谨慎地对基因编辑疗法进行了绿灯研究，此前当这些公司试图开始首次试验时，CRISPR Therapeutics，Beam Therapeutics和Verve Therapeutics停止了研究。

That the agency cleared Prime’s initial request without a preliminary hold is a positive sign, according to Dae Gon Ha, an analyst at Stifel. “Our view is that FDA is increasingly comfortable with gene editing technologies,” Ha wrote in a note to clients. “What's important is that with the ... clearance, prime editing is one-step closer to becoming an important toolkit as a therapeutic.”.

Stifel分析师Dae Gon Ha表示，该机构在没有初步搁置的情况下批准了Prime的初步请求，这是一个积极的迹象。“我们的观点是，FDA对基因编辑技术越来越满意，”Ha在给客户的一份说明中写道。“重要的是，有了……许可，prime编辑离成为治疗工具的重要工具包又近了一步。”。

The clearance means prime editing will have gone from academic paper to human trials in about 4.5 years, as Prime co-founder and noted gene editing researcher David Liu pointed out on X.

正如prime联合创始人兼著名基因编辑研究员DavidLiu在X上指出的那样，该许可意味着prime编辑将在大约4.5年内从学术论文进入人体试验。

While that’s fast, Prime has been under pressure to show the vast promise of its technology can translate to similarly sizable market opportunities. Company shares have declined steadily on doubts Prime will be able to prove that in the short term, particularly given its initial target of chronic granulomatous disease..

虽然速度很快，但Prime一直面临压力，要求它证明其技术的巨大前景可以转化为类似规模的市场机会。由于怀疑Prime能否在短期内证明这一点，公司股价稳步下跌，特别是考虑到其最初的目标是慢性肉芽肿性疾病。。

“The pushback/challenge here hasn't been on the prime editing technology, but rather the [total addressable market] of [chronic granulomatous disease] as a first indication is on the smaller end of the spectrum to meaningfully excite investors,” wrote Ha.

“这里的阻碍/挑战不在于主要的编辑技术，而在于[慢性肉芽肿性疾病]的[总可寻址市场]作为第一个迹象，是在频谱的较小一端，以有意义地激发投资者，”Ha写道。

Gene editing developers beyond Prime have faced similar questions, and companies like Beam, Verve and Intellia Therapeutics have seen their share prices fall over the past year.

Prime以外的基因编辑开发商也面临着类似的问题，Beam、Verve和Intellia Therapeutics等公司的股价在过去一年中出现了下跌。

Prime’s trial will follow participants for safety as well as biological markers of immune function and, over the longer term, resolution of the disease’s complications. Initial data is expected next year.

Prime的试验将跟踪参与者的安全性以及免疫功能的生物标志物，并从长远来看，解决疾病的并发症。预计明年将有初步数据。

The company’s therapy consists of patient stem cells that are modified in a laboratory using prime editing to correct the disease-causing mutation. Between one in 100,000 to 200,000 people born in the U.S. are estimated to have chronic granulomatous disease, which is associated with susceptibility to infection and an array of autoimmune complications.

该公司的疗法由患者干细胞组成，这些干细胞在实验室中使用初级编辑进行修饰，以纠正致病突变。据估计，在美国出生的每10万至20万人中就有一人患有慢性肉芽肿性疾病，这与感染易感性和一系列自身免疫并发症有关。

Refractory or antibiotic-resistant infections are the most common cause of death among people with the condition..

难治性或抗生素耐药性感染是该病患者最常见的死亡原因。。