If Editas Medicine’s CRISPR gene-editing therapy for rare blood diseases reaches the market, it will be in the hands of a different company. The biotech on Tuesday announced plans to partner or out-license that clinical-stage ex vivo therapy, choosing instead to focus its resources on in vivo R&D that now has preclinical proof-of-concept data..

如果Editas Medicine针对罕见血液疾病的CRISPR基因编辑疗法进入市场，它将由另一家公司负责。该生物技术公司周二宣布计划合作或授权该临床阶段的离体治疗，转而选择将其资源集中于体内研发，目前已有临床前概念验证数据。。

The in vivo therapy, renizgamglogene autogedtemcel (reni-cel), has reached late-stage clinical development for severe sickle cell disease. Reni-cel is also in early-stage development for transfusion-dependent beta thalassemia. Patients who have these rare blood disorders already have ex vivo gene-editing treatment options.

体内治疗renizgamglogene autogedtemcel（reni-cel）已达到严重镰状细胞病的晚期临床开发阶段。Reni-cel也处于输血依赖性β地中海贫血的早期发展阶段。患有这些罕见血液疾病的患者已经有了离体基因编辑治疗选择。

Last winter Vertex Pharmaceuticals’ Casgevy landed FDA approvals for sickle cell disease and beta thalassemia. Bluebird Bio’s last winter received FDA approval for Lygenia for sickle cell disease. A different Bluebird gene-editing therapy named Zynteglo won FDA approval in 2022 for beta thalassemia..

去年冬天，Vertex制药公司的Casgevy获得了FDA对镰状细胞病和β地中海贫血的批准。蓝鸟生物去年冬天获得FDA批准用于治疗镰状细胞病的Lygenia。2022年，另一种名为Zynteglo的蓝鸟基因编辑疗法获得了FDA对β地中海贫血的批准。。

Editas’s reni-cel would be third to market, but the Cambridge, Massachusetts-based company has said it could be best in the class of ex vivo therapies, made by editing a patient’s hematopoietic stem cells in a lab and infusing those cells back into the patient. These modified cells are intended to correct the anemia caused by these blood disorders and increase levels of fetal hemoglobin..

Editas的reni-cel将是第三家上市的公司，但这家总部位于马萨诸塞州剑桥的公司表示，它可能是离体疗法中最好的一种，通过在实验室编辑患者的造血干细胞并将这些细胞注入患者体内。这些经过修饰的细胞旨在纠正由这些血液疾病引起的贫血并增加胎儿血红蛋白水平。。

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Dosing is continuing in reni-cel’s pivotal sickle cell study with 28 adult patients dosed to date; dosing is being scheduled for adolescent patients. Editas said data will be presented during the upcoming American Society of Hematology meeting in December. The beta thalassemia study is on track to present clinical data by the end of this year..

reni cel关键的镰状细胞研究仍在继续给药，迄今为止已有28名成年患者给药；正在为青少年患者安排剂量。Editas说，数据将在即将于12月举行的美国血液学会会议上公布。β地中海贫血研究有望在今年年底前提供临床数据。。

Speaking during a Tuesday webcast, Editas CEO Gilmore O’Neill said the company is mindful of the financial cost that comes with commercializing an ex vivo autologous therapy. Placing reni-cel in the hands of another company would allow Editas to substantially reduce its 2025 spending.

Editas首席执行官吉尔莫·奥尼尔（GilmoreO'Neill）在周二的一次网络直播中表示，该公司注意到将离体自体疗法商业化所带来的财务成本。将reni cel交给另一家公司将使Editas大幅减少2025年的支出。

“We have previously said that we would be open to partnering reni-cel, but now, with the progress we’re making on in vivo editing, we are actively undertaking a process to partner or out-license reni-cel to most effectively drive it to commercialization while allowing us to apply our full attention and capital to in vivo medicines,” O’Neill said.

奥尼尔说：“我们之前说过，我们愿意与雷尼细胞合作，但现在，随着我们在体内编辑方面取得的进展，我们正在积极开展一个合作或授权雷尼细胞的过程，以最有效地推动其商业化，同时使我们能够将全部注意力和资金用于体内药物。”。

“The in vivo data we are sharing today makes us even more confident in this decision.”.

“我们今天共享的体内数据使我们对这一决定更有信心。”。

The data that has Editas excited show proof of concept for its in vivo editing approach in mouse models for both sickle cell disease and beta thalassemia. Specifically, the company reported its in vivo therapy enabled an editing level of 29% in hematopoietic stem cells after a single dose. This treatment led to fetal hemoglobin induction (HbF), indicated by the presence of HbF-expressing human red blood cells populating in the host by one month.

Editas兴奋的数据显示了其在镰状细胞病和β地中海贫血小鼠模型中的体内编辑方法的概念验证。具体而言，该公司报告称其体内治疗使单剂量后造血干细胞的编辑水平达到29%。。

O’Neill said this level of in vivo editing in mice after a single dose is “highly competitive” compared to data in the public domain for the development of an in vivo medicine for sickle cell disease and beta thalassemia..

奥尼尔说，与公共领域用于开发镰状细胞病和β地中海贫血的体内药物的数据相比，单次剂量后小鼠体内编辑的水平具有“高度竞争力”。。

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In a note sent to investors, Leerink Partners analyst Mani Foroohar said the decision to seek an external partner for reni-cel makes sense given the cost of commercializing autologous ex vivo therapies. The near-term focus for Editas shares is the additional reni-cel data and the extent to which the company can show differentiation for the therapy to drive business development interest in this program..

Leerink Partners分析师Mani Foroohar在发给投资者的一份报告中表示，考虑到自体离体疗法的商业化成本，为reni cel寻求外部合作伙伴的决定是合理的。Editas股票的近期重点是额外的reni cel数据，以及该公司能够在多大程度上表现出差异化的治疗方法，以推动该计划的业务发展兴趣。。

Editas said it has engaged investment bank Moelis & Company to lead the global process to partner or out-license reni-cel. Meanwhile, the company has secured non-dilutive financing to support its in vivo gene-editing focus. Earlier this month, Editas sold to DRI Healthcare Trust certain future license fees and payments owed under a Vertex Pharmaceuticals’ licensing agreement for Cas9 gene-editing technology.

Editas表示，它已聘请投资银行Moelis&Company领导全球合作进程，或授权reni cel。同时，该公司已获得非稀释性融资，以支持其体内基因编辑重点。本月早些时候，Editas向DRI Healthcare Trust出售了Vertex Pharmaceuticals针对Cas9基因编辑技术的许可协议项下的某些未来许可费和应付款项。

The DRI deal paid Editas $57 million up front. Editas reported its cash position as of the end of the third quarter of this year was about $265 million; or about $320 million including the DRI payment. O’Neill said the company has enough capital to fund operations into 2026..

DRI的交易为Editas预付了5700万美元。Editas报告称，截至今年第三季度末，其现金状况约为2.65亿美元；或约3.2亿美元，包括DRI付款。奥尼尔表示，该公司有足够的资本为2026年的运营提供资金。。

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