Editas Medicine Realigns Strategy to Focus on In Vivo Gene Editing, Targets Human Proof Of Concept By 2026
Editas Medicine Realigns Strategy to Focus on In Vivo Gene Editing, Targets Human Proof Of Concept By 2026
The transition follows recent in vivo preclinical research advancements, including proof of concept in multiple tissue types.On Thursday, Editas Medicine Inc. (NASDAQ:EDIT) announced a strategic realignment to focus on in vivo gene editing.
周四，Editas Medicine Inc.（纳斯达克：EDIT）宣布了一项战略重新调整，专注于体内基因编辑。
In vivo refers to studies or experiments conducted on living organisms or cells instead of dead organisms or tissue extracts. The opposite of in vivo is in vitro, which means outside the body or in a laboratory.
体内指的是在活体生物或细胞上进行的研究或实验，而不是在死去的生物或组织提取物上。体外的反义词是体内，这意味着在身体外或实验室中。
The shift is designed to optimize costs, extend its cash runway into the second quarter of 2027, and accelerate its progress toward achieving human proof of concept within two years.
这一转变旨在优化成本，延长其现金储备到2027年第二季度，并加速其在两年内实现人体概念验证的进展。
The transition follows recent in vivo preclinical research advancements, including proof of concept in multiple tissue types.
这一转变跟随近期在体内的临床前研究进展，包括在多种组织类型中的概念验证。
For hematopoietic stem cells (HSCs), Editas demonstrated ~40% editing of the HBG1/2 promoter using a proprietary targeted lipid nanoparticle (tLNP).
对于造血干细胞（HSCs），Editas展示了使用专有靶向脂质纳米颗粒（tLNP）对HBG1/2启动子的约40%编辑效果。
Also Read: Editas Medicine Downgraded: Analyst Sees Limited Near-Term Catalysts
另请阅读：Editas Medicine评级下调：分析师认为近期催化剂有限
The single-dose delivery of Cas12a editing machinery directly to human HSCs in mice yielded significant functional outcomes. Within one month, human red blood cells expressed fetal hemoglobin (HbF) at an average of 20%.
将Cas12a编辑机械直接传递到小鼠的人的HSCs的单剂量给药产生了显著的功能性结果。在一个月内，人类红血球的胎儿血红蛋白（HbF）平均表达达到20%。
The achievement builds on validated HBG1/2 biology from the RUBY trial with Editas' reni-cel program.
这一成就建立在Editas的reni-cel项目的RUBY试验中验证的HBG1/2生物学基础上。
In liver applications, Editas achieved high-efficiency gene editing in non-human primates in collaboration with Genevant.
在肝脏应用中，Editas与Genevant合作实现了非人类灵长类动物的高效基因编辑。
The company will share detailed preclinical data and development timelines in early 2025.
该公司将在2025年初分享详细的临床前数据和开发时间表。
As part of its transition, Editas announced a workforce reduction, eliminating approximately 65% of its employees over the next six months.
作为转型的一部分，Editas宣布裁员，将在接下来的六个月内裁减约65%的员工。
This includes several leadership team members, such as Chief Medical Officer Baisong Mei. The company stated that these changes align with its renewed focus on in vivo therapies.
这包括几位领导团队成员，如首席医疗官梅柏松。该公司表示，这些变化与其重新聚焦于体内疗法相一致。
By restructuring operations and prioritizing its in vivo platform, Editas aims to create a more streamlined and efficient organization.
通过重组运营并优先考虑其体内平台，Editas旨在创建一个更精简和高效的组织。
Cash, cash equivalents, and marketable securities as of September 30, 2024, were $265.1 million.
截至2024年9月30日，现金、现金等价物和可交易证券为26510万。
The company expects the existing cash, cash equivalents, and marketable securities, the upfront cash payment from DRI, and the retained portions of the payments payable under the license agreement with Vertex to fund operating expenses and capital expenditures into the second quarter of 2026.
该公司预计现有的现金、现金等价物和可交易证券、来自DRI的预付款，以及根据与Vertex的许可协议应支付的保留部分将用于资助营业费用和资本支出，直至2026年第二季度。
Price Action: At last check on Friday, EDIT stock was down 8.99% at $1.72 during the premarket session.
价格动态：在上周五最后检查时，EDIt股票在盘前交易中下跌8.99%，报1.72美元。
- What Does Dental Player Patterson's $4.1 Billion Deal Mean For Other Players?
- 帕特森的41亿交易对其他球员意味着什么？
