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Omega Therapeutics Presents New Preclinical Data at ASGCT 2024 Demonstrating Tunable and Durable Upregulation of Gene Expression With Epigenomic Controllers

Omega Therapeutics Presents New Preclinical Data at ASGCT 2024 Demonstrating Tunable and Durable Upregulation of Gene Expression With Epigenomic Controllers

Omega Therapeutics在2024年的ASGCT上公布了新的临床前数据,表明使用表观基因组控制器可以对基因表达进行可调和持久的上调
GlobeNewswire ·  05/08 07:00
  • Programmable epigenomic mRNA candidates enabled durable and robust upregulation of gene expression across a diverse set of gene types and regulatory mechanisms
  • Additional OMEGA platform capabilities demonstrated, including reversible downregulation and multiplexed upregulation of gene expression
  • 可编程的表观基因组mRNA候选药物能够持久而稳健地上调各种基因类型和调控机制的基因表达
  • OMEGA平台的其他功能已得到证实,包括可逆的下调和基因表达的多重上调

CAMBRIDGE, Mass., May 08, 2024 (GLOBE NEWSWIRE) -- Omega Therapeutics, Inc. (Nasdaq: OMGA) ("Omega"), a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines, today announced the presentation of new preclinical data demonstrating durable and tunable bidirectional regulation of gene expression in cellular models at the pre-transcriptional level at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, taking place in Baltimore, Maryland, May 7 – 11.

马萨诸塞州剑桥,2024年5月8日(GLOBE NEWSWIRE)——率先开发新一类可编程表观基因组mRNA药物的临床阶段生物技术公司欧米茄治疗公司(Nasdaq:OMGA)(“欧米茄”)今天宣布在美国基因学会发布新的临床前数据,这些数据表明在转录前水平上对细胞模型中的基因表达进行了持久和可调的双向调节细胞疗法 (ASGCT) 27第四 年会,5月7日至11日在马里兰州巴尔的摩举行。

"These exciting new data underscore the versatile capabilities and power of our OMEGA platform," said Thomas McCauley, Ph.D., Chief Scientific Officer of Omega Therapeutics. "We have demonstrated preclinically that we can prospectively engineer epigenomic controllers to predictably and durably upregulate gene expression across a diverse range of gene types, including turning on inactivated genes, augmenting the expression of genes with low baseline expression levels, and leveraging existing genomic regulatory processes to boost expression. These capabilities unlock a wide spectrum of possibilities to apply precision epigenomic control as a novel therapeutic modality to meaningfully address key drivers of many diseases."

欧米茄疗法首席科学官托马斯·麦考利博士说:“这些令人兴奋的新数据凸显了我们欧米茄平台的多功能能力和力量。”“我们已经在临床前证明,我们可以前瞻性地设计表观基因组控制器,以可预测和持久地上调各种基因类型的基因表达,包括开启灭活基因,以低基线表达水平增强基因的表达,以及利用现有的基因组调控程序来提高表达。这些能力为应用精确表观基因组控制作为一种新的治疗方式开辟了广泛的可能性,可以有意义地解决许多疾病的关键驱动因素。”

Details for the poster presentation are as follows:

海报展示详情如下:

Title: Tuned Upregulation of Diverse Gene Targets Using Programmable Epigenomic Controllers
Abstract Number: 697
Poster Session: Epigenetic Editing and RNA Editing
Date and Time: May 8, 2024, from 12:00 p.m. – 7:00 p.m. ET

标题:使用可编程表观基因组控制器调整上调不同基因靶点
摘要编号:697
海报会议:表观遗传学编辑和 RNA 编辑
日期和时间:2024 年 5 月 8 日,美国东部时间下午 12:00 至晚上 7:00

Key Findings

主要发现

These preclinical findings demonstrate the ability of epigenomic controllers (ECs) to enhance target gene expression across various baseline levels of expression and cellular conditions, achieving up to a 10,000-fold increase in expression for inactivated gene targets and maintaining a stable 2-fold increase in expression for up to 55 days for another gene target. Additionally, simultaneous targeting of two regulatory elements for the same gene with two separate ECs yielded a synergistic effect, more than doubling expression compared to single element targeting.

这些临床前发现表明,表观基因组控制器(EC)能够在不同的基线表达水平和细胞条件下增强靶基因表达,使失活基因靶标的表达量增加多达10,000倍,并将另一个基因靶标的表达量稳定增长2倍,持续长达55天。此外,使用两个单独的EC同时靶向同一基因的两个调节元素产生协同效应,与单个元素靶向相比,表达量增加了一倍以上。

The poster is available on the Omega website at

该海报可在欧米茄网站上找到,网址为

About Omega Therapeutics
Omega Therapeutics is a clinical-stage biotechnology company pioneering the development of a new class of programmable epigenomic mRNA medicines to treat or cure a broad range of diseases. By pre-transcriptionally modulating gene expression, Omega's approach enables precision epigenomic control of nearly all human genes, including historically undruggable and difficult-to-treat targets, without altering native nucleic acid sequences. Founded in 2017 by Flagship Pioneering following breakthrough research by world-renowned experts in the field of epigenetics, Omega is led by a seasoned and accomplished leadership team with a track record of innovation and operational excellence. The Company is committed to revolutionizing genomic medicine and has a pipeline of therapeutic candidates derived from its OMEGA platform spanning oncology, regenerative medicine, and multigenic diseases including inflammatory and cardiometabolic conditions.

关于欧米茄治疗公司
Omega Therapeutics是一家临床阶段的生物技术公司,率先开发了一类用于治疗或治愈各种疾病的新型可编程表观基因组mRNA药物。通过预转录调节基因表达,欧米茄的方法可以在不改变原生核酸序列的情况下对几乎所有人类基因(包括历史上不可药物治疗和难以治疗的靶标)进行精确的表观基因组控制。欧米茄由Flagship Pioneeringer在表观遗传学领域的世界知名专家进行了突破性研究后,于2017年创立,由一支经验丰富、成就卓越的领导团队领导,在创新和卓越运营方面有着良好的记录。该公司致力于革新基因组医学,并拥有一系列源自其OMEGA平台的候选治疗药物,涵盖肿瘤学、再生医学以及包括炎症和心脏代谢疾病在内的多基因疾病。

For more information, visit omegatherapeutics.com, or follow us on X and LinkedIn.

欲了解更多信息,请访问 omegatherapeutics.com,或在 X 和 LinkedIn 上关注我们。

About the OMEGA platform
The OMEGA platform leverages the Company's deep understanding of gene regulation, genomic architecture and epigenetic mechanisms to design programmable epigenomic mRNA medicines that precisely target and modulate gene expression at the pre-transcriptional level. Combining world-class data science capabilities with rational drug design and customized delivery, the OMEGA platform enables control of fundamental epigenetic processes and reprogramming of cellular physiology to address the root cause of disease. Omega's modular and programmable mRNA medicines, called epigenomic controllers, target specific genomic loci within insulated genomic domains with high specificity to durably tune single or multiple genes to treat and cure diseases through unprecedented precision epigenomic control.

关于 OMEGA 平台
OMEGA平台利用公司对基因调控、基因组结构和表观遗传学机制的深刻理解,设计可编程的表观基因组mRNA药物,在转录前水平上精确靶向和调节基因表达。OMEGA平台将世界一流的数据科学能力与合理的药物设计和定制交付相结合,可以控制基本的表观遗传学过程和重新编程细胞生理学以解决疾病的根本原因。欧米茄的模块化和可编程mRNA药物称为表观基因组控制器,以高特异性靶向绝缘基因组域内的特定基因组位点,通过前所未有的精确表观基因组控制来持久调整单个或多个基因,以治疗和治愈疾病。

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements contained in this press release that do not relate to matters of historical fact should be considered forward-looking statements, including without limitation statements regarding the versatile capabilities and power of the OMEGA platform, the wide spectrum of possibilities to apply precision epigenomic control as a novel therapeutic modality to meaningfully address key drivers of many diseases, the potential of the Company's pipeline of therapeutic candidates, and upcoming events and presentations. These statements are neither promises nor guarantees, but involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements, including, but not limited to, the following: the novel technology on which our product candidates are based makes it difficult to predict the time and cost of preclinical and clinical development and subsequently obtaining regulatory approval, if at all; the substantial development and regulatory risks associated with epigenomic controllers due to the novel and unprecedented nature of this new category of medicines; our limited operating history; the incurrence of significant losses and the fact that we expect to continue to incur significant additional losses for the foreseeable future; our need for substantial additional financing; volatility in capital markets and general economic conditions; our investments in research and development efforts that further enhance the OMEGA platform, and their impact on our results; uncertainty regarding preclinical development, especially for a new class of medicines such as epigenomic controllers; potential delays in and unforeseen costs arising from our clinical trials; the fact that our product candidates may be associated with serious adverse events, undesirable side effects or have other properties that could halt their regulatory development, prevent their regulatory approval, limit their commercial potential, or result in significant negative consequences; difficulties manufacturing the novel technology on which our epigenomic controller candidates are based; our ability to adapt to rapid and significant technological change; our reliance on third parties for the manufacture of materials; our ability to successfully acquire and establish our own manufacturing facilities and infrastructure; our reliance on a limited number of suppliers for lipid excipients used in our product candidates; our ability to advance our product candidates to clinical development; and our ability to obtain, maintain, enforce and adequately protect our intellectual property rights. These and other important factors discussed under the caption ""Risk Factors" in our Quarterly Report on Form 10-Q for the quarter ended March 31, 2024, and our other filings with the SEC, could cause actual results to differ materially from those indicated by the forward-looking statements made in this press release. Any such forward-looking statements represent management's estimates as of the date of this press release. While we may elect to update such forward-looking statements at some point in the future, we disclaim any obligation to do so, even if subsequent events cause our views to change.

前瞻性陈述
本新闻稿包含1995年《私人证券诉讼改革法》所指的前瞻性陈述。本新闻稿中包含的所有与历史事实无关的陈述均应被视为前瞻性陈述,包括但不限于关于欧米茄平台的多功能能力和力量、应用精确表观基因组控制作为一种新型治疗模式以有意义地解决许多疾病关键驱动因素的广泛可能性、公司候选治疗产品线的潜力以及即将举行的活动和演讲。这些陈述既不是承诺也不是保证,但涉及已知和未知的风险、不确定性和其他重要因素,这些因素可能导致我们的实际结果、业绩或成就与前瞻性陈述所表达或暗示的任何未来业绩、业绩或成就存在重大差异,包括但不限于以下内容:我们的候选产品所依据的新技术使我们难以预测临床前和临床开发以及随后获得监管的时间和成本批准(如果有的话);由于这种新药物前所未有的新颖性质,与表观基因组控制器相关的重大开发和监管风险;我们的运营历史有限;蒙受重大损失以及我们预计在可预见的将来将继续蒙受重大额外损失的事实;我们对大量额外融资的需求;资本市场和总体经济状况的波动;我们对进一步增强OMEGA平台的研发工作的投资,以及它们对我们研究结果的影响;临床前开发的不确定性,尤其是表观基因组控制剂等新药物的不确定性;临床试验产生的潜在延误和不可预见的成本;我们的候选产品可能与严重的不良事件、不良副作用有关或具有其他可能阻碍其监管发展、阻碍其监管批准、限制其商业潜力或导致重大负面后果的特性;难以制造新技术我们的表观基因组控制候选人是以此为基础的;我们适应快速而重大技术变革的能力;我们对第三方制造材料的依赖;我们成功收购和建立自己的制造设施和基础设施的能力;我们在候选产品中使用的脂质赋形剂依赖有限的供应商;我们推动候选产品进入临床开发的能力;以及我们获得、维护、执行和充分保护知识产权的能力。在截至2024年3月31日的季度10-Q表季度报告以及我们向美国证券交易委员会提交的其他文件中,在 “风险因素” 标题下讨论的这些和其他重要因素可能导致实际业绩与本新闻稿中前瞻性陈述所示的业绩存在重大差异。任何此类前瞻性陈述均代表管理层截至本新闻稿发布之日的估计。尽管我们可能会选择在未来的某个时候更新此类前瞻性陈述,但我们不承担任何更新此类前瞻性陈述的义务,即使随后发生的事件导致我们的观点发生变化。

Contact
Investor contact:
Eva Stroynowski
617.949.4370
estroynowski@omegatx.com

联系我们
投资者联系人:
伊娃·斯特罗伊诺夫斯基
617.949.4370
estroynowski@omegatx.com

Media contact:
Mollie Godbout
LifeSci Communications
646.847.1401
mgodbout@lifescicomms.com

媒体联系人:
Mollie Godbout
LifeSci
646.847.1401
mgodbout@lifescicomms.com


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