AskBio推进了肢带型肌营养不良2I/R9基因治疗临床试验，第二组首位参与者已给药-动脉网

AskBio Advances Gene Therapy Clinical Trial for Limb-Girdle Muscular Dystrophy Type 2I/R9 with Dosing of First Participant in Second Cohort

拜耳等信源发布 2025-03-07 21:08

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March

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2025

14:00 PM

Europe/Amsterdam

欧洲/阿姆斯特丹

AskBio Advances Gene Therapy Clinical Trial for Limb-Girdle Muscular Dystrophy Type 2I/R9 with Dosing of First Participant in Second Cohort

AskBio推进了肢带型肌营养不良2I/R9基因治疗临床试验，第二组首位参与者已接受剂量。

Summary

摘要

Data Safety Monitoring Board (DSMB) recommendation to initiate second cohort follows review of Phase 1/Phase 2 LION-CS101 trial safety data for AB-1003 / Second cohort enrollment is ongoing

数据安全监测委员会（DSMB）建议在审查AB-1003的1/2期LION-CS101试验安全性数据后启动第二组受试者招募/第二组受试者招募正在进行中

Research Triangle Park, N.C. – March 7, 2025 –

北卡罗来纳州研究三角园区 —— 2025年3月7日

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced the advancement of the Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) with dosing of the first participant in the second cohort.

AskBio Inc.（AskBio），一家由拜耳公司全资拥有并独立运营的基因治疗子公司，今日宣布了针对肢带型肌营养不良2I/R9型（LGMD2I/R9）患者的在研基因疗法AB-1003的1/2期LION-CS101临床试验取得进展，并完成了第二组首位受试者的给药。

The recommendation to advance to the second cohort followed the completion of a Data Safety Monitoring Board (DSMB) review of trial recruitment activity, and safety reporting from the first cohort that determined it was safe to proceed to cohort two. DSMBs are independent groups of experts appointed to periodically review information from clinical trials.

推荐进入第二组的决定是在数据安全监测委员会（DSMB）对试验招募活动进行了审查，并根据第一组的安全报告确定可以安全进入第二组后作出的。DSMB 是由独立专家组成的小组，负责定期审查来自临床试验的信息。

These reviews typically include risk-benefit assessments and monitoring for serious or unexpected adverse safety events alongside any significant benefit of therapies..

这些审查通常包括风险-收益评估，以及对严重或意外的不良安全事件和任何显著的治疗效益进行监测。

The LION-CS101 clinical trial is a double-blind, randomized, placebo-controlled, dose-escalation clinical trial to evaluate the safety of AB-1003 gene therapy in adult participants (18–65 years) who have genetic confirmation of LGMD2I/R9. The trial includes two sequential, dose-level cohorts. Adult participants diagnosed with LGMD2I/R9 will be given a single intravenous infusion of AB-1003 or placebo..

LION-CS101临床试验是一项双盲、随机、安慰剂对照、剂量递增的临床试验，旨在评估AB-1003基因疗法在18至65岁具有LGMD2I/R9基因确认的成年参与者中的安全性。该试验包括两个连续的剂量组。被诊断为LGMD2I/R9的成年参与者将接受单次静脉输注AB-1003或安慰剂。

The trial was initiated in 2023. It will include up to 14 participants at six sites throughout the United States.

该试验于2023年启动，将在美国六个地点包括多达14名参与者。

Participants in the first cohort remain in the study until completion. Enrollment in cohort two is ongoing. For more information about the LION-CS101 clinical trial, visit clinicaltrials.gov (NCT05230459) or askbio.com.

第一组的参与者将继续留在研究中直至完成。第二组的招募正在进行中。如需更多关于LION-CS101临床试验的信息，请访问clinicaltrials.gov（NCT05230459）或askbio.com。

“The burden of LGMD2I/R9 on patients and their families is profound,” said Nicholas Johnson, MD, Principal Investigator and Vice Chair of Research at the Department of Neurology, Virginia Commonwealth University School of Medicine. “Dosing the first participant in the second cohort of the trial is positive news for people living with LGMD2I/R9.

“LGMD2I/R9对患者及其家庭的负担是深远的，”弗吉尼亚联邦大学医学院神经病学系研究副主任兼首席研究员Nicholas Johnson博士说道。“为试验的第二组参与者进行首次给药，这对LGMD2I/R9患者来说是一个积极的消息。”

This is a rare and debilitating type of muscular dystrophy, and this advancement brings the LION-CS101 trial another step closer to completion.”.

这是一种罕见且使人衰弱的肌肉萎缩症，这一进展使LION-CS101试验更接近完成。”

AskBio has received rare pediatric disease designation (RPDD), orphan-drug designation (ODD) and fast track designation (FTD) for AB-1003 for the potential treatment of LGMD2I/R9 from the United States Food and Drug Administration (FDA).

AskBio 已获得美国食品药品监督管理局（FDA）授予的AB-1003用于潜在治疗LGMD2I/R9的罕见儿科疾病认定（RPDD）、孤儿药认定（ODD）和快速通道认定（FTD）。

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These designations serve as clear recognition of the significant unmet medical need in LGMD2I/R9, for which there is no approved therapy.

这些指定明确承认了LGMD2I/R9中存在显著未满足的医疗需求，目前尚无获批的疗法。

“The dosing of the first participant in cohort two marks an important milestone for the trial as enrollment continues for LION-CS101,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We are encouraged by the DSMB’s recommendation to advance our study, following their thorough assessment of AB-1003 in cohort one and are excited to proceed with the second cohort.” .

“第二组队列的首位参与者给药标志着该试验的一个重要里程碑，因为LION-CS101的招募仍在继续，” AskBio首席开发官兼首席医学官Canwen Jiang博士表示。“在数据安全监查委员会（DSMB）对第一组队列中的AB-1003进行彻底评估后，他们建议我们推进研究，这让我们备受鼓舞，并且我们很高兴能够继续开展第二组队列的研究。”

AB-1003 is an investigational recombinant adeno-associative virus (AAV)-based gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been fully established or evaluated. It is designed to restore FKRP enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9 as a one-time intravenous (IV) infusion..

AB-1003 是一种研究性重组腺相关病毒 (AAV) 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完全确立或评估。该疗法旨在恢复肌肉细胞内的 FKRP 酶活性，用于治疗 LGMD2I/R9，通过一次性静脉 (IV) 输注进行。

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About Limb-Girdle Muscular Dystrophy Type 2I/R9

关于肢带型肌营养不良症2I/R9型

LGMD2I/R9 is a rare form of LGMD caused by mutations in the FKRP gene and is associated with weakness and wasting of arm and leg muscles.

LGMD2I/R9是一种由FKRP基因突变引起的罕见LGMD类型，与四肢肌肉的无力和萎缩有关。

Those with LGMD2I/R9 notice symptoms in late childhood, typically around 11 years of age, and may experience difficulty running and walking. The symptoms gradually worsen over time, and affected people generally rely on wheelchairs for mobility and may experience impaired heart and lung function.

那些患有LGMD2I/R9的人在儿童晚期，通常在11岁左右开始出现症状，并可能经历跑步和行走困难。症状会随着时间的推移逐渐加重，受影响的人通常依靠轮椅行动，并可能出现心脏和肺功能受损的情况。

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LGMD2I/R9 is estimated to affect fewer than 5,000 people in the United States.

LGMD2I/R9在美国影响的人数估计不到5000人。

Currently, there is no approved treatment, and management is based on the signs and symptoms present in each individual.

目前，尚无获批的治疗方法，管理是基于每个个体所表现出的体征和症状。

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 developing life-saving medicines and changing lives. The company maintains a portfolio of clinical programs across a range of neuromuscular, central nervous system, cardiovascular, and metabolic disease indications 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，致力于开发挽救生命的药物并改变生命。该公司在神经肌肉、中枢神经系统、心血管和代谢疾病适应症范围内拥有多项临床计划，其临床阶段产品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 gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive array of capsids and promoters. 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的基因治疗平台包括Pro10™，这是一种行业领先的专有细胞系制造工艺，并拥有大量的衣壳和启动子。公司总部位于北卡罗来纳州研究三角园区，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 .

作为基因治疗领域的早期创新者，该公司在五个国家拥有超过900名员工，并在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。

www.askbio.com

or follow us on LinkedIn.

或在LinkedIn上关注我们。

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，无人饥饿”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 www.bayer.com..

研发费用达62亿欧元。欲了解更多信息，请访问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，包括无法达到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。这些原因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

1. Van Norman GA. Data Safety and Monitoring Boards Should Be Required for Both Early- and Late-Phase Clinical Trials. JACC Basic Transl Sci. 2021;6(11):887-896.

1. 范·诺曼 GA。数据安全和监查委员会应成为早期和晚期临床试验的必要条件。《JACC 基础与转化科学》。2021年；6(11)：887-896。

2. AskBio Announces First Patient Dosed in Phase 1 / Phase 2 Trial of AB-1003 Gene Therapy for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). Available at:

2. AskBio宣布AB-1003基因疗法针对肢带型肌营养不良2I/R9型（LGMD2I/R9）的1/2期试验中已完成首例患者给药。相关信息可查阅：

https://www.askbio.com/askbio-announces-first-patient-dosed-in-phase-1-phase-2-lgmd2i-r9/

. Accessed March 2025.

访问时间：2025年3月。

3. AskBio Receives FDA Rare Pediatric Disease and Orphan-Drug Designations for AB-1003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9. Available at:

3. AskBio公司获得FDA罕见儿科疾病和孤儿药资格认定，用于治疗肢带型肌营养不良症2I/R9型的AB-1003。相关信息可查阅：

https://www.askbio.com/askbio-receives-fda-rare-pediatric-disease-and-orphan-drug-designations-for-ab-1003-for-the-treatment-of-limb-girdle-muscular-dystrophy-type-2i-r9/

https://www.askbio.com/askbio获得FDA罕见儿科疾病和孤儿药指定AB-1003用于治疗肢带型肌营养不良症2i型R9/

. Accessed March 2025.

访问时间：2025年3月。

4. AskBio Receives FDA Fast Track Designation for LION-101, a Novel Investigational AAV Gene Therapy for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). Available at:

4. AskBio 获得 FDA 快速通道指定，用于 LION-101，一种新型的 AAV 基因疗法，用于治疗肢带型肌营养不良症 2I/R9 (LGMD2I/R9)。可用链接：

https://www.askbio.com/askbio-receives-fda-fast-track-designation-for-lion-101-a-novel-investigational-aav-gene-therapy-for-the-treatment-of-limb-girdle-muscular-dystrophy-type-2i-r9-lgmd2i-r9/

https://www.askbio.com/askbio获得FDA快速通道指定lion-101一种新型研究性AAV基因疗法用于治疗肢带型肌营养不良2i型r9-lgmd2i-r9/

. Accessed March 2025.

访问时间：2025年3月。

5. Limb-Girdle Muscular Dystrophy (LGMD). Available at:

5. 肢带型肌营养不良症 (LGMD)。可用资源：

https://my.clevelandclinic.org/health/diseases/limb-girdle-muscular-dystrophy-lgmd

https://my.clevelandclinic.org/health/diseases/肢带型肌营养不良症-LGMD

. Accessed March 2025.

。访问时间：2025年3月。

6. NIH – Autosomal recessive limb-girdle muscular dystrophy type 2i. Available at:

6. NIH – 常染色体隐性遗传的肢带型肌营养不良症2i型。可于以下网址获取：

https://rarediseases.info.nih.gov/diseases/12533/autosomal-recessive-limb-girdle-muscular-dystrophy-type-2i

https://rarediseases.info.nih.gov/diseases/12533/常染色体隐性遗传肢带型肌营养不良症2I型

. Accessed March 2025.

. 2025年3月访问。

7. Willis E, Moore SA, Cox MO, Stefans V, Aravindhan A, Gokden M, Veerapandiyan A. Limb-Girdle Muscular Dystrophy R9 due to a Novel Complex Insertion/Duplication Variant in FKRP Gene. Child Neurol Open. 2022 Apr 28;9.

7. Willis E, Moore SA, Cox MO, Stefans V, Aravindhan A, Gokden M, Veerapandiyan A. 由 FKRP 基因中一种新的复杂插入/重复变异引起的肢带型肌营养不良 R9。《儿童神经病学开放期刊》。2022年4月28日；第9卷。

Media Contact:

媒体联系人:

Phil McNamara

菲尔·麦克纳马拉

Vice President, Corporate Communications, AskBio

副总裁，企业传播，AskBio

错误：

mcnamara@askbio.com

麦克纳马拉@askbio.com

T +1 (984) 520 7211

电话：+1 (984) 520 7211