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.
週四，Editas Medicine Inc.（納斯達克：EDIT）宣佈了一項戰略重新調整，專注於體內基因編輯。
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.
體內指的是在活體生物或細胞上進行的研究或實驗，而不是在死去的生物或組織提取物上。體外的反義詞是體內，這意味着在身體外或實驗室中。
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.
這一轉變旨在優化成本，延長其現金儲備到2027年第二季度，並加速其在兩年內實現人體概念驗證的進展。
The transition follows recent in vivo preclinical research advancements, including proof of concept in multiple tissue types.
這一轉變跟隨近期在體內的臨床前研究進展，包括在多種組織類型中的概念驗證。
For hematopoietic stem cells (HSCs), Editas demonstrated ~40% editing of the HBG1/2 promoter using a proprietary targeted lipid nanoparticle (tLNP).
對於造血幹細胞（HSCs），Editas展示了使用專有靶向脂質納米顆粒（tLNP）對HBG1/2啓動子的約40%編輯效果。
Also Read: Editas Medicine Downgraded: Analyst Sees Limited Near-Term Catalysts
另請閱讀：Editas Medicine評級下調：分析師認爲近期催化劑有限
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%.
將Cas12a編輯機械直接傳遞到小鼠的人的HSCs的單劑量給藥產生了顯著的功能性結果。在一個月內，人類紅血球的胎兒血紅蛋白（HbF）平均表達達到20%。
The achievement builds on validated HBG1/2 biology from the RUBY trial with Editas' reni-cel program.
這一成就建立在Editas的reni-cel項目的RUBY試驗中驗證的HBG1/2生物學基礎上。
In liver applications, Editas achieved high-efficiency gene editing in non-human primates in collaboration with Genevant.
在肝臟應用中，Editas與Genevant合作實現了非人類靈長類動物的高效基因編輯。
The company will share detailed preclinical data and development timelines in early 2025.
該公司將在2025年初分享詳細的臨床前數據和開發時間表。
As part of its transition, Editas announced a workforce reduction, eliminating approximately 65% of its employees over the next six months.
作爲轉型的一部分，Editas宣佈裁員，將在接下來的六個月內裁減約65%的員工。
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.
這包括幾位領導團隊成員，如首席醫療官梅柏松。該公司表示，這些變化與其重新聚焦於體內療法相一致。
By restructuring operations and prioritizing its in vivo platform, Editas aims to create a more streamlined and efficient organization.
通過重組運營並優先考慮其體內平台，Editas旨在創建一個更精簡和高效的組織。
Cash, cash equivalents, and marketable securities as of September 30, 2024, were $265.1 million.
截至2024年9月30日，現金、現金等價物和可交易證券爲26510萬。
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.
該公司預計現有的現金、現金等價物和可交易證券、來自DRI的預付款，以及根據與Vertex的許可協議應支付的保留部分將用於資助營業費用和資本支出，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.
價格動態：在上週五最後檢查時，EDIt股票在盤前交易中下跌8.99%，報1.72美元。
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譯文內容由第三人軟體翻譯。
