AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced it will present initial safety data from the first cohort of participants from its Phase 1/Phase 2 LION-CS101 clinical trial of investigational gene therapy AB-1003 in participants with limb-girdle muscular dystrophy (LGMD) 2I/R9 at the 30th Annual International Congress of the World Muscle Society, taking place October 7–11, 2025, in Vienna, Austria..

AskBio Inc. (AskBio) 是一家基因治疗公司，作为拜耳集团的全资子公司独立运营，今天宣布将在2025年10月7日至11日于奥地利维也纳举行的第30届世界肌肉学会国际大会期间，展示其针对肢带型肌营养不良症（LGMD）2I/R9患者的1/2期LION-CS101临床试验中首个参与者队列的初步安全数据。

The presentation represents interim, blinded Cohort 1 safety data. Participants enrolled in Cohort 1 received a single intravenous infusion of AB-1003 or placebo and were followed for 52 weeks post-treatment during the main trial before entering a planned four-year long-term follow-up period. Safety assessments included adverse event monitoring, laboratory testing, physical exams, vital signs, electrocardiograms, and echocardiograms..

该报告展示了中期的、盲态的队列1安全性数据。队列1的参与者接受了单次静脉输注AB-1003或安慰剂，并在主要试验期间接受治疗后52周的随访，随后进入计划中的四年长期随访期。安全性评估包括不良事件监测、实验室检测、体格检查、生命体征、心电图和超声心动图。

There were no dose-limiting toxicities or serious adverse events reported up to 52 weeks post-treatment. Commonly reported (>2 participants) treatment-emergent adverse events were mild-to-moderate in severity and included headaches, falls, and nausea. Three participants reported asymptomatic transient transaminase elevations without changes in bilirubin levels, which returned to baseline levels after adjusting corticosteroid treatment..

治疗后长达52周内未报告剂量限制性毒性或严重不良事件。常见（>2名参与者）的治疗相关不良事件为轻至中度，包括头痛、跌倒和恶心。三名参与者报告了无症状的短暂转氨酶升高，胆红素水平未发生变化，在调整皮质类固醇治疗后转氨酶恢复到基线水平。

The data will be presented by Chris Passalacqua, MD, Vice President of Neuromuscular Medical Affairs, AskBio, at 3:45 p.m. CEST Friday, October 10, 2025.

该数据将于 2025 年 10 月 10 日星期五下午 3 点 45 分（欧洲中部夏令时间）由 AskBio 神经肌肉医学事务副总裁克里斯·帕萨拉奎医学博士发表。

“These initial safety data are encouraging and suggest an acceptable safety profile for AB-1003,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “We believe AAV-mediated gene therapy has the potential to restore FKRP function and stabilize disease progression, and we are excited to continue our clinical research efforts with the goal of developing an effective treatment for limb-girdle muscular dystrophy.”.

“这些初步的安全性数据令人鼓舞，表明AB-1003具有可接受的安全性特征，” AskBio公司首席开发官兼首席医学官姜灿文博士表示，“我们相信AAV介导的基因疗法有潜力恢复FKRP功能并稳定疾病进展，我们很高兴继续我们的临床研究工作，目标是为肢带型肌营养不良开发一种有效的治疗方法。”

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 receive 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岁经基因确认患有LGMD2I/R9的成人参与者中的安全性。该试验包括两个顺序进行的不同剂量组。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. Five participants were enrolled in the first cohort. All are actively participating and should remain in the trial until completion. Enrollment in Cohort 2 is ongoing. For more information about the LION-CS101 clinical trial, visit clinicaltrials.gov (NCT05230459) or askbio.com..

该试验于2023年启动，将在美国六个地点招募多达14名参与者。第一组已招募了五名参与者，他们均积极参与并将持续参与试验直至结束。第二组的招募正在进行中。如需了解更多关于LION-CS101临床试验的信息，请访问clinicaltrials.gov（NCT05230459）或askbio.com。

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 fukutin-related protein (FKRP) enzyme activity, primarily inside muscle cells, for the treatment of LGMD2I/R9 as a one-time intravenous (IV) infusion..

AB-1003 是一种研究性的基于重组腺相关病毒（AAV）的基因疗法，尚未获得任何监管机构的批准，其疗效和安全性也尚未完全确立或评估。它旨在恢复肌肉细胞内的福克汀相关蛋白（FKRP）酶活性，通过一次性静脉（IV）输注用于治疗LGMD2I/R9。

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，与手臂和腿部肌肉的无力和萎缩有关。

Symptoms may start to appear from childhood to adulthood, and affected individuals 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.

症状可能从儿童期到成年期开始出现，受影响的个体可能会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进行移动，并可能会经历心脏和肺功能受损。

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

LGMD2I/R9在美国估计影响不到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出现的症状和体征。

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领到一个新时代，使其能够改变更广泛患有罕见和常见疾病人群的生活。该公司在多个与单基因或多因素相关的疾病适应症中拥有临床项目组合，涵盖心血管、中枢神经系统和神经肌肉疾病等领域，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、多系统萎缩症、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级制造成本，使基因疗法更加普及。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，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。

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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，集团拥有约93,000名员工，销售额达466亿欧元。