精准生物科学的基因编辑平台具有差异化机制，具有潜在竞争力：分析师-动脉网

Precision BioSciences' Gene Editing Platform Has Differentiated Mechanism, And Is Potentially Competitive: Analyst

benzinga 等信源发布 2025-01-10 14:18

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On Thursday,

周四，

Precision BioSciences Inc.’s

精密生物科学公司

DTIL

partner iECURE reported clinical efficacy and safety data in the first patient dosed with ECUR-506 in the

partner iCure报告了第一例服用ECUR-506的患者的临床疗效和安全性数据

Phase 1/2 OTC-HOPE study

第1/2阶段OTC-HOPE研究

ECUR-506 is iECURE’s in vivo gene insertion program designed to treat neonatal-onset Ornithine Transcarbamylase (OTC) deficiency. It utilizes a PCSK9-specific ARCUS nuclease, licensed from Precision, that enables the insertion of a functional copy of the OTC gene.

ECUR-506是iCure的体内基因插入程序，旨在治疗新生儿发作的鸟氨酸转氨甲酰酶（OTC）缺乏症。它利用了从Precision获得许可的PCSK9特异性ARCUS核酸酶，可以插入OTC基因的功能拷贝。

Ornithine transcarbamylase deficiency is a rare genetic condition that causes ammonia to build up in the blood. The condition is more common in boys than girls and tends to be more severe when symptoms emerge shortly after birth.

鸟氨酸转氨甲酰酶缺乏症是一种罕见的遗传病，会导致血液中产生氨。这种情况在男孩中比女孩更常见，并且在出生后不久出现症状时往往更为严重。

Treatment with ECUR-506 was generally well tolerated in first infant dosed with no significant clinical safety concerns apart from asymptomatic transaminitis at four weeks.

在第一个服用ECUR-506的婴儿中，ECUR-506的治疗通常耐受性良好，除了在四周时无症状的转氨酶外，没有明显的临床安全性问题。

BMO Capital Markets

蒙特利尔银行资本市场

upgraded Precision BioSciences, “Acknowledging the N=1, iECURE data appear very promising, providing early clinical validation for ARCUS.”

升级的Precision BioSciences，“承认N=1，iCure数据看起来非常有希望，为ARCUS提供了早期临床验证。”

Analyst Kostas Biliouris writes that early ECUR-506 efficacy provides initial clinical derisking of in vivo ARCUS efficacy/editing platform.

分析师Kostas Biliouris写道，早期ECUR-506的功效提供了体内ARCUS功效/编辑平台的初步临床衍生。

In November, Precision BioSciences shared preclinical data for the PBGENE-HBV program that reinforced the safety profile and potential to deliver a functional cure for chronic hepatitis B, supporting advancement into first-in-human clinical study.

11月，Precision BioSciences分享了PBGENE-HBV项目的临床前数据，该项目增强了慢性乙型肝炎功能性治疗的安全性和潜力，支持了人类临床研究的首次进展。

The company is conducting ELIMINATE-B Phase 1 trial. ELIMINATE-B is open and currently screening and accruing patients, and Precision expects to report data from the study as it

该公司正在进行EXCLUSE-B第一阶段试验。ELIMINATE-B是开放的，目前正在筛查和累积患者，Precision预计会报告研究数据

matures throughout 2025

2025年到期

BMO Capital analyst writes that the HBV readout in 2025 could drive a 100-200%+ stock upside.

BMO Capital分析师写道，2025年的HBV读数可能会推动股票上涨100-200%。

On Thursday, BMO upgraded from Market Perform to Outperform with a

price target of $34

价格目标为34美元

based on ECUR-506 initial clinical derisking of in vivo ARCUS editing and a proprietary gene editing platform with a differentiated mechanism and potentially competitive.

基于ECUR-506体内ARCUS编辑的初步临床衍生和具有差异化机制和潜在竞争性的专有基因编辑平台。

Price Action:

价格行动：

DTIL stock is up 25.8% at $5.66 at last check Friday.

DTIL股价周五上涨25.8%，至5.66美元。