DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, today announced a poster presentation at the European Society of Gene & Cell Therapy (ESGCT) 31st Annual Congress being held October 22-25, 2024, in Rome, Italy.

北卡罗来纳州达勒姆（商业新闻短讯）--Precision BioSciences，Inc.（纳斯达克：DTIL），一家临床阶段基因编辑公司，利用其新型专有ARCUS®平台开发用于复杂基因编辑的体内基因编辑疗法，今天宣布在2024年10月22日至25日在意大利罗马举行的欧洲基因与细胞治疗学会（ESGCT）第31届年会上发布海报。

The poster highlighted preclinical data demonstrating the ability of ARCUS to achieve high-efficiency gene insertion, gene replacement and base correction via homology-directed repair (HDR)..

海报强调了临床前数据，证明了ARCUS通过同源定向修复（HDR）实现高效基因插入，基因替代和碱基校正的能力。。

“Gene editing enzymes that support efficient HDR are relatively rare. However, we demonstrate here that ARCUS is capable of utilizing HDR with high efficiency to achieve a range of gene editing outcomes, including specific base changes, insertions, and the replacement of large segments of DNA within the genome,' said Jeff Smith, PhD, Co-Founder and Chief Research Officer of Precision BioSciences.

Precision BioSciences联合创始人兼首席研究官杰夫·史密斯（Jeff Smith）博士说：“支持高效HDR的基因编辑酶相对较少。然而，我们在这里证明，ARCUS能够高效利用HDR来实现一系列基因编辑结果，包括特定的碱基变化、插入和基因组内大片段DNA的替换。”。

“These data continue to validate the ARCUS platform as an ideal modality for treating diseases that require gene insertion or repair to restore function whereas other gene editing modalities are largely focused on knocking-out function. In addition to the preclinical data presented today, we look forward to seeing supportive clinical validation of HDR-based ARCUS gene insertion through our partner iECURE, who is advancing their ECUR-506 clinical stage program utilizing an ARCUS nuclease for treating OTC deficiency,” added Jeff Smith..

“这些数据继续验证了ARCUS平台是治疗需要基因插入或修复以恢复功能的疾病的理想方式，而其他基因编辑方式主要集中在敲除功能上。除了今天提供的临床前数据外，我们期待通过我们的合作伙伴iCure看到对基于HDR的ARCUS基因插入的支持性临床验证，iCure正在推进其利用ARCUS核酸酶治疗OTC缺陷的ECUR-506临床阶段计划，”杰夫·史密斯补充道。。

Title: High-efficiency homology-directed insertion into the genome using ARCUS nucleases

标题：使用ARCUS核酸酶将高效同源性定向插入基因组

Poster Number: #PO678

海报编号：#PO678

Presenter: Adam Mischler, PhD, Senior Scientist, Precision BioSciences Gene Discovery

主持人：Adam Mishler博士，Precision BioSciences Gene Discovery高级科学家

Date and Time: Thursday, October 24, 2024, 6:00-7:30pm CEST

日期和时间：2024年10月24日星期四，东部标准时间下午6:00-7:30

In preclinical work presented today, we show that targeted gene insertion can be achieved using ARCUS in greater than 85% of T cells and 39% of non-dividing primary human hepatocytes. These high rates of gene insertion were accomplished primarily through HDR as evidenced by the dependency on homology arms in the repair template and on the unique ARCUS 3’ 4 base pair overhang cut that resembles a processed end for stimulating HDR.

在今天介绍的临床前工作中，我们表明，使用ARCUS可以在超过85%的T细胞和39%的非分裂原代人肝细胞中实现靶向基因插入。这些高基因插入率主要是通过HDR完成的，这可以通过依赖修复模板中的同源臂和独特的ARCUS 3'4碱基对突出切口来证明，该切口类似于刺激HDR的加工末端。

Using HDR, ARCUS successfully demonstrates the ability to support a wide range of DNA editing approaches using a template to define the specific edit. Unlike base editors’ limitation to two possible base changes, ARCUS was able to produce all twelve possible base changes, in addition to producing very specific base removal and insertions.

使用HDR，ARCUS成功证明了使用模板定义特定编辑来支持多种DNA编辑方法的能力。与基础编辑器对两个可能的基础更改的限制不同，ARCUS能够产生所有十二个可能的基础更改，此外还能产生非常具体的基础删除和插入。

Unlike prime editors, ARCUS was also able to generate whole gene insertions and even replace large segments within a gene to restore function. The characteristic enzymology of ARCUS nucleases drives high rates of HDR, which can potentially be leveraged to achieve a multitude of highly precise gene editing functions for therapeutic benefit..

与prime编辑器不同，ARCUS还能够产生全基因插入，甚至可以替换基因内的大片段以恢复功能。。。

About Precision BioSciences, Inc.

关于Precision BioSciences，Inc。

Precision BioSciences, Inc. is an advanced gene editing company dedicated to improving life (DTIL) with its novel and proprietary ARCUS genome editing platform that differs from other technologies in the way it cuts, its smaller size, and its simpler structure. Key capabilities and differentiating characteristics may enable ARCUS nucleases to drive more intended, defined therapeutic outcomes.

Precision BioSciences，Inc.是一家致力于改善生命（DTIL）的先进基因编辑公司，其新颖且专有的ARCUS基因组编辑平台在切割方式，尺寸较小和结构更简单方面与其他技术不同。关键能力和差异化特征可能使ARCUS核酸酶能够驱动更预期的，明确的治疗结果。

Using ARCUS, the Company’s pipeline is comprised of in vivo gene editing candidates designed to deliver lasting cures for the broadest range of genetic and infectious diseases where no adequate treatments exist. For more information about Precision BioSciences, please visit www.precisionbiosciences.com..

使用ARCUS，该公司的管道由体内基因编辑候选人组成，旨在为没有足够治疗方法的最广泛的遗传和传染病提供持久的治疗。有关精密生物科学的更多信息，请访问www.precisionbiosciences.com。。

The ARCUS platform is being used to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion (inserting DNA into a gene to cause expression/add function), elimination (removing a genome, e.g., viral DNA or mutant mitochondrial DNA), and excision (removing a large portion of a defective gene by delivering two ARCUS nucleases in a single AAV)..

ARCUS平台正在用于开发用于复杂基因编辑的体内基因编辑疗法，包括基因插入（将DNA插入基因以引起表达/添加功能），消除（去除基因组，例如病毒DNA或突变线粒体DNA）和切除（通过在单个AAV中递送两个ARCUS核酸酶来去除大部分缺陷基因）。。

Forward-Looking Statements

前瞻性声明

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 clinical development and expected safety, efficacy and benefit of our product candidates and gene editing approaches including editing efficiency, key capabilities and differentiating characteristics, the ability of ARCUS to achieve any type of edit including high-efficiency gene insertion, gene replacement, and base correction, the ability of ARCUS to stimulate gene editing by homology directed repair, the potentially broader therapeutic potential of ARCUS for diseases that require a gain in function than current gene editing modalities and the suitability of ARCUS nucleases for gene elimination, insertion and excision and differentiation from other gene editing approaches due to its small size, simplicity and distinctive cut.

本新闻稿包含1995年《私人证券诉讼改革法案》所指的前瞻性声明。本新闻稿中包含的所有与历史事实无关的陈述都应被视为前瞻性陈述，包括但不限于关于我们候选产品和基因编辑方法的临床开发和预期安全性、有效性和益处的陈述，包括编辑效率、关键能力和区别特征、ARCUS实现任何类型编辑的能力，包括高效基因插入、基因替代和碱基校正、ARCUS通过同源定向修复刺激基因编辑的能力、ARCUS对需要比当前基因编辑方式获得功能的疾病的潜在更广泛的治疗潜力，以及ARCUS核酸酶因其体积小而适合于基因消除、插入和切除以及与其他基因编辑方法的区别。简约别致的剪裁。

In some cases, you can identify forward-looking statements by terms such as “aim,” “anticipate,” “approach,” “believe,” “contemplate,” “could,” “designed,” “estimate,” “expect,” “goal,” “intend,” “look,” “may,” “mission,” “plan,” “possible,” “potential,” “predict,” “project,” “pursue,” “should,” “strive,” “target,” “will,” “would,” or the negative thereof and similar words and expressions..

在某些情况下，您可以通过诸如“目标”、“预期”、“方法”、“相信”、“沉思”、“可能”、“设计”、“估计”、“预期”、“目标”、“打算”、“看起来”、“可能”、“任务”、“计划”、“可能”、“潜力”、“预测”、“项目”、“追求”、“应该”、“奋斗”、“目标”、“意志”、“将会”或其否定词以及类似的词语和表达来识别前瞻性陈述。。

Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. These statements are neither promises nor guarantees, and involve a number of known and unknown risks, uncertainties and assumptions, and actual results may differ materially from those expressed or implied in the forward-looking statements due to various important factors, including, but not limited to, our ability to become profitable; our ability to procure sufficient funding to advance our programs; risks associated with our capital requirements, anticipated cash runway, requirements under our current debt instruments and effects of restrictions thereunder, including our ability to raise additional capital due to market conditions and/or our market capitalization; our operating expenses and our ability to predict what those expenses will be; our limited operating history; the progression and success of our programs and product candidates in which we expend our resources; our limited ability or inability to assess the safety and efficacy of our product candidates; the risk that other genome-editing technologies may provide significant advantages over our ARCUS technology; our dependence on our ARCUS technology; the initiation, cost, timing, progress, achievement of milestones and results of research and development activities and preclinical and clinical studies, including clinical trial and investigational new drug applications; public perception about genome editing technology and its applications; competition in the genome editing, biopharmaceutical, and biotechnology fields; our or our collaborators’ or other licensees’ ability to identify, develop and commercialize product candidates; pending and potential product liabi.

前瞻性陈述基于管理层当前的期望、信念和假设以及我们目前可获得的信息。这些陈述既不是承诺也不是保证，涉及许多已知和未知的风险、不确定性和假设，由于各种重要因素，包括但不限于我们的盈利能力，实际结果可能与前瞻性陈述中明示或暗示的结果存在重大差异；我们有能力获得足够的资金来推进我们的计划；与我们的资本要求、预期现金周转、我们当前债务工具的要求和限制的影响相关的风险，包括我们由于市场条件和/或我们的市值而筹集额外资本的能力；我们的运营费用和预测这些费用的能力；我们有限的经营历史；我们花费资源的计划和候选产品的进展和成功；我们的能力有限或无法评估候选产品的安全性和有效性；其他基因组编辑技术可能比我们的ARCUS技术具有显着优势的风险；我们对ARCUS技术的依赖；研究和开发活动以及临床前和临床研究（包括临床试验和研究性新药应用）的启动，成本，时间，进度，里程碑的实现和结果；公众对基因组编辑技术及其应用的看法；基因组编辑，生物制药和生物技术领域的竞争；我们或我们的合作者或其他被许可方识别、开发和商业化候选产品的能力；未决和潜在产品责任。