拜耳子公司AskBio宣布在《自然医学》上发表AB-1002基因疗法1期临床试验的12个月数据-动脉网

AskBio Announces Publication in Nature Medicine of 12-month data from Phase 1 Trial of AB-1002 Gene Therapy in Participants with Congestive Heart Failure

拜耳等信源发布 2025-10-21 20:31

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October

十月

2025

14:30 PM

Europe/Amsterdam

欧洲/阿姆斯特丹

AskBio Announces Publication in Nature Medicine of 12-month data from Phase 1 Trial of AB-1002 Gene Therapy in Participants with Congestive Heart Failure

AskBio 宣布在《自然医学》上发表 AB-1002 基因治疗 congestive heart failure 患者一期试验 12 个月数据

Summary

摘要

First-in-human trial of investigational gene therapy AB-1002 designed to evaluate safety and preliminary efficacy in participants with New York Heart Association (NYHA) Class III heart failure

首次人体试验，评估研究性基因疗法AB-1002在纽约心脏协会（NYHA）III级心力衰竭患者中的安全性和初步疗效。

No adverse events were deemed related to AB-1002, and clinically meaningful improvement of several efficacy assessments was seen in participants with non-ischemic congestive heart failure (CHF)

与AB-1002相关的不良事件被认为不存在，且在非缺血性充血性心力衰竭（CHF）患者中观察到多项疗效评估指标有临床意义上的改善。

Phase 2 GenePHIT trial is currently enrolling in Canada, Europe, the United Kingdom, and the United States

第二阶段的GenePHIT试验目前正在加拿大、欧洲、英国和美国招募参与者。

Research Triangle Park, N.C.– October 21, 2025

北卡罗来纳州研究三角园区——2025年10月21日

–

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the publication in peer-reviewed journal

AskBio Inc.（AskBio）是一家基因治疗公司，为拜耳集团全资拥有并独立运营的子公司，今天宣布在同行评审期刊上发表文章。

Nature Medicine

自然医学

of 12-month data from its Phase 1 trial of AB-1002 investigational gene therapy in participants with congestive heart failure (CHF)

为期12个月的AB-1002基因治疗一期试验数据，针对充血性心力衰竭（CHF）参与者

。

This non-randomized, sequential dose-escalation trial (

这项非随机、顺序剂量递增试验（

NCT04179643

) includes escalating dose cohorts to evaluate the safety and preliminary efficacy of investigational gene therapy AB-1002 in participants with New York Heart Association (NYHA) Class III non-ischemic heart failure with reduced ejection fraction (HFrEF).

包括逐步增加剂量组，以评估研究性基因疗法AB-1002在纽约心脏协会（NYHA）III级非缺血性心力衰竭伴射血分数降低（HFrEF）患者中的安全性和初步疗效。

It is estimated that 64 million people worldwide are living with heart failure, and despite standard of care, mortality and morbidity remain very high.

据估计，全球有 6400 万人患有心力衰竭，尽管有标准的治疗方法，但死亡率和发病率仍然非常高。

2,3

The publication, which is available

该出版物现已可用。

online

在线

, confirms that no adverse events were deemed related to AB-1002 in this trial and that clinically meaningful improvements were recorded across several efficacy assessments in participants with non-ischemic CHF.

，证实在此试验中没有不良事件被认为与AB-1002相关，并且在非缺血性充血性心力衰竭（CHF）的参与者中，多个疗效评估显示有临床意义的改善。

The data further support that the AB-1002 capsid may be highly cardiotrophic when administered as a single intracoronary injection.

数据进一步支持，当作为单次冠状动脉内注射给药时，AB-1002衣壳可能具有高度的心脏嗜性。

AskBio thanks the participants who volunteered for this important clinical trial, the sites that made this effort possible, and the skilled investigators who conducted this invaluable research and contributed to the scientific body of knowledge related to AB-1002.

AskBio感谢参与这项重要临床试验的志愿者、使这项努力成为可能的机构，以及进行这项宝贵研究并为与AB-1002相关的科学知识体系做出贡献的熟练研究人员。

'We believe there is a critical need to progress innovative therapies that target the root causes of congestive heart failure, so we’re pleased to see these data for AB-1002 published and shared with the scientific community via

“我们认为，迫切需要推进针对充血性心力衰竭根本原因的创新疗法，因此我们很高兴看到AB-1002的数据发表，并通过以下方式与科学界分享：

Nature Medicine

自然医学

, a high-impact peer-reviewed journal,' said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer at AskBio. 'We’re eager to further assess the safety and efficacy of AB-1002 in our ongoing Phase 2 trial, GenePHIT, which

“，这是一份高影响力的同行评审期刊，” AskBio的首席开发官兼首席医学官江灿文博士说道。“我们渴望在正在进行的第二阶段试验GenePHIT中进一步评估AB-1002的安全性和有效性，这项试验

is currently enrolling in Canada, Europe, the United Kingdom, and the United States, and look forward to sharing those results once available

目前在加拿大、欧洲、英国和美国招募患者，并期待在结果出来后分享这些结果。

。'

GenePHIT is a Phase 2, adaptive, randomized, double-blind, placebo-controlled trial investigating the safety and efficacy of AB-1002 in non-ischemic heart failure.

GenePHIT 是一项二期、适应性、随机、双盲、安慰剂对照试验，研究 AB-1002 在非缺血性心力衰竭中的安全性和有效性。

About AB-1002

关于 AB-1002

AB-1002 is an investigational one-time gene therapy administered to the heart to promote production of a modified version of the therapeutic inhibitor 1 (I-1c) protein designed to block the action of protein phosphatase 1, which is linked to CHF.

AB-1002是一种研究性一次性基因疗法，施用于心脏，以促进治疗性抑制剂1（I-1c）蛋白的修饰版本的生成，该蛋白旨在阻断与充血性心力衰竭（CHF）相关的蛋白磷酸酶1的作用。

4,5

This investigational gene therapy has not been approved by any regulatory authority, and its efficacy and safety have not yet been established or fully evaluated.

这种研究性基因疗法尚未获得任何监管机构的批准，其疗效和安全性尚未确定或完全评估。

About Congestive Heart Failure

关于充血性心力衰竭

Heart failure occurs when the heart cannot pump blood efficiently enough to meet the body’s needs, including providing sufficient oxygen to the organs.

心力衰竭是指心脏无法有效泵血以满足身体需求，包括为器官提供足够的氧气。

Congestive heart failure results in the slowing of the blood flow out of the heart, which causes the blood returning to the heart through the veins to back up.

充血性心力衰竭导致心脏流出的血液流速减慢，从而使通过静脉回流到心脏的血液受阻。

This causes congestion in the body’s tissues.

这会导致身体组织充血。

Symptoms may include shortness of breath, swelling in the legs and ankles caused by fluid retention, and fatigue.

症状可能包括呼吸急促、因体液潴留导致的腿部和脚踝肿胀以及疲劳。

More than 64 million people worldwide are estimated to be living with heart failure.

据估计，全球有超过 6400 万人患有心力衰竭。

About GenePHIT

关于GenePHIT

GenePHIT is a Phase 2 adaptive, double-blinded, placebo-controlled, randomized, multi-center trial conducted to evaluate the safety and efficacy of the one-time administration of investigational gene therapy AB-1002, via antegrade intracoronary artery infusion, in males and females age >18 years with non-ischemic cardiomyopathy and New York Heart Association (NYHA) Class III heart failure symptoms..

GenePHIT 是一项二期适应性、双盲、安慰剂对照、随机、多中心试验，旨在评估通过顺行冠状动脉内输注一次性给予研究性基因疗法 AB-1002 的安全性和有效性，适用于年龄大于 18 岁、患有非缺血性心肌病和纽约心脏协会 (NYHA) III 级心力衰竭症状的男性和女性。

For more information, please visit euclinicaltrials.eu (EUCT#2024-510581-17-00), clinicaltrials.gov (

有关更多信息，请访问 euclinicaltrials.eu （EUCT#2024-510581-17-00）、clinicaltrials.gov （

NCT#05598333

), or

），或者

askbio.com

。

GenePHIT is being conducted at 46 locations across the United States, Austria, Germany, the Netherlands, Spain, and the United Kingdom.

GenePHIT 正在美国、奥地利、德国、荷兰、西班牙和英国的 46 个地点进行。

About AskBio

关于AskBio

AskBio Inc., a wholly owned and independently operated subsidiary of Bayer AG, is a fully integrated gene therapy company dedicated to steering gene therapy into a new era where it can transform the lives of a wider range of people living with rare and more common diseases. The company maintains a portfolio of clinical programs across a range of disease indications related to a single gene or multiple factors across cardiovascular, central nervous system, and neuromuscular conditions, with a clinical-stage pipeline that includes investigational therapeutics for congestive heart failure, limb-girdle muscular dystrophy, multiple system atrophy, Parkinson’s disease, and Pompe disease.

AskBio公司是拜耳集团全资拥有并独立运营的子公司，是一家完全集成的基因治疗公司，致力于将基因治疗引入一个新时代，使其能够改变更广泛患有罕见和常见疾病人群的生活。该公司在心血管、中枢神经系统和神经肌肉疾病领域，针对单基因或多因素相关的多种疾病适应症，拥有多项临床计划组合，其临床阶段的研发管线包括用于治疗充血性心力衰竭、肢带型肌营养不良、多系统萎缩、帕金森病和庞贝病的研究性疗法。

AskBio’s end-to-end gene therapy platform includes our Pro10™ technology, which makes gene therapies more accessible by making research and commercial grade manufacturing more affordable. With global headquarters in Research Triangle Park, North Carolina, the company has generated hundreds of proprietary capsids and promoters, several of which have entered pre-clinical and clinical testing.

AskBio的端到端基因治疗平台包括我们的Pro10™技术，该技术通过降低研究和商业化生产成本，使基因治疗更加可及。公司总部位于北卡罗来纳州三角研究园，在全球范围内已经开发了数百种专有的衣壳和启动子，其中一些已进入临床前和临床测试阶段。

An early innovator in the gene therapy field with over 900 employees in five countries, the company holds more than 600 patents and patent applications in areas such as AAV production and chimeric capsids. Learn more at .

一家基因治疗领域的早期创新者，在五个国家拥有900多名员工，该公司在AAV生产和嵌合衣壳等领域持有600多项专利和专利申请。欲了解更多信息，请访问。

http://www.askbio.com/

or follow us on

或关注我们

领英

。

About Bayer

关于拜耳

Bayer is a global enterprise with core competencies in the life science fields of health care and nutrition. In line with its mission, “Health for all, Hunger for none,” the company’s products and services are designed to help people and the planet thrive by supporting efforts to master the major challenges presented by a growing and aging global population.

拜耳是一家在医疗保健和营养等生命科学领域具有核心竞争力的全球性企业。秉承“人人健康，无饥饿”的使命，公司通过支持应对不断增长和老龄化的全球人口所带来的重大挑战，设计其产品和服务以帮助人类和地球繁荣发展。

Bayer is committed to driving sustainable development and generating a positive impact with its businesses. At the same time, the Group aims to increase its earning power and create value through innovation and growth. The Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2024, the Group employed around 93,000 people and had sales of 46.6 billion euros.

拜耳致力于通过其业务推动可持续发展并产生积极影响。同时，集团旨在通过创新和增长提高盈利能力并创造价值。拜耳品牌在全球范围内代表信任、可靠性和质量。在2024财年，该集团拥有约93,000名员工，销售额达466亿欧元。

R&D expenses amounted to 6.2 billion euros. For more information, go to .

研发费用达62亿欧元。欲了解更多信息，请访问。

http://www.bayer.com

。

AskBio Forward-Looking Statements

AskBio前瞻性声明

This press release contains “forward-looking statements.” Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible,” and similar expressions are intended to identify forward-looking statements.

本新闻稿包含“前瞻性陈述”。本新闻稿中包含的任何非历史事实的陈述可能被视为前瞻性陈述。诸如“相信”、“预期”、“计划”、“预计”、“将”、“意图”、“潜力”、“可能”等词语及类似表达旨在识别前瞻性陈述。

These forward-looking statements include, without limitation, statements regarding AskBio’s clinical trials. These forward-looking statements involve risks and uncertainties, many of which are beyond AskBio’s control. Known risks include, among others: AskBio may not be able to execute on its business plans and goals, including meeting its expected or planned clinical and regulatory milestones and timelines, its reliance on third-parties, clinical development plans, manufacturing processes and plans, and bringing its product candidates to market, due to a variety of reasons, including possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved in a timely manner, potential disagreements or other issues with our third-party collaborators and partners, and regulatory, court or agency feedback or decisions, such as feedback and decisions from the United States Food and Drug Administration or the United States Patent and Trademark Office.

这些前瞻性声明包括但不限于有关AskBio临床试验的声明。这些前瞻性声明涉及许多风险和不确定性，其中许多是AskBio无法控制的。已知的风险包括但不限于：由于各种原因，AskBio可能无法执行其商业计划和目标，包括无法达成其预期或计划的临床和监管里程碑与时间表、对第三方的依赖、临床开发计划、制造流程与计划，以及将其产品候选者推向市场的能力。这些原因包括公司财务和其他资源的可能限制、可能未能及时预见或解决的制造限制、与第三方合作者和合作伙伴之间潜在的分歧或其他问题，以及来自监管机构、法院或相关机构的反馈或决定，例如来自美国食品药品监督管理局或美国专利商标局的反馈和决定。

Any of the foregoing risks could materially and adversely affect AskBio’s business and results of operations. You should not place undue reliance on the forward-looking statements contained in this press release. AskBio does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof. .

上述任何风险都可能对AskBio的业务和经营结果产生重大不利影响。您不应过度依赖本新闻稿中包含的前瞻性陈述。AskBio不承担基于此后发生的事件或情况公开更新其前瞻性陈述的义务。

References

参考文献

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2. Savarese G, et al. Global burden of heart failure: a comprehensive and updated review of epidemiology. Cardiovasc Res. 2023 Jan 18;118(17):3272-3287.

Savarese G, 等。全球心力衰竭负担：流行病学的全面更新综述。《心血管研究》。2023年1月18日；118(17):3272-3287。

3. Myhre P, et al. Digital tools in heart failure: addressing unmet needs. Lancet Digit Health. 2024 Oct;6(10):e755-e766.

3. Myhre P, 等。心力衰竭中的数字工具：满足未满足的需求。《柳叶刀数字健康》。2024年10月；6(10):e755-e766。

4. Henry T, et al. Preliminary safety and efficacy of a Phase 1 clinical gene therapy trial in patients with advanced heart failure using a rationally designed cardiotropic AAV vector targeting Protein Phosphatase Inhibitor-1. Presented at American Heart Association Scientific Sessions, November 2023..

亨利·T等人。使用合理设计的靶向蛋白磷酸酶抑制剂-1的心脏趋向性AAV载体治疗晚期心力衰竭患者的1期临床基因治疗试验的初步安全性和有效性。于2023年11月在美国心脏协会科学会议上发表。

5. Nicolaou P & Kranias E. Role of PP1 in the regulation of Ca cycling in cardiac physiology and pathophysiology. Front Biosci (Landmark Ed). 2009 Jan 1;14(9):3571-85.

5. 尼古拉乌 P 和克拉尼亚斯 E。PP1 在心脏生理和病理生理中钙循环调节中的作用。《前沿生物科学》（里程碑版）。2009年1月1日；14(9):3571-85。

6. Centers for Disease Control and Prevention. Heart failure. Published 2022. Available at:

6. 疾病控制与预防中心。心力衰竭。发布于2022年。可访问：

https://www.cdc.gov/heart-disease/about/heart-failure.html

. Accessed September 2025.

. 2025年9月访问。

7. American Heart Association. Types of Heart Failure. Available at:

7. 美国心脏协会。心力衰竭的类型。可访问：

https://www.heart.org/en/health-topics/heart-failure/what-is-heart-failure/types-of-heart-failure

https://www.heart.org/zh/health-topics/heart-failure/what-is-heart-failure/types-of-heart-failure

. Accessed September 2025.

。访问时间：2025年9月。