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- Approval in Hong Kong marks the first clinical trial application clearance of an in vivo gene editing approach for chronic hepatitis B in Hong Kong and the second CTA approval for PBGENE-HBV in 2024

-香港的批准标志着香港首次批准慢性乙型肝炎体内基因编辑方法的临床试验应用，以及2024年CTA第二次批准PBGENE-HBV

- ELIMINATE-B is a global, multi-site study now actively recruiting patients; expected to report clinical data as it matures throughout 2025

-EXCLEATE-B是一项全球多地点研究，目前正在积极招募患者；随着2025年临床数据的成熟，预计将报告临床数据

- U.S. investigational new drug (IND) anticipated in 2025

-美国研究性新药（IND）预计2025年上市

DURHAM, N.C.--(BUSINESS WIRE)--Dec. 18, 2024--

北卡罗来纳州达勒姆——（商业新闻短讯）——2024年12月18日--

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 Hong Kong to study PBGENE-HBV in the ongoing ELIMINATE-B Phase I trial.

Precision BioSciences，Inc.（纳斯达克股票代码：DTIL）是一家临床阶段基因编辑公司，利用其新颖的专有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，它已在香港获得临床试验应用（CTA）批准，在正在进行的EXCLEATE-B I期试验中研究PBGENE-HBV。

PBGENE-HBV is Precision’s lead 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), and inactivating integrated HBV DNA in hepatocytes. The company is actively recruiting patients for the ELIMINATE-B trial in Moldova and has begun activating a top infectious disease clinical site in Hong Kong as part of its global regulatory and clinical operations strategy..

PBGENE-HBV是Precision的领先全资体内基因编辑程序，旨在通过消除cccDNA（复制乙型肝炎病毒（HBV）的关键来源）和灭活肝细胞中整合的HBV DNA来治疗慢性乙型肝炎。。。

“We are pleased to receive our second CTA approval for PBGENE-HBV, expanding our ELIMINATE-B trial into a world-class HBV clinical trial site in Hong Kong,” said Dr. Murray Abramson, Senior Vice President, Head of Clinical Development of Precision BioSciences. “PBGENE-HBV has been designed to target a viral site that is prevalent across all HBV genotypes, including in Asia, and we believe that this will enable us to evaluate and provide access to as many patients as possible.

Precision BioSciences高级副总裁兼临床开发负责人Murray Abramson博士说：“我们很高兴获得CTA对PBGENE-HBV的第二次批准，将我们的EXCLEATE-B试验扩展到香港的世界级HBV临床试验地点。”。“PBGENE-HBV旨在针对包括亚洲在内的所有HBV基因型中普遍存在的病毒位点，我们相信这将使我们能够评估并提供尽可能多的患者。

In addition to Hong Kong, our clinical trial site in Moldova continues to execute as planned, and we look forward to sharing clinical data as it matures throughout 2025.”.

除香港外，我们在摩尔多瓦的临床试验基地继续按计划执行，随着2025年临床数据的成熟，我们期待着共享临床数据。”。

“Over 400,000 individuals in Hong Kong are currently living with chronic hepatitis B, and many patients continue to develop liver cancer or cirrhosis even on existing long-term treatment with standard of care treatments,” said Dr. MF Yuen, DSc, MD, PhD, Chair Professor of Gastroenterology and Hepatology, Li Shu Fan Medical Foundation and Professor in Medicine, The University of Hong Kong.

“香港目前有40多万人患有慢性乙型肝炎，许多患者甚至在现有的长期标准治疗下仍继续发展为肝癌或肝硬化，”香港大学李书凡医学基金会胃肠学和肝病学主席教授、DSc医学博士、博士MF Yuen博士说。

“By targeting and eliminating covalently closed circular (ccc) DNA, the root cause of the disease, PBGENE-HBV has the potential to completely eliminate the source of viral replication from the body, not just reduce it, which could transform the treatment landscape for patients in Hong Kong and millions of others worldwide.

“通过靶向和消除共价闭合环状（ccc）DNA（疾病的根本原因），PBGENE-HBV有可能完全消除体内病毒复制的来源，而不仅仅是减少它，这可能会改变香港和全球数百万患者的治疗格局。

Our field has long searched for a novel way to eliminate the root cause of chronic hepatitis B, the cccDNA, and I look forward to further investigating PBGENE-HBV in clinic.”.

我们的领域长期以来一直在寻找一种新的方法来消除慢性乙型肝炎的根本原因，即cccDNA，我期待着在临床上进一步研究PBGENE-HBV。”。

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。

The ARCUS nuclease specifically cuts a highly conserved sequence in the hepatitis B viral genome and is designed to eliminate the root cause of the disease, 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..

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，旨在消除疾病的根本原因，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。。

Precision has submitted multiple global clinical trial applications and remains on track for a U.S. IND in 2025 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，并将在2025年成为美国IND的一部分，作为其PBGENE-HBV全球1期监管战略的一部分。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。。

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.

。

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); the unique design of PBGENE-HBV to eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures; the expected timing of regulatory processes (including filings such as IND’s and CTA’s for PBGENE-HBV and the acceptance of these filings by regulatory agencies); the suitability of PBGENE-HBV for the treatment of hepatitis and the targeting of the root cause of the disease; the key advantages of ARCUS and its key capabilities and differentiating characteristics ; expectations about operational initiatives, strategies, and further development of PBGENE-HBV; and anticipated timing of patient dosing and 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均应视为前瞻性声明，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；PBGENE-HBV的独特设计可消除cccDNA并以高特异性灭活整合的HBV DNA，可能导致功能性治愈；监管过程的预期时间（包括PBGENE-HBV的IND和CTA等备案以及监管机构对这些备案的接受）；PBGENE-HBV治疗肝炎的适用性和针对疾病根本原因的针对性；ARCUS的关键优势及其关键功能和差异化特征；对PBGENE-HBV的运营举措，策略和进一步发展的期望；以及患者给药和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,” “design”, “designed,” “endeavor,” “engineered,” “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”、“设计”、“努力”、“设计”、“估计”、“预期”、“目标”、“打算”、“看起来”、“可能”、“使命”、“计划”、“可能”、“潜力”、“预测”、“项目”、“追求”、“应该”、“努力”、“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。

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。。

View source version on businesswire.com: https://www.businesswire.com/news/home/20241218082974/en/

在businesswire.com上查看源代码版本：https://www.businesswire.com/news/home/20241218082974/en/

Investor Contact:

投资者联系人：

Naresh Tanna

纳雷什·坦纳

Vice President, Investor Relations

投资者关系副总裁

Naresh.Tanna@precisionbiosciences.com

Naresh.Tanna@precisionbiosciences.com

Source: Precision BioSciences, Inc.

资料来源：Precision BioSciences，Inc。