DURHAM, N.C.--(BUSINESS WIRE)--Precision BioSciences, Inc. (Nasdaq: DTIL), an advanced gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for sophisticated gene edits, including gene insertion, excision, and elimination, today announced that the company will present two posters at the ESGCT 30th Annual Congress that highlight ARCUS’ differentiated ability to make efficient, durable, and targeted insertion edits in non-human primates (NHPs), as well as preclinical data demonstrating the potential of ARCUS to achieve large excision of a commonly mutated region of the dystrophin gene for the potential treatment of Duchenne muscular dystrophy (DMD)..

DURHAM，N.C.-（BUSINESS WIRE）-Precision BioSciences，Inc。（纳斯达克股票代码：DTIL），一家先进的基因编辑公司，利用其新颖的专有ARCUS®平台开发体内基因编辑疗法，用于复杂的基因编辑，包括基因插入，切除和消除，今天宣布，该公司将在ESGCT第30届年会上发表两张海报，强调ARCU在非人类灵长类动物（NHPs）中进行高效，持久和有针对性的插入编辑的不同能力，以及临床前数据，这些数据表明ARCU有可能实现肌营养不良蛋白基因常见突变区域的大切除，从而有可能治疗杜氏肌营养不良症（DMD）。。

“We believe ARCUS has the potential to redefine gene editing by enabling sophisticated gene edits such as gene insertion, excision, and elimination,” said Jeff Smith, Chief Research Officer of Precision BioSciences. “ARCUS’ ability to create 4 base pair, 3’ overhangs is designed to make predictable, highly consistent, therapeutic edits and enable high efficiency gene insertion through homology directed repair (HDR).

Precision BioSciences首席研究官杰夫·史密斯（Jeff Smith）说：“我们相信ARCUS有可能通过进行复杂的基因编辑（例如基因插入，切除和消除）来重新定义基因编辑。“ARCUS”创建4个碱基对，3个突出端的能力旨在进行可预测，高度一致的治疗性编辑，并通过同源性定向修复（HDR）实现高效基因插入。

In preclinical work presented today at ESGCT, ARCUS demonstrated 17 times higher gene insertion efficiency compared to CRISPR-Cas9, validating the importance of the cut made by ARCUS. In addition, data presented at ESGCT continue to support ongoing development of our gene excision program focused on DMD, in development with Prevail Therapeutics, a wholly-owned subsidiary of Eli Lilly and Company.”.

在ESGCT今天提出的临床前工作中，ARCU的基因插入效率比CRISPR-Cas9高17倍，证实了ARCU切割的重要性。此外，ESGCT提供的数据继续支持我们针对DMD的基因切除计划的持续发展，并与礼来公司（Eli Lilly and Company）的合资子公司Prevail Therapeutics一起开发。

Title: Unique features of ARCUS nucleases enable high efficiency, targeted gene insertion in vivo

题目：ARCUS核酸酶的独特功能可在体内实现高效，靶向的基因插入

Poster Number: #P641

海报编号：＃P641

Presenter: Cassie Gorsuch, VP of Gene Therapy, Precision Biosciences

主持人：Cassie Gorsuch，精准生物科学基因治疗副总裁

Date and Time: Wednesday, October 25, 2023, 5:00 PM - 6:15 PM CEST and Thursday, October 26, 2023, 8:30 PM - 9:30 PM CEST

日期和时间：2023年10月25日，星期三，下午5:00，下午6:15和2023年十月26日，星期四，晚上8:30，晚上9:30

Location: Gare Maritime

地点：Gare Maritime

In preclinical work presented today, ARCUS showed high efficiency gene insertion in vitro (primary human T cells and hepatocytes) and in vivo (infant and adult NHPs). ARCUS’ 3’ overhangs were shown to drive high efficiency gene insertion compared to blunt cuts, and in vivo ARCUS demonstrated high efficiency and durable insertion in newborn and infant NHPs when used with OTC and F9 transgene insertion templates after adeno-associated virus (AAV) delivery.

在当今提出的临床前工作中，ARCU在体外（原代人T细胞和肝细胞）和体内（婴儿和成人NHP）显示出高效的基因插入。与钝性切割相比，ARCUS'3'突出端显示出驱动高效基因插入，并且当在腺相关病毒（AAV）后与OTC和F9转基因插入模板一起使用时，体内ARCUS在新生儿和婴儿NHP中表现出高效和持久的插入。）交货。

ARCUS showed up to ~45% insertion when administered by LNP along with an AAV carrying a gene insertion template in adult NHPs, and demonstrated high efficiency gene insertion via HDR in nondividing, primary human hepatocytes..

当通过LNP与携带基因插入模板的AAV一起在成人NHP中施用时，ARCUS显示高达~45%的插入，并且在非分裂的原代人肝细胞中证明了通过HDR的高效基因插入。。

Title: ARCUS-mediated excision of the “hot spot” region of the human dystrophin gene results in functional improvement in a mouse model of Duchenne muscular dystrophy (DMD)

题目：ARCUS介导的人肌营养不良蛋白基因“热点”区域的切除导致杜兴氏肌营养不良症（DMD）小鼠模型的功能改善

Poster Number: Poster #P653

海报编号：海报＃P653

Presenter: Cassie Gorsuch, VP of Gene Therapy, Precision Biosciences

主持人：Cassie Gorsuch，精准生物科学基因治疗副总裁

Date and Time: Wednesday, October 25, 2023, 5:00 PM - 6:15 PM CEST and Thursday, October 26, 2023, 8:30 PM - 9:30 PM CEST

日期和时间：2023年10月25日，星期三，下午5:00，下午6:15和2023年十月26日，星期四，晚上8:30，晚上9:30

Location: Gare Maritime

地点：Gare Maritime

In the data on display today using early generation ARCUS nucleases, Precision scientists observed the edited dystrophin protein variant in multiple tissue types frequently involved in progression of DMD, including heart, diaphragm, and skeletal muscle. Furthermore, the maximum force output of the gastrocnemius muscle in ARCUS-treated animals was significantly improved compared to untreated mice, reaching 86% of the maximum force output levels observed in non-diseased, control animals..

在当今使用早期ARCUS核酸酶显示的数据中，精密科学家观察到了在多种组织类型中编辑的肌营养不良蛋白变体，这些组织类型经常参与DMD的进展，包括心脏，diaphragm肌和骨骼肌。此外，与未治疗的小鼠相比，经ARCUS治疗的动物腓肠肌的最大力量输出显着改善，达到了未患病的对照动物中观察到的最大力量输出水平的86%。。

About Duchenne muscular dystrophy

关于杜兴氏肌肉营养不良症

DMD is a genetic disorder associated with mutations in the dystrophin gene that prevent production of the dystrophin protein. Dystrophin stabilizes the cell membrane during muscle contraction to prevent damage, and the absence of intact dystrophin protein leads to inflammation, fibrosis, and progressive loss of muscle function and mass.

DMD是与肌营养不良蛋白基因突变相关的遗传性疾病，可阻止肌营养不良蛋白的产生。肌营养不良蛋白在肌肉收缩过程中稳定细胞膜以防止损伤，而完整的肌营养不良蛋白的缺乏会导致炎症，纤维化以及肌肉功能和质量的逐步丧失。

Over time, children with DMD will develop problems walking and breathing, eventually leading to death in the second or third decade of life due to progressive cardiomyopathy and respiratory insufficiency. DMD occurs in 1 in 3,500 to 5,000 male births, and currently there are limited approved therapies available for patients..

随着时间的流逝，患有DMD的儿童将出现行走和呼吸问题，最终由于进行性心肌病和呼吸功能不全而在生命的第二个或第三个十年中死亡。DMD发生在3500至5000例男性婴儿中，其中1例发生，目前可用于患者的批准疗法有限。。

About ARCUS

关于ARCUS

ARCUS is a proprietary genome editing technology discovered and developed by scientists at Precision BioSciences. It uses sequence-specific DNA-cutting enzymes, or nucleases, that are designed to either insert (knock-in), excise (knock-out), eliminate, or repair DNA of living cells and organisms. ARCUS is based on a naturally occurring genome editing enzyme, I-CreI, that evolved in the algae Chlamydomonas reinhardtii to make highly specific cuts in cellular DNA and stimulate gene insertion at the cut site by homologous recombination.

ARCUS是由Precision BioSciences的科学家发现和开发的专有基因组编辑技术。它使用序列特异性DNA切割酶或核酸酶，设计用于插入（敲入），切除（敲除），消除或修复活细胞和生物体的DNA。ARCUS基于天然存在的基因组编辑酶I-CreI，其在藻类莱茵衣藻中进化以在细胞DNA中进行高度特异性切割并通过同源重组刺激切割位点处的基因插入。

Precision's platform and products are protected by a comprehensive portfolio including nearly 100 patents to date..

Precision的平台和产品受到全面产品组合的保护，其中包括迄今为止近100项专利。。

About Precision BioSciences, Inc.

关于精密生物科学公司。

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.是一家先进的基因编辑公司，致力于改善生命（DTIL），其新颖专有的ARCUS®基因组编辑平台在切割方式，体积更小，结构更简单等方面与其他技术不同。关键功能和区别特征可能使ARCUS核酸酶能够驱动更多预期的，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，旨在为没有适当治疗方法的最广泛的遗传和传染病提供持久的治疗。有关Precision BioSciences的更多信息，请访问www.precisionbiosciences.com。。

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 expected conference participation and disclosure of preclinical data, the ability of ARCUS to make predictable, highly consistent, therapeutic edits as well as durable, high-efficiency gene insertion capabilities through HDR, including NHPs, the potential to redefine gene editing by enabling sophisticated gene edits such as gene insertion, excision, and elimination, the clinical development, nomination, and goals of our PBGENE-DMD program, therapeutic potential of an ARCUS gene editing approach for the treatment of DMD, including ability of ARCUS to achieve large gene excisions, enabling significant functional muscle improvement in a DMD mouse model, and expected safety, efficacy, and benefit of our gene editing approaches.

本新闻稿包含1995年“私人证券诉讼改革法”含义内的前瞻性声明。本新闻稿中包含的与历史事实事项无关的所有声明均应视为前瞻性声明，包括但不限于有关预期会议参与和临床前数据披露的声明，ARCU做出可预测，高度一致的能力，治疗编辑以及耐用，通过HDR（包括NHP）实现高效基因插入功能，通过启用复杂的基因编辑（例如基因插入，切除和消除）重新定义基因编辑的潜力，我们PBGENE-DMD计划的临床开发，提名和目标，治疗潜力ARCUS基因编辑方法治疗DMD，包括ARCU实现大基因切除的能力，在DMD小鼠模型中实现显着的功能性肌肉改善以及我们的基因编辑方法的预期安全性，有效性和益处。

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

在某些情况下，您可以通过“目标”，“预期”，“方法”，“相信”，“沉思”，“可以”，“估计”，“期望”，“目标”，“意图”，“看”，“可能”，“使命”，“计划”，“可能”，“潜力”，“预测”，“追求”，“应该”，“目标”，“将会”或否定的，类似的词语和表达。。

Forward-looking statements are based on management’s current expectations, beliefs and assumptions and on information currently available to us. Such statements are subject to 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, the important factors discussed under the caption “Risk Factors” in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2023, as any such factors may be updated from time to time in our other filings with the SEC, which are accessible on the SEC’s website at www.sec.gov and the Investors page of our website under SEC Filings at investor.precisionbiosciences.com..

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。此类陈述受制于许多已知和未知的风险，不确定性和假设，实际结果可能与表达或暗示的结果大不相同由于各种重要因素，在前瞻性陈述中，包括，但不限于我们在2023年6月30日结束的季度表格10-Q的季度报告中标题为“风险因素”下讨论的重要因素，因为任何此类因素都可能在我们的其他文件中不时更新与SEC，可在美国证券交易委员会网站www.SEC.gov和我们网站的投资者页面上的investor.precisionbiosciences.com网站上的SEC Filings访问。。

All forward-looking statements speak only as of the date of this press release and, except as required by applicable law, we have no obligation to update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise..

所有前瞻性陈述仅在本新闻稿发布之日才发表，除适用法律要求外，我们没有义务更新或修改此处包含的任何前瞻性陈述，无论是由于任何新信息，未来事件，改变的情况或其他。。