Metagenomi Presents Highly Specific and Efficient Genome Editing Tools at Nature Conference "RNA at the Bench and Bedside IV"
Metagenomi Presents Highly Specific and Efficient Genome Editing Tools at Nature Conference "RNA at the Bench and Bedside IV"
MGX-001, utilizing a highly specific and efficient MG29-1 nuclease, exhibits no identifiable off-target editing
MGX-001利用一種高度特異性和高效的MG29-1核酸酶,未顯示出可識別的離靶編輯。
MG29-1 nuclease targeting the albumin safe harbor locus showed no evidence of translocations in primary human hepatocytes
針對白蛋白安全位點的MG29-1核酸酶在原代人類肝細胞中未顯示出任何易位的證據。
Metagenomi Adenine Base Editor (ABE) demonstrates no detectable translocations and no significant genomic base composition differences in primary T-cells
Metagenomi腺嘌呤鹼基編輯器(ABE)在原代T細胞中未檢測到可見的易位以及顯著的基因組鹼基組成差異。
EMERYVILLE, Calif., Dec. 11, 2024 (GLOBE NEWSWIRE) -- Metagenomi, Inc. (Nasdaq: MGX), a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary gene editing toolbox, today presented a talk titled "Specific and efficient genome editing with metagenomics-derived tools for in vivo and ex vivo therapeutic applications" at the Nature Conference: RNA at the Bench and Bedside IV.
加利福尼亞州埃默裏維爾,2024年12月11日(環球新聞通訊)——Metagenomi公司(納斯達克:MGX),一家致力於開發治療性精準基因藥物的公司,使用其專有的基因編輯工具箱,今天在《自然會議:RNA在實驗室與病牀邊 IV》上發表了題爲「利用來源於宏基因組的工具進行體內和體外治療應用的特異性和高效基因組編輯」的演講。
"We believe the value proposition for single-dose gene editing therapies requires exquisite specificity characterization to ensure safety and efficacy. Today's presentation highlights the precision of Metagenomi's next-generation nucleases and ABEs, discovered through the company's proprietary metagenomics platform and tailored for both in vivo and ex vivo therapeutic applications," said Alan Brooks, SVP and Head of Preclinical. "MGX-001, Metagenomi's development candidate for hemophilia A, which utilizes the novel nuclease MG29-1, exhibits no identifiable off-target editing using a series of orthogonal assays employed to evaluate potential off-target sites in the genome. The MG29-1 nuclease targeting the albumin safe harbor locus showed no evidence of translocations in primary human hepatocytes. For Metagenomi's novel next-generation ABE for ex vivo cell therapy indications via multiplex editing, the data showed no detectable translocations and no significant genomic base composition differences in primary T-cells when compared to unedited cells. These examples demonstrate our strong capabilities in developing highly specific next-generation gene editing tools and support the company's ability to potentially progress these systems toward the clinic for the benefit of patients."
「我們相信單劑量基因編輯療法的價值主張需要精確的特異性特徵,以確保安全性和有效性。今天的演示突出了Metagenomi下一代核酸酶和ABE的精準性,這些都是通過公司的專利宏基因組平台發現的,針對體內和體外治療應用進行了定製,」預臨床副總裁兼負責人Alan Brooks說道。「MGX-001是Metagenomi針對血友病A研發的候選藥物,利用新型核酸酶MG29-1,採用一系列正交檢測方法評估基因組中的潛在脫靶位點,未發現可識別的脫靶編輯。MG29-1核酸酶靶向白蛋白安全港位點時,在初級人肝細胞中未顯示出轉位的證據。對於Metagenomi的新型下一代ABE,通過多重編輯進行體外電芯治療指示的數據表明,與未編輯細胞相比,初級T細胞未檢測到轉位,也沒有顯著的基因組鹼基組成差異。這些例子展示了我們在開發高度特異性下一代基因編輯工具方面的強大能力,並支持公司將這些系統推進臨床以造福患者的潛力。」
About Metagenomi
About Metagenomi
Metagenomi is a precision genetic medicines company committed to developing curative therapeutics for patients using its proprietary, comprehensive metagenomics-derived toolbox. Metagenomi is harnessing the power of metagenomics, the study of genetic material recovered from the natural environment, to unlock four billion years of microbial evolution to discover and develop a suite of novel editing tools capable of correcting any type of genetic mutation found anywhere in the genome. Its comprehensive genome editing toolbox includes programmable nucleases, base editors, and RNA and DNA-mediated integration systems (including prime editing systems and clustered regularly interspaced short palindromic repeat associated transposases (CAST)). Metagenomi believes its diverse and modular toolbox positions the company to access the entire genome and select the optimal tool to unlock the full potential of genome editing for patients. For more information, please visit https://metagenomi.co.
Metagenomi是一家致力於開發治癒性基因藥物的精準遺傳學公司,利用其獨有的、全面的源於宏基因組的工具箱爲患者開發治療方法。Metagenomi正在利用宏基因組學的力量,即從自然環境中恢復的遺傳物質的研究,解鎖40億年的微生物進化,以發現和開發一套能夠糾正基因組中任何類型基因突變的新型編輯工具。其全面的基因組編輯工具箱包括可編程核酸酶,鹼基編輯器,RNA和DNA介導的整合系統(包括初級編輯系統和CRISPR相關轉座酶(CAST))。Metagenomi相信其多樣和模塊化的工具箱使該公司能夠訪問整個基因組並選擇最佳工具,釋放基因組編輯爲患者帶來的全部潛力。欲了解更多信息,請訪問https://metagenomi.co。
Cautionary Note Regarding Forward‐Looking Statements
關於前瞻性聲明的警示說明
This press release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, each as amended. Such statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to," "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions, include, but are not limited to, any statements relating to our growth strategy and product development programs, including the timing of and our ability to conduct IND-enabling studies, make regulatory filings such as INDs, statements concerning the potential of therapies and product candidates, and any other statements that are not historical facts. Forward-looking statements are based on management's current expectations and are subject to risks and uncertainties that could negatively affect our business, operating results, financial condition, and stock value. Factors that could cause actual results to differ materially from those currently anticipated include: risks relating to our growth strategy; our ability to obtain, perform under, and maintain financing and strategic agreements and relationships; risks relating to the results of research and development activities; risks relating to the timing of starting and completing clinical trials; uncertainties relating to preclinical and clinical testing; our dependence on third party suppliers; our ability to attract, integrate and retain key personnel; the early stage of products under development; our need for substantial additional funds; government regulation; patent and intellectual property matters; competition; as well as other risks described in "Risk Factors," in our most recent Form 10-K and our most recent 10-Qs on file with the Securities and Exchange Commission. We expressly disclaim any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is based, except as required by law, and we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995.
本新聞發佈包含根據1933年證券法第27A節和1934年證券交易法第21E節(均已修訂)的定義而生成的"前瞻性陳述"。這些陳述通常用諸如"預計、""相信、""可能、""估計、""期望、""目標、""打算、""期待、""可能、""計劃、""潛在、""預測、""項目、""應該、""將、""會"及類似表達的詞語來表示,包括但不限於與我們的增長策略和產品開發計劃相關的任何陳述,包括進行IND啓用研究的時間和能力、提交監管申請如IND的能力、與療法和產品候選者潛力相關的陳述,以及任何不是歷史事實的其他陳述。前瞻性陳述基於管理層當前的期望,並且受到可能對我們的業務、經營結果、財務狀況和股票價值產生負面影響的風險和不確定性的影響。導致實際結果與當前預期存在實質性差異的因素包括:與我們增長策略相關的風險;我們獲得、履行和維持融資和戰略協議及關係的能力;與研究和開發活動結果相關的風險;開始和完成臨床試驗的時間相關的風險;與臨床前和臨床測試相關的不確定性;我們對第三方供應商的依賴;我們吸引、整合和保留關鍵人員的能力;在開發中的產品的早期階段;我們對大量額外資金的需求;政府監管;專利和知識產權問題;競爭;以及在我們最近的10-K表格和最近的10-Q文件中描述的"風險因素"中列出的其他風險。除法律要求外,我們明確聲明無義務或承諾公開發布與此處包含的任何前瞻性陳述的任何更新或修訂,以反映我們的期望變化或與任何此類聲明所依據的事件、條件或情況變化有關的任何更改,我們要求根據1995年《私人證券訴訟改革法》享有前瞻性陳述的安全港保護。
Contact:
Simon Harnest - CIO, SVP Investor Relations
IR@metagenomi.co
聯繫:
西蒙·哈內斯特 - 首席投資官,高級副總裁 投資者關係
IR@metagenomi.co
譯文內容由第三人軟體翻譯。