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, today announced that the company will present preclinical data for its PBGENE-HBV clinical candidate at the European Association for the Study of the Liver Congress (EASL), highlighting the differentiated ability of ARCUS to make efficient, durable, and targeted elimination edits.

北卡罗来纳州达勒姆市——（商业新闻短讯）——Precision BioSciences，Inc.（纳斯达克：DTIL）是一家先进的基因编辑公司，利用其新型专有ARCUS®平台开发体内基因编辑疗法，今天宣布该公司将在欧洲肝脏研究协会（EASL）上提供其PBGENE-HBV临床候选人的临床前数据，突出了ARCUS进行高效，持久和有针对性的消除编辑的差异化能力。

The poster presentation highlights data in primary human hepatocytes demonstrating the high specificity and lack of detectable off-target editing for PBGENE-HBV at therapeutically relevant doses. The poster will also showcase non-human primate data demonstrating good tolerability of a multi-dosing approach for the treatment of chronic hepatitis B..

海报展示突出了原代人肝细胞中的数据，证明了在治疗相关剂量下PBGENE-HBV的高特异性和缺乏可检测的脱靶编辑。海报还将展示非人灵长类动物数据，证明多剂量方法治疗慢性乙型肝炎具有良好的耐受性。。

“We are pleased to present new safety data at EASL for our lead PBGENE-HBV program. These data highlight our ability to specifically target the HBV viral genome, with no detectable off-target editing in the human genome at relevant doses. In addition, the new non-human primate data shows that multiple administrations of PBGENE-HBV are well tolerated, enabling us to multi-dose in clinic,” said Jeff Smith, PhD, Chief Research Officer of Precision BioSciences.

Precision BioSciences首席研究官杰夫·史密斯（Jeff Smith）博士说：“我们很高兴在EASL上为我们的主要PBGENE-HBV项目提供新的安全性数据。这些数据突出了我们专门针对HBV病毒基因组的能力，在相关剂量下人类基因组中没有可检测到的脱靶编辑。此外，新的非人类灵长类动物数据显示，多次服用PBGENE-HBV的耐受性良好，使我们能够在临床上进行多剂量服用。”。

“Our work to date continues to highlight the potential of PBGENE-HBV as a curative treatment for chronic hepatitis B and further validates the capabilities of our proprietary ARCUS® platform to deliver safe and efficacious in vivo gene editing therapies. Looking ahead, we are well positioned to progress PBGENE-HBV, our first wholly owned ARCUS program into the clinic with an Investigational New Drug Application (IND) and/or Clinical Trial Application (CTA) in the 2024.”.

“迄今为止，我们的工作继续突出了PBGENE-HBV作为慢性乙型肝炎治疗药物的潜力，并进一步验证了我们专有的ARCUS®平台提供安全有效的体内基因编辑疗法的能力。展望未来，我们将在2024年通过研究性新药申请（IND）和/或临床试验申请（CTA）将PBGENE-HBV这一我们第一个全资拥有的ARCUS项目推向临床。”。

The data to be presented highlights that PBGENE-HBV specifically cuts HBV DNA leading to elimination of cccDNA and inactivation of integrated HBV DNA without impacting any sites in the human genome, including no editing-associated translocations in HBV infected primary human hepatocytes. In addition, PBGENE-HBV was well-tolerated in non-human primates across multiple dose administrations, with only minor and transient elevations in liver transaminases that are normalized within 2 weeks and non-adverse changes in blood parameters.

所提供的数据强调，PBGENE-HBV特异性切割HBV DNA，导致cccDNA的消除和整合的HBV DNA的失活，而不影响人类基因组中的任何位点，包括在HBV感染的原代人肝细胞中没有编辑相关的易位。此外，PBGENE-HBV在多次给药的非人灵长类动物中耐受性良好，肝转氨酶只有轻微和短暂的升高，在2周内恢复正常，血液参数无不良变化。

Preclinical safety data supports the advancement of PBGENE-HBV to clinical trials as a potentially curative treatment for chronic hepatitis B..

临床前安全性数据支持PBGENE-HBV作为慢性乙型肝炎的潜在治疗方法进入临床试验。。

Full poster details are included below:

完整的海报细节如下：

Title: Preclinical safety data for PBGENE-HBV gene editing program supports advancement to clinical trials as a potentially curative treatment for chronic hepatitis B

标题：PBGENE-HBV基因编辑程序的临床前安全性数据支持临床试验的进展，作为慢性乙型肝炎的潜在治疗方法

Abstract Number: LB195

摘要编号：LB195

Presenter: Emily Harrison, PhD, Senior Scientist - Hepatitis Research Leader, Precision BioSciences

主持人：艾米丽·哈里森博士，精密生物科学公司肝炎研究负责人高级科学家

Date and Time: Wednesday, June 5, 2024, 8:30 AM – Saturday, June 8, 2024, 5:00 PM CEST

日期和时间：2024年6月5日星期三上午8:30至2024年六月8日星期六下午5:00

About Hepatitis B and PBGENE-HBV:

关于乙型肝炎和PBGENE-HBV：

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 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。

PBGENE-HBV is a highly specific, novel therapeutic approach to treating patients with chronic HBV infection. It’s designed to directly eliminate cccDNA and inactivate integrated HBV DNA with high specificity, potentially leading to functional cures..

PBGENE-HBV是治疗慢性HBV感染患者的一种高度特异性的新型治疗方法。它旨在直接消除cccDNA并以高特异性灭活整合的HBV DNA，可能导致功能性治愈。。

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 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）。。