AskBio的AB-1005和AB-1002在日本获得先锋再生医学产品指定-动脉网

AskBio’s AB-1005 and AB-1002 Receive Pioneering Regenerative Medical Product Designation in Japan

拜耳等信源发布 2025-12-09 15:33



可切换为仅中文







December

十二月

2025

08:29 AM

早上08:29

Europe/Amsterdam

欧洲/阿姆斯特丹

AskBio’s AB-1005 and AB-1002 Receive Pioneering Regenerative Medical Product Designation in Japan

AskBio的AB-1005和AB-1002在日本获得开创性再生医疗产品指定。

Not intended for UK

不适用于英国

Summary

摘要

Product designation supports development of innovative investigational gene therapies for Parkinson’s disease and congestive heart failure

产品指定支持开发用于帕金森病和充血性心力衰竭的创新研究基因疗法

Berlin, Germany, and Durham, N.C., USA, December 9, 2025

德国柏林，美国北卡罗来纳州达勒姆，2025年12月9日

–

AskBio Inc., a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted the Pioneering Regenerative Medical Product designation (SAKIGAKE) for two of AskBio’s investigational gene therapy programs: AB-1005, for the treatment of Parkinson’s disease (PD), and AB-1002, for the treatment of non-ischemic heart failure with reduced left ventricular ejection fraction and New York Heart Association (NYHA) Class III heart failure despite appropriate medical therapy..

AskBio公司是拜耳集团全资拥有并独立运营的基因治疗子公司，该公司今日宣布，日本厚生劳动省（MHLW）已授予其两项在研基因治疗项目“先锋再生医疗产品”称号（SAKIGAKE），这两个项目分别是：用于治疗帕金森病（PD）的AB-1005，以及用于治疗尽管接受适当药物治疗但仍存在左心室射血分数降低和纽约心脏协会（NYHA）III级心衰的非缺血性心力衰竭的AB-1002。

The designation reflects Japan’s commitment to expediting the development and review of breakthrough therapies and is awarded to products demonstrating innovativeness (a new mode of action), prominent efficacy or safety data, and the potential to address severe diseases, especially when submitted first or simultaneously with other countries.

该认定反映了日本致力于加快突破性疗法的开发和审查，授予那些展示出创新性（新的作用模式）、显著的有效性或安全性数据，并有潜力解决严重疾病的产品，特别是当这些产品首次提交或与其他国家同时提交时。

This recognition offers significant advantages, including priority consultations and accelerated review timelines, thereby facilitating earlier participant access to transformative treatments..

这一认定带来了显著的优势，包括优先咨询和加快审查时间，从而促进参与者更早获得变革性的治疗。

“Having AB-1005 and AB-1002 receive the Pioneering Regenerative Medical Product designation in Japan highlights our dedication to advancing innovative gene therapies for participants facing serious diseases,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “This recognition not only accelerates regulatory review but also reaffirms our commitment to delivering advanced treatments to those living with serious chronic diseases that lack therapies targeting root causes.”.

“AB-1005和AB-1002在日本获得开创性再生医学产品称号，彰显了我们致力于为面临严重疾病的患者推进创新基因疗法的决心，”AskBio首席开发官兼首席医学官Canwen Jiang博士表示，“这一认可不仅加快了监管审查，还重申了我们为那些身患缺乏针对根本原因的治疗方法的严重慢性病患者提供先进治疗方案的承诺。”

AB-1005, currently being evaluated in the Phase II REGENERATE-PD trial, is an investigational gene therapy with adeno-associated viral (AAV) vector-mediated delivery of the glial cell line-derived neurotrophic factor (GDNF) gene for participants with moderate-stage PD. The therapy aims to restore neuronal function and potentially slow disease progression for people with limited treatment options.

AB-1005 是目前正在 II 期 REGENERATE-PD 试验中评估的一种研究性基因疗法，通过腺相关病毒（AAV）载体递送胶质细胞源性神经营养因子（GDNF）基因，用于中度帕金森病（PD）患者。该疗法旨在恢复神经元功能，并可能减缓那些治疗选择有限的患者的疾病进展。

AB-1005 previously received US Food and Drug Administration (FDA) Regenerative Medicine Advanced Therapy (RMAT), FDA Fast Track, and UK Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Passport designations, underscoring its global significance and potential for participants..

AB-1005 先前获得了美国食品药品监督管理局（FDA）的再生医学先进疗法（RMAT）、FDA快速通道以及英国药品和健康产品管理局（MHRA）的创新护照资格，凸显了其全球重要性及对参与者的潜力。

AB-1002 is an investigational AAV gene therapy being studied for the treatment of adults with NYHA Class III heart failure with non-ischemic etiology. It previously received FDA Fast Track designation, and is designed as a one-time gene therapy targeting protein phosphatase 1 inhibition, with the intention of improving cardiac function and addressing the substantial global burden of congestive heart failure..

AB-1002 是一种正在研究中的 AAV 基因疗法，用于治疗纽约心脏病协会 (NYHA) III 级心力衰竭且病因为非缺血性的成年患者。该疗法之前已获得美国食品药品监督管理局 (FDA) 的快速通道资格，旨在通过一次性基因治疗抑制蛋白磷酸酶 1，以期改善心脏功能，并应对全球充血性心力衰竭的重大负担。

“Bayer and AskBio’s collaboration continues to drive progress in gene therapy with a robust pipeline targeting central nervous system, cardiovascular, and other disease indications,” said Christian Rommel, PhD and Global Head of Research and Development for Bayer's Pharmaceuticals Division. “Receiving the designation in Japan, which is a first for Bayer, marks an important milestone in expanding global access to pioneering therapies and reinforces our shared commitment to delivering breakthrough science to improve outcomes for patients worldwide.”.

“拜耳与AskBio的合作继续推动基因治疗的进展，其强大的研发管线针对中枢神经系统、心血管系统及其他疾病领域，”拜耳制药部门全球研发负责人克里斯蒂安·罗梅尔博士表示。“此次在日本获得认定，是拜耳的首次，标志着扩大全球获取前沿疗法的重要里程碑，同时也进一步彰显了我们共同致力于通过突破性科学改善全球患者预后的承诺。”

FDA RMAT is a designation granted by the FDA to regenerative therapies, including gene therapies, being developed to treat, modify, reverse, or cure serious or life-threatening diseases or conditions.

FDA RMAT是FDA授予再生疗法（包括基因疗法）的指定，这些疗法旨在治疗、修饰、逆转或治愈严重或危及生命的疾病或状况。

Investigational products receiving this designation must have produced preliminary clinical evidence indicating that they may have the potential to address unmet medical needs for such diseases or conditions.

获得这一指定的在研产品必须已产生初步临床证据，表明它们可能有潜力满足此类疾病或状况的未满足医疗需求。

RMAT provides recipients with enhanced access to the FDA, which could include intensive guidance on efficient drug development, rolling Biologics License Application (BLA) review, and other actions to expedite review.

RMAT为受助者提供了更便捷的FDA通道，其中可能包括针对高效药物开发的深入指导、滚动生物制品许可申请（BLA）审查以及其他加快审查的措施。

The FDA Fast Track program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and address unmet medical needs.

FDA快速通道计划旨在促进用于治疗严重疾病和满足未满足医疗需求的新疗法的开发，并加快其审查进程。

The purpose of the program is to get important new therapeutics to participants earlier.

该计划的目的是让参与者更早获得重要的新疗法。

Therapeutics that receive this designation benefit from eligibility for more frequent meetings with the FDA to discuss the clinical development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.

获得此指定的治疗药物可享有与FDA更频繁会面讨论临床开发计划的机会，并且在符合相关标准的情况下，还可享有加速批准和优先审查的资格。

The UK MHRA Innovation Passport is the entry point to the Innovative Licensing and Access Pathway (ILAP), which aims to accelerate time to market, facilitating participants access to innovative medicines. This designation provides Innovation Passport holders with the opportunity to work with the UK MHRA and partners to create product-specific Target Development Profiles (TDP) for new therapies.

英国药品和健康产品管理局（MHRA）的创新护照是进入创新许可和获取途径（ILAP）的入口，该途径旨在加快产品上市时间，帮助参与者获得创新药物。此指定为创新护照持有者提供了与英国MHRA及其合作伙伴合作的机会，为新疗法创建特定产品的目标开发概况（TDP）。

The TDP will define key regulatory and development features, identify potential pitfalls, offer access to specialist toolkits, and create a roadmap for delivering early participants access..

TDP 将定义关键的监管和开发特性，识别潜在的隐患，提供对专业工具包的访问权限，并为向早期参与者提供访问创建路线图。

4,5

AB-1005 and AB-1002 are investigational gene therapies that have not been approved by any regulatory health authority, and their efficacy and safety have not been established or fully evaluated.

AB-1005 和 AB-1002 是尚未获得任何监管卫生机构批准的在研基因疗法，其有效性和安全性尚未确立或完全评估。

AskBio is also exploring AB-1005 beyond PD and is currently conducting the MSA-101 trial in the U.S. for participants diagnosed with multiple system atrophy of the parkinsonian subtype (MSA-P) in a Phase I trial to assess the preliminary safety, tolerability, and efficacy of GDNF gene therapy for this rapidly progressing condition for which no treatment options are currently available..

AskBio还在探索AB-1005在帕金森病以外的应用，目前正在美国进行MSA-101试验，针对被诊断为帕金森亚型多系统萎缩（MSA-P）的参与者开展一期试验，以评估GDNF基因疗法对于这一目前尚无治疗选择的快速进展疾病的初步安全性、耐受性和有效性。

About Parkinson’s disease

关于帕金森病

Parkinson’s disease (PD) is a progressive neurodegenerative disease.

帕金森病（PD）是一种进展性神经退行性疾病。

It has a significant impact on a person’s daily life.

它对一个人的日常生活有重大影响。

In PD, the death of dopamine producing nerve cells in the brain leads to the continuous loss of motor function.

在帕金森病中，大脑中产生多巴胺的神经细胞死亡导致运动功能的持续丧失。

Symptoms include tremors, muscle rigidity, and slowness of movement.

症状包括震颤、肌肉僵硬和运动迟缓。

Additionally, people with PD experience non-motor symptoms, including fatigue and lack of energy, cognitive issues, and depression.

此外，帕金森病患者还会经历非运动症状，包括疲劳、缺乏精力、认知问题和抑郁。

Symptoms typically intensify over time and make everyday tasks increasingly demanding.

症状通常会随着时间的推移而加重，使日常任务变得越来越艰巨。

The prevalence of PD has doubled over the past 25 years.

过去25年中，PD的患病率翻了一番。

Today, more than 10 million people worldwide are estimated to be living with PD.

今天，据估计全世界有超过一千万人患有帕金森病。

This makes it the world’s second most prevalent neurodegenerative disease.

这使得它成为世界上第二常见的神经退行性疾病。

It is also the most frequent movement disorder.

它也是最常见的运动障碍。

7,12

7，12

At present there is no cure, and current treatment options lack the holistic management of symptoms, so there is an urgent need for new therapies.

目前尚无治愈方法，且当前的治疗方案缺乏对症状的整体管理，因此迫切需要新的疗法。

About AB-1005

关于 AB-1005

AB-1005 is an investigational gene therapy based on an adeno-associated viral vector containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, which allows for stable and continuous expression of GDNF in localized regions of the brain after direct neurosurgical injection with convection-enhanced delivery..

AB-1005 是一种基于腺相关病毒载体的实验性基因疗法，该载体包含人类胶质细胞系衍生的神经营养因子（GDNF）转基因，可通过直接神经外科手术注射结合对流增强递送技术，在大脑局部区域实现GDNF的稳定和持续表达。

14,15

14，15

In nonclinical studies, GDNF has been shown to promote the survival and morphological differentiation of dopaminergic neurons, which could aid in the preservation and restoration of dopaminergic neuronal circuitries normally lost in the disease.

在非临床研究中，GDNF 已被证明能够促进多巴胺能神经元的存活和形态分化，这有助于保护和恢复通常在疾病中丧失的多巴胺能神经元回路。

15,16

15，16

Recombinant GDNF has long been evaluated as a potential treatment for diseases, such as Parkinson’s disease (PD), marked by progressive degeneration of midbrain dopaminergic neurons.

重组GDNF长期以来一直被评估为治疗诸如帕金森病（PD）等疾病的潜在方法，这些疾病以中脑多巴胺能神经元的进行性退化为特征。

Through a combination of an investigational gene therapy and innovative neurosurgical delivery approach, the GDNF hypothesis in PD can now be tested by getting this neurotrophic factor to these degenerating nigrostriatal neurons in a potentially more clinically relevant fashion.

通过结合一种实验性的基因疗法和创新的神经外科递送方法，帕金森病中的GDNF假设现在可以通过将这种神经营养因子以可能更具临床相关性的方式递送到这些正在退化的黑质纹状体神经元来进行测试。

About the AB-1005 Phase Ib trial

关于AB-1005 Phase Ib试验

In the AB-1005 Phase Ib, multicenter, open-label, uncontrolled trial, participants were administered AB-1005 to the putamen via one-time bilateral convection-enhanced delivery.

在AB-1005 Phase Ib期多中心、开放标签、无对照的试验中，参与者通过一次性双侧对流增强递送方式将AB-1005施用于壳核。

Participants were enrolled into two cohorts, mild (6 participants) and moderate (5 participants), based upon the duration and stage of their Parkinson’s disease (PD).

参与者根据帕金森病 (PD) 的持续时间和阶段被分为两个队列，轻度（6 名参与者）和中度（5 名参与者）。

The objective of this investigation was to evaluate the safety and potential clinical effect of AB-1005 delivered to the putamen in participants with either mild- or moderate-stage PD. The outcomes assessed at 36 months were incidence of treatment-emergent adverse events (TEAEs) as reported by the participants or assessed clinically by physical and neurological examinations, motor symptoms as reported via the Movement Disorder Society’s Unified Parkinson’s Disease Rating Scale (MDS-UPDRS), and PD Motor Diary self-assessments, non-motor symptoms of PD, and brain dopaminergic network integrity as measured by DaTSCAN..

本研究的目的是评估将AB-1005递送至丘脑底核在轻度或中度帕金森病（PD）参与者中的安全性和潜在临床效果。在36个月时评估的结果包括：参与者报告或通过体格和神经学检查评估的治疗相关不良事件（TEAEs）发生率、通过运动障碍协会统一帕金森病评定量表（MDS-UPDRS）报告的运动症状和PD运动日记自我评估、PD的非运动症状，以及通过DaTSCAN测量的脑多巴胺能网络完整性。

These assessments will continue for up to five years. For more information, visit clinicaltrials.gov (

这些评估将持续长达五年。欲了解更多信息，请访问 clinicaltrials.gov (

NCT04167540

) or

) 或

askbio.com

。

About REGENERATE-PD

关于REGENERATE-PD

REGENERATE-PD is a Phase II, randomized, double-blind, surgery controlled trial of the efficacy and safety of intraputaminal AB-1005 in the treatment of adults (45–75 years) with moderate-stage Parkinson’s disease.

REGENERATE-PD 是一项二期、随机、双盲、手术对照试验，评估了脑内注射 AB-1005 治疗中度帕金森病成人（45-75 岁）的疗效和安全性。

The trial will include an estimated 87 participants with trial sites located in Germany, Poland, the United Kingdom, and the United States.

试验将包括约87名参与者，试验地点位于德国、波兰、英国和美国。

For more information about the REGENERATE-PD clinical trial, visit clinicaltrials.gov (

要了解有关REGENERATE-PD临床试验的更多信息，请访问clinicaltrials.gov（

NCT06285643

), or visit

），或访问

askbio.com

问生物网

。

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 AB-1002

关于 AB-1002

AB-1002 is an investigational one-time gene therapy based on an adeno-associated viral vector 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 是一种基于腺相关病毒载体的一次性基因疗法，通过向心脏给药，促进生成一种经过修饰的治疗性抑制蛋白 1（I-1c），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。

24,25

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立或完全评估。

About the AB-1002 Phase I trial

关于AB-1002一期试验

This non-randomized, sequential dose-escalation trial (NCT04179643) includes escalating dose cohorts to evaluate the safety and preliminary efficacy of investigational gene therapy AB-1002 in 11 participants with New York Heart Association (NYHA) Class III non-ischemic heart failure with reduced ejection fraction (HFrEF)..

这项非随机、剂量递增的试验（NCT04179643）包括递增剂量组，以评估在11名纽约心脏协会（NYHA）III级非缺血性心力衰竭伴射血分数降低（HFrEF）患者中，研究性基因疗法AB-1002的安全性和初步疗效。

No adverse events were deemed related to AB-1002 in this trial, and clinically meaningful improvements were recorded across several efficacy assessments.

在此试验中，没有不良事件被认为与AB-1002有关，并且在多个疗效评估中记录到了有临床意义的改善。

The data further support that AB-1002 may be highly cardiotropic when administered as a single intracoronary injection. Full results were published in October 2025 in the prestigious peer-reviewed journal

数据进一步支持，当AB-1002作为单一冠状动脉注射给药时，可能具有高度的心脏趋向性。完整结果于2025年10月发表在备受推崇的同行评审期刊上。

Nature Medicine

自然医学

and are available via open access.

并通过开放获取方式提供。

About GenePHIT

关于GenePHIT

GenePHIT is a Phase II 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，旨在评估通过前向冠状动脉输注一次性给予研究性基因疗法 AB-1002 在年龄大于 18 岁、患有非缺血性心肌病和纽约心脏病协会 (NYHA) III 级心力衰竭症状的男性和女性中的安全性和有效性。

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

问生物网

。

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 Inc. 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的人们的生活。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适应症，拥有多项临床项目组合，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在研疗法。

AskBio’s end-to-end gene therapy platform includes our Pro10™ technology and Aava™ manufacturing platform, which make 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™技术和Aava™制造平台，通过使研究和商业级制造更加经济实惠，这些技术使基因治疗更易于获取。公司总部位于北卡罗来纳州研究三角园区，在全球范围内已开发出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拥有600多项专利和专利申请。欲了解更多信息，请访问。

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.

拜耳致力于推动可持续发展，并通过其业务产生积极影响。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 .

研发费用总计达62亿欧元。欲了解更多信息，请访问。

www.bayer.com

。

Forward-Looking Statements

前瞻性声明

This release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here.

本发布可能包含基于拜耳管理层当前假设和预测的前瞻性声明。各种已知和未知的风险、不确定性和其他因素可能导致公司实际未来结果、财务状况、发展或业绩与这里给出的估计存在重大差异。

These factors include those discussed in Bayer’s public reports which are available on the Bayer website at .

这些因素包括拜耳公开报告中讨论的因素，该报告可在拜耳网站上查阅。

www.bayer.com

. The company assumes no liability whatsoever to update these forward-looking statements or to conform

公司不承担更新这些前瞻性声明或使其符合的任何责任。

them to future events or developments.

将它们应用于未来的事件或发展。

Forward-Looking Statements AskBio

前瞻性声明 AskBio

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关于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，包括未能达到预期或计划的临床和监管里程碑及时间表，其对第三方的依赖，临床开发计划、制造流程和计划，以及将其产品候选药物推向市场的能力。这些原因可能包括公司财务及其他资源的可能限制、可能未被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、法院或其它机构的反馈或决定，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。您不应过分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

1. Askbio.com. AskBio receives FDA Fast Track and MHRA Innovation Passport designations for AB-1005 investigational GDNF gene therapy for Parkinson’s disease. Available at:

1. Askbio.com. AskBio 的 AB-1005 调查性 GDNF 基因疗法针对帕金森病获得 FDA 快速通道和 MHRA 创新护照资格。可用链接：

https://www.askbio.com/askbio-receives-fda-fast-track-and-mhra-innovation-passport-designations-for-ab-1005-investigational-gdnf-gene-therapy-for-parkinsons-disease

https://www.askbio.com/askbio获得FDA快速通道和MHRA创新护照指定-ab-1005调查性gdnf基因治疗帕金森病

. Accessed: November 2025.

访问日期：2025年11月。

2. Clinicaltrials.gov. AB-1002 in Patients With Class III Heart Failure (NAN-CS101). Available at:

2. Clinicaltrials.gov. AB-1002在III级心力衰竭患者中的应用 (NAN-CS101)。可访问：

https://clinicaltrials.gov/study/NCT04179643

. Accessed: November 2025.

访问日期：2025年11月。

3. Food and Drug Administration. Expedited Programs for Regenerative Medicine Therapies for Serious Conditions. Available at:

3. 食品药品监督管理局。严重疾病再生医学疗法的快速审批程序。可见于：

https://www.fda.gov/media/120267/download

. Accessed: November 2025.

访问日期：2025年11月。

4. UK Government. Innovative Licensing and Access Pathway. Available at:

4. 英国政府。创新许可和准入途径。可于以下网址获取：

https://www.gov.uk/guidance/innovative-licensing-and-access-pathway

. Accessed: November 2025.

访问日期：2025年11月。

5. UK Government. First Innovation Passport awarded to help support development and access to cutting-edge medicines. Available at:

5. 英国政府。首个创新护照颁发，以帮助支持前沿药物的开发和获取。可在：

https://www.gov.uk/government/news/first-innovation-passport-awarded-to-help-support-development-and-access-to-cutting-edge-medicines

. Accessed: November 2025.

访问日期：2025年11月。

6. ClinicalTrials.gov. GDNF Gene Therapy for Multiple System Atrophy. Available at:

6. ClinicalTrials.gov。GDNF基因疗法治疗多系统萎缩症。可用地址：

https://clinicaltrials.gov/study/NCT04680065

. Accessed: November 2025.

访问日期：2025年11月。

7. World Health Organization. Parkinson Disease. Available at:

7. 世界卫生组织。帕金森病。可访问于：

https://www.who.int/news-room/fact-sheets/detail/parkinson-disease

. Accessed November 2025.

访问时间：2025年11月。

8. Ramesh S & Arachchige A. Depletion of dopamine in Parkinson’s disease and relevant therapeutic options: A review of the literature. AIMS Neurosci. 2023 Aug 14;10(3):200-231.

8. Ramesh S & Arachchige A. 帕金森病中多巴胺的耗竭及相关治疗选择：文献综述。《AIMS神经科学》2023年8月14日；第10卷第3期：200-231页。

9. National Institutes of Health. Parkinson’s Disease. Available at:

9. 国立卫生研究院。帕金森病。可见于：

https://www.ninds.nih.gov/health-information/disorders/parkinsons-disease

. Accessed: November 2025.

访问日期：2025年11月。

10. Maserejian N, et al. Estimation of the 2020 Global Population of Parkinson’s Disease (PD). MDS Virtual Congress 2020. Abstract number 198. Available at:

10. Maserejian N 等。2020年全球帕金森病（PD）人口估计。MDS虚拟大会2020。摘要编号198。可见于：

https://www.mdsabstracts.org/abstract/estimation-of-the-2020-global-population-of-parkinsons-disease-pd/

. Accessed: November 2025.

访问日期：2025年11月。

11. National Institute of Environmental Health Sciences. Neurodegenerative Diseases. Available at:

11. 国家环境健康科学研究所。神经退行性疾病。可访问：

https://www.niehs.nih.gov/research/supported/health/neurodegenerative

. Accessed: November 2025.

访问日期：2025年11月。

12. Stoker T & Barker R. Recent developments in the treatment of Parkinson’s Disease. F1000Res. 2020 Jul 31;9:F1000 Faculty Rev-862.

12. Stoker T 和 Barker R. 帕金森病治疗的最新进展。F1000Res. 2020年7月31日；9:F1000 教授评审-862。

13. Mayo Clinic. Parkinson’s disease. Available at:

13. 梅奥诊所。帕金森病。可访问于：

https://www.mayoclinic.org/diseases-conditions/parkinsons-disease/diagnosis-treatment/drc-20376062

. Accessed: November 2025.

访问日期：2025年11月。

14. Heiss J, et al. Trial of magnetic resonance-guided putaminal gene therapy for advanced Parkinson’s disease. Mov Disord. 2019 Jul;34(7):1073-1078.

14. Heiss J 等。磁共振引导下丘脑基因治疗晚期帕金森病的试验。《运动障碍》2019年7月；34(7):1073-1078。

15. Kells A, et al. Regeneration of the MPTP-lesioned dopaminergic system after convection-enhanced delivery of AAV2-GDNF. J Neurosci. 2010 Jul 14;30(28):9567-77.

15. Kells A, 等。对流增强递送AAV2-GDNF后MPTP损伤的多巴胺能系统的再生。《神经科学杂志》。2010年7月14日；30(28):9567-77。

16. Lin L, et al. GDNF: a glial cell line-derived neurotrophic factor for midbrain dopaminergic neurons. Science. 1993;260(5111):1130-1132.

16. 林琳等。GDNF：一种对中脑多巴胺能神经元起作用的胶质细胞系源性神经营养因子。《科学》。1993；260（5111）：1130-1132。

17. Barker RA, et al. GDNF and Parkinson’s Disease: Where Next? A Summary from a Recent Workshop. J Parkinsons Dis. 2020;10(3):875-891.

17. 巴克尔 RA 等。GDNF 与帕金森病：接下来的方向？最近研讨会的总结。《帕金森病杂志》。2020年；10(3): 875-891。

18. Clinicaltrials.gov. GDNF Gene Therapy for Parkinson’s Disease. Available at:

18. Clinicaltrials.gov。GDNF基因疗法治疗帕金森病。可见于：

https://clinicaltrials.gov/study/NCT04167540

. Accessed: November 2025.

访问日期：2025年11月。

19. ClinicalTrials.gov. A Study of AAV2-GDNF in Adults With Moderate Parkinson’s Disease (REGENERATE-PD). Available at:

19. ClinicalTrials.gov. 一项关于中度帕金森病成人使用AAV2-GDNF的研究（REGENERATE-PD）。可用地址：

https://clinicaltrials.gov/study/NCT06285643

. Accessed: November 2025.

访问日期：2025年11月。

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

20. 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: November 2025.

访问日期：2025年11月。

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

21. 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: November 2025.

访问日期：2025年11月。

22. American Heart Association. Heart Failure Signs and Symptoms. Available at:

22. 美国心脏协会。心力衰竭的体征和症状。可访问：

https://www.heart.org/en/health-topics/heart-failure/warning-signs-of-heart-failure

. Accessed November 2025.

访问时间：2025年11月。

23. 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.

23. 萨瓦雷塞 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。

24. 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..

24. Henry T 等。使用合理设计的心脏趋向性AAV载体靶向蛋白磷酸酶抑制剂-1的I期临床基因治疗试验在晚期心力衰竭患者中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。

25. 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.

25. Nicolaou P 和 Kranias 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。

26. Henry, T. et. al. Cardiotropic AAV gene therapy for heart failure: a phase 1 trial. Nature Medicine. 2025 Oct 21; 10.1038/s41591-025-04011-z.

26. Henry, T. 等。心力衰竭的心脏靶向AAV基因治疗：一期试验。《自然医学》。2025年10月21日；10.1038/s41591-025-04011-z。

27. Clinicaltrials.gov. Phosphatase Inhibition by Intracoronary Gene Therapy in Subjects With Non-Ischemic NYHA Class III Heart Failure (GenePHIT). Available at:

27. Clinicaltrials.gov。非缺血性纽约心脏协会III级心力衰竭患者冠状动脉内基因治疗的磷酸酶抑制（GenePHIT）。可用链接：

https://www.clinicaltrials.gov/study/NCT05598333

. Accessed: November 2025.

访问日期：2025年11月。

Contact for media inquiries Bayer:

媒体咨询请联系拜耳：

Dr. Imke Meyer, phone +49 (214) 600 012 75

伊姆克·迈耶博士，电话 +49 (214) 600 012 75

Email:

电子邮件：

imke.meyer@bayer.com

Contact for media inquiries AskBio: