Editas Medicine is pivoting once again, announcing Tuesday that it will seek a partner to advance its lead gene editing therapy, or potentially out-license rights to the treatment entirely.

Editas Medicine于周二宣布，将寻求合作伙伴推进其领先的基因编辑疗法，或可能完全取消该疗法的许可权。

The therapy, called reni-cel, is designed to treat sickle cell disease and beta thalassemia — the same two diseases targeted by Casgevy, a similar gene editing treatment from Vertex Pharmaceuticals and CRISPR Therapeutics that won U.S. clearance last year. Clinical trial results to date have indicated reni-cel can work equivalently well in treating sickle cell, but it’s likely at least several years from an approval of its own..

该疗法被称为reni-cel，旨在治疗镰状细胞病和β地中海贫血，这两种疾病也是Casgevy针对的，这是Vertex Pharmaceuticals和CRISPR Therapeutics去年获得美国批准的类似基因编辑疗法。迄今为止的临床试验结果表明，reni-cel在治疗镰状细胞方面可以发挥同等作用，但它可能至少需要几年才能获得批准。。

“We believe that the best option for both patients and our shareholders is for us to seek an alternative such as a global partner or out-licensing, which would allow for further development and ultimately commercialization of reni-cel with or by another party and would allow us to substantially reduce spend in 2025,” said Gilmore O’Neill, Editas’ CEO, in the company’s Tuesday statement..

Editas首席执行官吉尔莫·奥尼尔（Gilmore O'Neill）在周二的声明中表示：“我们认为，对于患者和股东来说，最好的选择是寻求一种替代方案，例如全球合作伙伴或外部许可，这将允许reni-cel与另一方或由另一方进一步开发并最终商业化，并将使我们能够在2025年大幅减少支出。”。。

Editas has hired the investment bank Moelis & Company to lead its search for a partner or acquirer.

Editas已聘请投资银行Moelis&Company牵头寻找合作伙伴或收购方。

A deal, if reached, would pass on the challenge of reaching market to a better-resourced firm. But it also leaves Editas hitting reset two years after the biotechnology company turned away from using CRISPR gene editing to treat diseases of the eye.

如果达成协议，将把进入市场的挑战传递给资源更好的公司。但在生物技术公司放弃使用CRISPR基因编辑来治疗眼部疾病两年后，这也让Editas陷入了重置状态。

While Editas was one of the first biotechs founded to turn CRISPR technology into medicines, it’s struggled with research delays and substantial executive turnover. It has previously laid off staff, cut spending and sold off rights to some cancer cell therapy research it had been pursuing.

虽然Editas是首批将CRISPR技术转化为药物的生物技术公司之一，但它一直在与研究延迟和高管大幅更替作斗争。此前，该公司曾裁员、削减开支，并出售了其一直在进行的一些癌细胞治疗研究的权利。

Now, Editas is turning its focus to “in vivo” gene editing, where a CRISPR therapy does its work modifying genes after it’s infused into the body. Reni-cel and Casgevy are both built from a patient’s own stem cells, which are collected and then edited in a laboratory.

现在，Editas正在将重点转向“体内”基因编辑，即CRISPR疗法在注入体内后对基因进行修饰。Reni-cel和Casgevy都是由患者自己的干细胞构建的，这些干细胞被收集并在实验室进行编辑。

Along with its announcement Tuesday, Editas also shared preclinical data that it described as “proof of concept” for using CRISPR gene editing in vivo to treat sickle cell and beta thalassemia. Working with mice engrafted with human stem cells, company researchers were able to shuttle CRISPR tools into the cells, where they edited a specific part of the genome that helps to govern production of a fetal form of the blood protein hemoglobin..

Editas在周二发布公告的同时，还分享了临床前数据，称其为在体内使用CRISPR基因编辑治疗镰状细胞和β地中海贫血的“概念验证”。公司研究人员利用植入人类干细胞的小鼠，能够将CRISPR工具穿梭到细胞中，在那里他们编辑了基因组的特定部分，有助于控制胎儿形式的血液蛋白血红蛋白的产生。。

Turning fetal hemoglobin back on is known to help protect people with sickle cell and beta thalassemia from disease symptoms. Reni-cel and Casgevy, while working in different ways, are also designed to boost fetal hemoglobin production.

众所周知，重新打开胎儿血红蛋白有助于保护镰状细胞和β地中海贫血患者免受疾病症状的影响。雷尼·塞尔（ReniCel）和卡西维（Casgevy）虽然以不同的方式工作，但也被设计用于促进胎儿血红蛋白的产生。

An in vivo medicine would hold advantages over reni-cel and Casgevy, most notably removing the need for preparatory chemotherapy treatment that comes with safety risks. Treatment might also be more accessible, as a patient’s stem cells wouldn’t need to be shipped back to central manufacturing laboratories..

体内药物将比reni-cel和Casgevy具有优势，最显着的是不需要伴随安全风险的准备性化疗。治疗也可能更容易获得，因为患者的干细胞不需要运回中央制造实验室。。

But it’s a more distant dream and, like the research journeys of reni-cel and Casgevy, likely to bring new research challenges. Editas is also considering how else it might use its drug delivery technology to bring in vivo gene editing to harder-to-reach tissues outside of the liver.

但这是一个更遥远的梦想，就像雷尼·塞尔和卡西维的研究之旅一样，可能会带来新的研究挑战。Editas也在考虑如何利用其药物输送技术将体内基因编辑带到更难到达肝脏以外的组织。

In the short term, stepping back from reni-cel development will improve Editas’ financial outlook by reducing anticipated spending. The company recently struck a deal with Vertex that brought it some needed money, and then traded future payments promised under that alliance for upfront cash in a separate agreement with DRI Healthcare Trust..

从短期来看，退出reni cel开发将通过减少预期支出来改善Editas的财务前景。该公司最近与Vertex达成了一项协议，为其带来了一些所需资金，然后在与DRI Healthcare Trust的另一份协议中，用该联盟承诺的未来付款换取预付现金。。

Adding in the funds from the DRI deal, Editas estimates it will end the third quarter with about $320 million in cash, cash equivalents and marketable securities.

加上DRI交易的资金，Editas估计第三季度末将有约3.2亿美元的现金、现金等价物和有价证券。

“Whether a partnership comes to fruition — and under what financial terms — is a point of interest, but in short, [Editas]’s intention for capital-efficient development should be a step in a positive direction,” wrote Dae Gon Ha, an analyst at Stifel, in an investor note.

Stifel分析师Dae Gon Ha在一份投资者笔记中写道：“合伙关系是否取得成果以及在何种财务条件下取得成果，是一个关注点，但简而言之，[Editas]对资本高效发展的意图应该是朝着积极方向迈出的一步。”。

Shares in Editas fell by about 8% Tuesday morning after rising Monday afternoon. The company on Monday announced a research collaboration with Genevant Sciences.

Editas股价在周一下午上涨后，周二上午下跌约8%。该公司周一宣布与Genevant Sciences进行研究合作。