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 that it has received Clinical Trial Application (CTA) approval in Moldova for its lead candidate, PBGENE-HBV.

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PBGENE-HBV is Precision’s wholly owned in vivo gene editing program designed to cure chronic hepatitis B by eliminating cccDNA, the key source of replicating hepatitis B virus (HBV), while also inactivating integrated HBV DNA in hepatocytes. The company has opened the Phase 1 clinical program and is moving towards dosing patients..

PBGENE-HBV是Precision全资拥有的体内基因编辑程序，旨在通过消除复制乙型肝炎病毒（HBV）的关键来源cccDNA来治疗慢性乙型肝炎，同时还可以灭活肝细胞中整合的HBV DNA。该公司已经启动了第一阶段临床计划，并正在朝着给患者服用药物的方向发展。。

“At Precision, we remain laser-focused on execution, and our first CTA approval for PBGENE-HBV represents another significant milestone as we complete our transition to a clinical stage in vivo gene editing company. With rapid regulatory approval in hand, we are working diligently to screen and dose patients at our first clinical site in Moldova,” said Michael Amoroso, Chief Executive Officer of Precision BioSciences.

Precision BioSciences首席执行官迈克尔·阿莫罗索（MichaelAmoroso）表示：“在Precision，我们仍然专注于执行，我们对PBGENE-HBV的首次CTA批准代表了另一个重要里程碑，因为我们完成了向临床阶段体内基因编辑公司的过渡。随着监管部门的快速批准，我们正在努力在摩尔多瓦的第一个临床站点筛查和给患者服用剂量。”。

“In parallel, we are leveraging our robust regulatory package, which highlights the safety and potent antiviral effects of PBGENE-HBV in a variety of models, including non-human primates, to pursue additional CTA and IND approvals globally. This multi-track approach will enable us to accelerate enrollment into the trial and generate important clinical safety and efficacy data with the aim of bringing a potentially curative treatment to the nearly 300 million patients living with chronic hepatitis B globally.”.

“与此同时，我们正在利用我们强大的监管方案，该方案强调了PBGENE-HBV在包括非人类灵长类动物在内的多种模型中的安全性和有效抗病毒作用，以在全球范围内寻求额外的CTA和IND批准。这种多轨方法将使我们能够加速参加试验，并产生重要的临床安全性和有效性数据，旨在为全球近3亿慢性乙型肝炎患者带来潜在的治疗方法。”。

Prior to PBGENE-HBV, no other modality has been investigated in a clinical trial setting that is designed to eliminate the root cause of disease, the cccDNA. Additionally, the current standard of care requires daily chronic treatment with nucleos(t)ide analogs and only offers patients a 1-3% chance of functional cure.

在PBGENE-HBV之前，尚未在旨在消除疾病根本原因cccDNA的临床试验环境中研究其他方式。。

Through its precision cutting, compact design, and simple structure, PBGENE-HBV is engineered to target the HBV viral genome and drive functional cures for patients with chronic hepatitis B. PBGENE-HBV leverages the ARCUS® gene editing platform by delivering an ARCUS nuclease-encoding mRNA to the liver via lipid nanoparticles.

通过其精确切割，紧凑设计和简单结构，PBGENE-HBV被设计为靶向HBV病毒基因组并驱动慢性乙型肝炎患者的功能性治疗。PBGENE-HBV利用ARCUS®基因编辑平台，通过脂质纳米颗粒将ARCUS核酸酶编码mRNA递送至肝脏。

When expressed in HBV-infected hepatocytes, the ARCUS nuclease specifically cuts a highly conserved sequence in the hepatitis B viral genome and is designed to eliminate cccDNA and inactivate integrated HBV genomes. The ARCUS platform is derived from a naturally occurring enzyme and has been optimized for over 20 years into a ground-breaking and highly precise gene editing tool..

当在HBV感染的肝细胞中表达时，ARCUS核酸酶特异性切割乙型肝炎病毒基因组中高度保守的序列，并被设计用于消除cccDNA并使整合的HBV基因组失活。ARCUS平台源自一种天然存在的酶，经过20多年的优化，已成为一种突破性且高度精确的基因编辑工具。。

“We are thrilled to receive CTA approval in Moldova given the high prevalence of chronic hepatitis B in the country and their expertise gained from multiple Phase 1 clinical trials in hepatitis B. Our clinical team, guided by our hepatitis scientific advisory board, has deep expertise in hepatitis B, and we believe that we are well-positioned to advance PBGENE-HBV towards dosing the first patient in the clinic,” added Dr.

“鉴于摩尔多瓦慢性乙型肝炎的高发病率以及他们从乙型肝炎的多个1期临床试验中获得的专业知识，我们很高兴在摩尔多瓦获得CTA的批准。我们的临床团队在我们的肝炎科学咨询委员会的指导下，在乙型肝炎方面拥有丰富的专业知识，我们相信我们有能力推动PBGENE-HBV在临床上为第一位患者服用，”Dr。

Murray Abramson, Senior Vice President, Head of Clinical Development of Precision BioSciences. “PBGENE-HBV is the first and only clinical stage approach to directly target and eliminate cccDNA, giving it the potential to achieve a functional cure. Next month, we plan to share additional information about our Phase 1 program, including the final preclinical safety package, with investigators and investors ahead of the upcoming AASLD meeting.

Murray Abramson，高级副总裁，精密生物科学临床开发负责人。“PBGENE-HBV是第一个也是唯一一个直接靶向和消除cccDNA的临床阶段方法，使其有可能实现功能性治愈。下个月，我们计划在即将举行的AASLD会议之前，与研究人员和投资者分享有关我们第一阶段计划的更多信息，包括最终的临床前安全包。

With a clear regulatory pathway ahead of us, and our deep clinical expertise, we are focused on rapidly enrolling patients into the study and generating robust clinical data in patients with chronic hepatitis B who have for too long been unable to achieve meaningful functional cures.”.

有了明确的监管途径，以及我们深厚的临床专业知识，我们专注于快速将患者纳入研究，并为长期无法实现有意义的功能治疗的慢性乙型肝炎患者提供强大的临床数据。”。

Precision has submitted multiple global clinical trial applications and is on track to submit additional CTA and IND filings as part of its global Phase 1 regulatory strategy for PBGENE-HBV. The company will provide updates as it receives additional regulatory approvals to begin treating patients in those markets.

Precision已经提交了多项全球临床试验申请，并正在按计划提交额外的CTA和IND文件，作为其PBGENE-HBV全球1期监管策略的一部分。该公司将提供最新信息，因为它将获得额外的监管批准，开始在这些市场治疗患者。

The company plans to provide an update on the PBGENE-HBV program on November 15th before the American Association for the Study of Liver Diseases (AASLD) Annual Meeting in November. Detailed information on how to join the webcast will be provided in the future..

该公司计划在11月15日美国肝病研究协会（AASLD）年会之前提供PBGENE-HBV计划的最新信息。未来将提供有关如何加入网络广播的详细信息。。

About Hepatitis B:

关于乙型肝炎：

Hepatitis B is a leading cause of morbidity in the US and death globally, with no curative options currently available for patients. In 2019, despite the availability of approved antiviral therapies, an estimated 300 million people globally and more than 1 million people in the US were estimated to have chronic hepatitis B infection.

乙型肝炎是美国发病率和全球死亡的主要原因，目前患者尚无治疗选择。2019年，尽管获得了批准的抗病毒治疗，但据估计，全球约有3亿人和美国超过100万人患有慢性乙型肝炎感染。

An estimated 15% to 40% of patients with HBV infections may develop complications, such as cirrhosis, liver failure, or liver cancer (hepatocellular carcinoma), which account for the majority of HBV-related deaths..

据估计，15%至40%的HBV感染患者可能会出现并发症，如肝硬化，肝衰竭或肝癌（肝细胞癌），这是HBV相关死亡的主要原因。。

Chronic hepatitis B infection is primarily driven by persistence of HBV cccDNA and integration of HBV DNA into the human genome in liver cells, the primary source of hepatitis B surface antigen (HBsAg) in late-stage disease. Current treatments for patients with HBV infection include agents that result in long-term viral suppression as indicated by reduction of circulating HBV DNA, but these therapies do not eradicate HBV cccDNA, rarely lead to functional cure, and require lifelong administration..

慢性乙型肝炎感染主要由HBV cccDNA的持续存在和HBV DNA整合到肝细胞中的人类基因组中驱动，肝细胞是晚期疾病中乙型肝炎表面抗原（HBsAg）的主要来源。目前对HBV感染患者的治疗包括导致长期病毒抑制的药物，如循环HBV DNA减少所示，但这些疗法不能根除HBV cccDNA，很少导致功能性治愈，需要终身服用。。

About PBGENE-HBV:

关于PBGENE-HBV：

PBGENE-HBV is a potentially curative approach to treating patients with chronic HBV infection through a highly specific, novel therapeutic approach. PBGENE leverages the ARCUS® platform and is designed to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures..

PBGENE-HBV是一种通过高度特异性的新型治疗方法治疗慢性HBV感染患者的潜在治疗方法。PBGENE利用ARCUS®平台，旨在直接消除cccDNA并以高特异性灭活整合的HBV DNA，从而可能导致功能性治愈。。

About Precision BioSciences, Inc.

关于Precision BioSciences，Inc。

Precision BioSciences, Inc. is a clinical stage 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.是一家临床阶段基因编辑公司，致力于改善生活（DTIL），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 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 (including PBGENE-HBV) and gene editing approaches including editing efficiency; the design of PBGENE-HBV to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures or providing a better chance of functional cures; 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; the expected timing of regulatory processes (including filings such as IND’s and CTA’s and studies for PBGENE-HBV and the acceptance of these filings by regulatory agencies); the translation of preclinical safety and efficacy studies and models to safety and efficacy in humans, the suitability of PBGENE-HBV for the treatment of hepatitis and the targeting of the root cause of the disease, expectations about operational initiatives, strategies, and further development of our programs; expectations about achievement of key milestones; and anticipated timing of clinical data.

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声明都应被视为前瞻性声明，包括但不限于关于我们候选产品（包括PBGENE-HBV）的临床开发和预期安全性，有效性和益处的声明）和基因编辑方法，包括编辑效率；设计PBGENE-HBV以直接消除cccDNA并以高特异性灭活整合的HBV DNA，可能导致功能性治愈或提供更好的功能性治愈机会；ARCUS核酸酶由于其小尺寸，简单性和独特的切割而适用于基因消除，插入和切除以及与其他基因编辑方法的区别；监管过程的预期时间（包括IND和CTA等备案以及PBGENE-HBV研究以及监管机构对这些备案的接受）；将临床前安全性和有效性研究和模型转化为人类的安全性和有效性，PBGENE-HBV治疗肝炎的适用性和疾病根本原因的针对性，对运营举措，策略的期望以及我们计划的进一步发展；；以及临床数据的预期时间。

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,” “w.

在某些情况下，您可以通过“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”、“w”等术语来识别前瞻性陈述。

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。

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..

所有前瞻性声明仅在本新闻稿发布之日有效，除适用法律要求外，我们没有义务更新或修订本新闻稿中包含的任何前瞻性声明，无论是由于任何新信息、未来事件、情况变化或其他原因。。