Voyager在2025年AD/PD™会议上展示了针对Tau蛋白的基因治疗和抗体项目的强大临床前数据-动脉网

Voyager Presents Robust Preclinical Data from Tau Targeting Gene Therapy and Antibody Programs at AD/PD™ 2025

Voyager 等信源发布 2025-03-31 19:10

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可切换为仅中文

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- Single IV administration of tau silencing gene therapy VY1706 significantly reduced tau mRNA and protein levels, with broad brain distribution and liver de-targeting, in NHP study -

- 在非人灵长类动物研究中，单次静脉注射tau沉默基因疗法VY1706显著降低了tau mRNA和蛋白水平，具有广泛的脑部分布并减少了对肝脏的靶向性 -

- Preclinical murine data strengthen case for specifically targeting pathologic forms of tau with the clinical-stage anti-tau antibody VY7523 -

- 临床前鼠类数据加强了使用临床阶段抗tau抗体VY7523特异性靶向病理形式tau的理由 -

- Voyager to host live webcast on

- 旅行者将主持现场网络直播

April 7

4月7日

recapping key AD/PD™ 2025 data -

回顾关键的AD/PD™ 2025数据 -

LEXINGTON, Mass.

马萨诸塞州列克星敦

，

March 31, 2025

2025年3月31日

(GLOBE NEWSWIRE) --

（环球新闻网）——

Voyager Therapeutics, Inc.

Voyager Therapeutics公司

(Nasdaq: VYGR), a biotechnology company dedicated to leveraging genetics to treat neurological diseases, today reported new data from its two preclinical programs targeting tau for the treatment of Alzheimer’s disease (AD). Data on tau silencing gene therapy VY1706 and anti-tau antibody VY7523 will be presented at the upcoming 2025 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders (AD/PD™ 2025), taking place April 1-5, 2025, in Vienna.

（纳斯达克：VYGR），一家致力于利用遗传学治疗神经疾病的生物技术公司，今日公布了其针对阿尔茨海默病（AD）治疗的两个临床前项目的新数据。有关tau沉默基因疗法VY1706和抗tau抗体VY7523的数据将在即将举行的2025年阿尔茨海默病和帕金森病及相关神经系统疾病国际会议（AD/PD™ 2025）上展示，会议将于2025年4月1日至5日在维也纳举行。

Additionally, Voyager announced it will host a live webcast recapping key AD/PD™ 2025 data at .

此外，Voyager 宣布将举办一场直播网络会议，回顾 AD/PD™ 2025 的关键数据。

4:30 p.m. ET

下午4点30分（东部时间）

打开

Monday, April 7, 2025

2025年4月7日，星期一

。

“Emerging data continue to strengthen our conviction that tau is the next critical target in Alzheimer’s disease, which supports Voyager’s advancement of both antibody and gene therapy approaches,” said

“新兴数据不断加深我们的信念，即tau是阿尔茨海默病的下一个关键靶点，这支持了Voyager在抗体和基因治疗两种方法上的进展，”

Toby Ferguson

托比·弗格森

, M.D., Ph.D., Chief Medical Officer of Voyager Therapeutics. “When I look across the design of these two molecules, the preclinical data each has generated, and the emerging clinical profile for our antibody, I believe our anti-tau antibody and gene therapy each have the potential to be transformative for patients.”.

医学博士、哲学博士、Voyager Therapeutics首席医学官。“当我审视这两种分子的设计、它们各自产生的临床前数据以及我们抗体的新兴临床特征时，我相信我们的抗tau抗体和基因疗法都有可能为患者带来变革。”

VY1706 tau silencing gene therapy:

VY1706 tau沉默基因疗法：

CROSS-SPECIES BBB-PENETRANT IV-DELIVERED AAV GENE THERAPY PROVIDES BROAD AND ROBUST CNS TAU LOWERING IN TAUOPATHY MOUSE AND NON-HUMAN PRIMATE.

跨物种BBB渗透IV递送AAV基因治疗在tau蛋白病小鼠和非人灵长类动物中提供广泛且强劲的中枢神经系统tau蛋白降低。

Wencheng Liu

刘文成

, Ph.D., ID:419.

，博士，ID：419。

New preclinical data from a non-human primate (NHP) study show that a single, intravenous (IV) 1.3E13 vg/kg dose of Voyager’s tau silencing gene therapy VY1706 resulted in dose-dependent knockdown of tau mRNA (44% to 73%) and tau protein (27% to 55%). Results were sustained up to three months following dosing, and VY1706 was well-tolerated at both the 5-week and 11-week timepoints assessed in the study.

来自非人灵长类动物（NHP）研究的新临床前数据显示，单次静脉注射（IV）1.3E13 vg/kg剂量的Voyager公司tau沉默基因疗法VY1706导致了剂量依赖性的tau mRNA（44%至73%）和tau蛋白（27%至55%）的敲低。给药后结果持续长达三个月，且在研究评估的5周和11周时间点，VY1706均显示出良好的耐受性。

Broad distribution across the brain was achieved, including throughout areas impacted by AD such as the hippocampus, entorhinal cortex, temporal cortex, and frontal cortex regions, while the liver was de-targeted by approximately 30-fold compared to wild type AAV9. VY1706 is in Investigational New Drug (IND)-enabling studies with an IND filing expected in 2026..

在大脑中的广泛分布已经实现，包括受到阿尔茨海默病（AD）影响的区域，如海马体、内嗅皮层、颞叶皮层和额叶皮层区域，而与野生型AAV9相比，肝脏的靶向性降低了约30倍。VY1706目前正处于研究性新药（IND）申报支持性研究阶段，预计将在2026年提交IND申请。

VY7523 anti-tau antibody:

VY7523抗tau抗体：

DISCRIMINATION OF ANTI-TAU ANTIBODIES TARGETING DIFFERENT TAU EPITOPES BY A P301S MOUSE HIPPOCAMPAL SEEDING MODEL OF TAUOPATHY.

通过P301S小鼠海马体TAU病理播种模型区分针对不同TAU表位的抗TAU抗体。

Wencheng Liu

刘文成

, Ph.D., ID:1403.

，博士，ID：1403。

New preclinical data show that the murine version of Voyager’s VY7523 demonstrates selectivity for binding pathologic tau tangles. Additionally, Voyager assessed several anti-tau antibodies using a P301S mouse hippocampal seeding model of tau spread. Antibodies that bind the N-terminal of tau and previously failed to achieve their primary endpoints in clinical studies also failed to significantly reduce tau spread in the model.

新的临床前数据显示，Voyager公司的VY7523鼠源版本展示了对病理性tau缠结的选择性结合。此外，Voyager使用P301S小鼠海马tau传播模型评估了多种抗tau抗体。结合tau N端的抗体在之前的临床研究中未能达到其主要终点，在该模型中也未能显著减少tau的传播。

Conversely, the murine version of Voyager’s VY7523, which targets the C-terminal of tau, reduced tau spread in the model, as did a third-party mid-domain antibody that has been shown to reduce tau accumulation in a clinical trial. Voyager is currently assessing VY7523 in a multiple ascending dose trial in patients with early AD and expects initial tau positron emission tomography (PET) imaging data in the second half of 2026..

相反，Voyager公司针对tau蛋白C端的鼠源版本VY7523在模型中减少了tau的扩散，同一种已在临床试验中被证明可减少tau积累的第三方中部结构域抗体也起到了类似作用。Voyager目前正在早期阿尔茨海默病（AD）患者中进行VY7523的多剂量递增试验，并预计将在2026年下半年获得初步的tau正电子发射断层扫描（PET）成像数据。

The posters can be accessed on Voyager’s website at:

海报可以在旅行者网站上访问：

https://www.voyagertherapeutics.com/science-publications/

。

Dr. Ferguson

弗格森博士

will be speaking on two panels at AD/PD™ 2025:

将在AD/PD™ 2025的两个专题讨论会上发言：

Challenges and Opportunities for Anti-Tau Therapies in Clinical Trials – How Can We Make Hopes Come True?

临床试验中抗Tau蛋白疗法的挑战与机遇——我们如何让希望成真？

Saturday, April 5, 2025

2025年4月5日，星期六

, at

，在

2:10 p.m.

下午2点10分

in Hall A

在A厅

Drug Development and Biomarkers in Rare CNS Diseases (ALS, FTD): From Basics to Approval – How to Define Success?

药物开发和罕见中枢神经系统疾病（ALS、FTD）中的生物标志物：从基础到审批——如何定义成功？

Saturday, April 5, 2025

2025年4月5日，星期六

, at

，在

4:40 p.m.

下午4点40分

in Hall A

在A厅

Live Webcast

实时网络直播

Voyager will host a live conference call and webcast at

航行者将举办一场现场电话会议和网络直播

4:30 p.m. ET

下午4点30分（东部时间）

打开

Monday, April 7, 2025

2025年4月7日，星期一

, to discuss data from AD/PD™ 2025. A live webcast of the call will be available on the Investors section of the Voyager website at

，讨论来自AD/PD™ 2025的数据。此次电话会议的现场网络直播将在Voyager网站的投资者部分提供，网址为

https://ir.voyagertherapeutics.com/

, and a replay of the call will be available at the same link approximately two hours after its completion. The replay will be available for at least 30 days following the conclusion of the call.

，电话会议的重播将在同一链接大约两小时后提供。重播将在电话会议结束后至少30天内可用。

About VY1706

关于VY1706

VY1706 is an IV-administered gene therapy for Alzheimer’s disease that combines a potent siRNA construct to decrease the expression of tau with an IV-delivered, blood-brain barrier-penetrant TRACER™ capsid. In preclinical non-human primate (NHP) studies, a single IV administration of VY1706 was well-tolerated and resulted in dose-dependent knockdown of tau mRNA (44% to 73%) and tau protein (27% to 55%) broadly across the brain.

VY1706 是一种用于治疗阿尔茨海默病的静脉注射基因疗法，它结合了一种高效的 siRNA 构建体以减少 tau 的表达，以及一种可通过静脉递送并穿透血脑屏障的 TRACER™ 衣壳。在临床前非人灵长类动物 (NHP) 研究中，单次静脉注射 VY1706 耐受性良好，并在大脑广泛区域内导致了剂量依赖性的 tau mRNA（44% 至 73%）和 tau 蛋白（27% 至 55%）的降低。

Voyager anticipates filing an investigational new drug (IND) application with the .

Voyager 预计将向美国食品药品监督管理局提交一份新药研究（IND）申请。

U.S. Food and Drug Administration

美国食品药品监督管理局

(FDA) and a clinical trial application (CTA) with

（FDA）和临床试验申请（CTA）

Health Canada

加拿大卫生部

in 2026.

2026年。

About VY7523

关于 VY7523

VY7523 is an IV-administered, recombinant, humanized IgG4 monoclonal antibody developed to inhibit the spread of pathological tau, which is closely correlated with disease progression and cognitive decline in Alzheimer’s disease. VY7523 is selective for pathological tau and targets a specific C-terminal epitope of tau.

VY7523 是一种通过静脉注射的重组人源化 IgG4 单克隆抗体，旨在抑制病理性 tau 的扩散，这与阿尔茨海默病的疾病进展和认知能力下降密切相关。VY7523 对病理性 tau 具有选择性，并靶向 tau 的特定 C 端表位。

In preclinical in vivo studies, the murine version of VY7523 inhibited seeding and spread of pathological tau by approximately 70%. In a Phase 1, single ascending dose (SAD) clinical trial in healthy volunteers, VY7523 demonstrated a safety, tolerability, immunogenicity, and pharmacokinetic profile warranting advancement.

在临床前体内研究中，鼠源版本的VY7523抑制了病理性tau蛋白的播种和扩散约70%。在一项针对健康志愿者的1期单次递增剂量（SAD）临床试验中，VY7523展示了安全性、耐受性、免疫原性和药代动力学特性，值得进一步推进。

Voyager is currently assessing VY7523 in a multiple ascending dose trial in patients with early Alzheimer’s disease and expects initial tau PET imaging data in the second half of 2026..

Voyager目前正在早期阿尔茨海默病患者中进行多剂量递增试验以评估VY7523，并预计将在2026年下半年获得初步的tau PET成像数据。

About Alzheimer’s Disease

关于阿尔茨海默病

Alzheimer’s disease is a progressive neurodegenerative disease estimated to affect 7 million people in the U.S.

阿尔茨海默病是一种渐进性神经退行性疾病，据估计会影响美国 700 万人。

我

and up to 416 million people globally

全球范围内多达4.16亿人

二

. The disease causes memory loss and may escalate to decreased independence, communication challenges, behavioral disorders such as paranoia and anxiety, and lack of physical control

这种疾病会导致记忆力丧失，并可能升级为独立性下降、沟通困难、行为障碍（如偏执和焦虑）以及缺乏身体控制。

iii

. In 2023, the total cost of caring for people living with Alzheimer’s and other dementias in the U.S. was estimated at $345 billion

2023年，美国照顾阿尔茨海默病及其他痴呆症患者的总成本估计为3450亿美元。

四

About

关于

Voyager Therapeutics

Voyager Therapeutics, Inc.

Voyager Therapeutics公司

(Nasdaq: VYGR) is a biotechnology company dedicated to leveraging the power of human genetics to modify the course of – and ultimately cure – neurological diseases. Our pipeline includes programs for Alzheimer’s disease, Friedreich’s ataxia, Parkinson’s disease, amyotrophic lateral sclerosis (ALS), and multiple other diseases of the central nervous system.

（纳斯达克：VYGR）是一家生物技术公司，致力于利用人类遗传学的力量来改变神经类疾病的发展进程，并最终治愈这些疾病。我们的研发管线包括针对阿尔茨海默病、弗里德赖希共济失调、帕金森病、肌萎缩侧索硬化症（ALS）以及中枢神经系统的多种其他疾病的项目。

Many of our programs are derived from our TRACER™ AAV capsid discovery platform, which we have used to generate novel capsids and identify associated receptors to potentially enable high brain penetration with genetic medicines following intravenous dosing. Some of our programs are wholly owned, and some are advancing with partners including Alexion, AstraZeneca Rare Disease; .

许多我们的项目源自我们的TRACER™ AAV衣壳发现平台，我们已经利用该平台生成了新型衣壳并鉴定了相关受体，有望通过静脉给药使基因药物实现高脑渗透。我们的一些项目完全自有，另一些则与包括Alexion、阿斯利康罕见病部门在内的合作伙伴共同推进。

Novartis Pharma AG

诺华制药公司

; and Neurocrine Biosciences, Inc. For more information, visit

；以及Neurocrine Biosciences, Inc. 欲了解更多信息，请访问

http://www.voyagertherapeutics.com

。

Voyager Therapeutics

is a registered trademark, and TRACER™ is a trademark, of

是注册商标，TRACER™ 是商标，属于

Voyager Therapeutics, Inc.

Voyager Therapeutics公司

Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “will,” “expect,” “believe,” “anticipate,” “potential,” “may,” or “continue,” and other similar expressions are intended to identify forward-looking statements..

本新闻稿包含根据1995年《私人证券诉讼改革法案》及其他联邦证券法规定的安全港条款所作出的前瞻性陈述。使用“将”、“预期”、“相信”、“预计”、“潜在”、“可能”或“继续”等词语以及其他类似表述旨在识别前瞻性陈述。

For example, all statements Voyager makes regarding Voyager’s ability to advance its AAV-based gene therapy programs and tau antibody program, including expectations for Voyager’s achievement of preclinical and clinical development milestones for its potential development candidates such as the IND and CTA filings, the initiation of clinical trials, clinical trial enrollment, and the generation of clinical data; Voyager’s plans to present scientific data at future conferences; the commercial potential for VY7523 and VY1706; the importance of tau as a target for the treatment of AD; and the potential for third-party clinical data to inform Voyager’s clinical development plans are forward looking..

例如，Voyager关于其推进基于AAV的基因治疗计划和tau抗体计划的能力的所有声明，包括对Voyager实现其潜在开发候选药物（如IND和CTA申报、临床试验启动、临床试验入组及临床数据生成）的临床前和临床开发里程碑的预期；Voyager计划在未来的会议上展示科学数据；VY7523和VY1706的商业潜力；tau作为AD治疗靶点的重要性；以及第三方临床数据可能为Voyager的临床开发计划提供参考的潜力，均为前瞻性陈述。

All forward-looking statements are based on estimates and assumptions by Voyager’s management that, although Voyager believes such forward-looking statements to be reasonable, are inherently uncertain and subject to risks and uncertainties that may cause actual results to differ materially from those that Voyager expected.

所有前瞻性陈述均基于Voyager管理层的估计和假设，尽管Voyager认为这些前瞻性陈述是合理的，但其本质上存在不确定性，并受可能致使实际结果与Voyager预期有重大差异的风险和不确定因素影响。

Such risks and uncertainties include, among others, the expectations and decisions of regulatory authorities; the timing, initiation, conduct and outcomes of Voyager’s preclinical and clinical studies; the availability of data from clinical trials; the availability or commercial potential of product candidates under collaborations; the success of Voyager’s product candidates; the willingness and ability of Voyager's collaboration partners to meet obligations under collaboration agreements with Voyager; the continued development of Voyager’s technology platforms, including Voyager’s TRACER platform and its antibody screening technology; Voyager’s scientific approach and program development progress, and the restricted supply of critical research components; the development by third parties of capsid identification platforms that may be competitive to Voyager’s TRACER capsid discovery platform; Voyager’s ability to create and protect intellectual property rights associated with the TRACER capsid discovery platform, the capsids identified by the platform, and development candidates for Voyager’s pipeline programs; the possibility or the timing of Voyager’s receipt of program reimbursement, development or commercialization milestones, option exercise, and other payments under Voyager’s existing licensing or collaboration agreements; the ability of Voyager to negotiate and complete licensing or collaboration agreements with other parties on terms acceptabl.

此类风险和不确定性包括：监管机构的期望与决策；Voyager公司临床前和临床研究的时间、启动、执行及结果；临床试验数据的可用性；合作项目下候选产品的可用性或商业潜力；Voyager公司候选产品的成功；Voyager合作方履行与Voyager合作协议义务的意愿和能力；Voyager技术平台的持续开发，包括Voyager的TRACER平台及其抗体筛选技术；Voyager的科学方法与项目开发进展，以及关键研究组件供应的限制；第三方可能开发出与Voyager的TRACER衣壳发现平台竞争的衣壳识别平台；Voyager保护与其TRACER衣壳发现平台、该平台识别的衣壳及Voyager管线计划中开发候选产品相关的知识产权的能力；Voyager获得项目报销、开发或商业化里程碑付款、期权行使及其他根据现有许可或合作协议支付的可能性或时间；Voyager与其他方就可接受条款谈判并完成许可或合作协议的能力。

These statements are also subject to a number of material risks and uncertainties that are described in Voyager’s most recent Annual Report on Form 10-K filed with the

这些声明也受到许多重大风险和不确定因素的影响，这些风险和不确定因素在 Voyager 最近提交的 10-K 表年度报告中有描述，

Securities and Exchange Commission

证券交易委员会

. All information in the press release is as of the date of this press release, and any forward-looking statement speaks only as of the date on which it was made. Voyager undertakes no obligation to publicly update or revise this information or any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law..

新闻稿中的所有信息均截至本新闻稿发布之日，任何前瞻性声明仅在其作出之日有效。Voyager 没有义务公开更新或修改此信息或任何前瞻性声明，无论是由于新信息、未来事件或其他原因，除非法律要求。

Contacts

联系人

Trista Morrison

特里斯塔·莫里森

, NACD.DC,

，NACD.DC，

tmorrison@vygr.com

Investors:

投资者：

Sarah McCabe

萨拉·麦凯布