Bio-Techne的Simple Western™技术在最近FDA批准ZEVASKYN™细胞基因疗法中得到应用-动脉网

Bio-Techne's Simple Western™ Technology Utilized in Recent FDA Approval of ZEVASKYN™ Cellased Gene Therapy

CISION 等信源发布 2025-07-14 18:59

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MINNEAPOLIS

明尼阿波利斯

，

July 14, 2025

2025年7月14日

/PRNewswire/ -- Bio-Techne Corporation (NASDAQ:

TECH

技术

) today announced that its Simple Western™ Technology played a key role in supporting the FDA approval of ZEVASKYN™ (prademagene zamikeracel), the first autologous cell-based gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB), developed by Abeona Therapeutics

）今天宣布其Simple Western™技术在支持Abeona Therapeutics开发的ZEVASKYN™（prademagene zamikeracel）获得FDA批准中发挥了关键作用，这是首个针对隐性营养不良性大疱性表皮松解症（RDEB）患者的自体细胞基因疗法。

(NASDAQ:

（纳斯达克：

ABEO

). Throughout clinical development, Simple Western enabled precise identification and quantification of Collagen VII, a complex therapeutic target critical to ZEVASKYN's potency, supporting lot release testing for the viral vector and the cell therapy in a GMP setting.

). 在整个临床开发过程中，Simple Western 实现了对复杂治疗靶点胶原蛋白 VII 的精确识别和定量，这对 ZEVASKYN 的效力至关重要，并在 GMP 环境下支持了病毒载体和细胞疗法的批次放行测试。

Abeona selected Simple Western over competing methods like traditional western blot for its speed, reproducibility, picogram-level sensitivity, and minimal sample volume requirements. The platform effectively detected both the trimeric and monomeric forms of Collagen VII, addressing the analytical challenges posed by the protein's size and structure and supporting the regulatory requirements for potency and consistency..

阿贝奥纳公司选择了Simple Western方法，而非传统的蛋白质印迹等竞争方法，原因是其速度快、可重复性高、灵敏度达到皮克级别，并且所需样品量极少。该平台有效检测了胶原蛋白VII的三聚体和单体形式，解决了因该蛋白质大小和结构带来的分析挑战，并支持了效价和一致性的监管要求。

RDEB is a rare and debilitating genetic skin disorder caused by mutations in the

RDEB是一种由基因突变引起的罕见且使人衰弱的遗传性皮肤病。

COL7A1

gene, which leads to the misfolding of Collagen VII, a protein essential for skin integrity. Patients with RDEB suffer from severe blistering, chronic wounds, scarring, and are at significantly increased risk of life-threatening complications such as squamous cell carcinoma. The current standard of care focuses on symptomatic management, with no durable therapies available until now..

基因，导致胶原蛋白VII的错误折叠，这种蛋白质对皮肤完整性至关重要。RDEB患者遭受严重的水疱、慢性伤口、疤痕，并且面临显著增加的危及生命的并发症风险，如鳞状细胞癌。目前的护理标准侧重于症状管理，至今尚无持久的治疗方法。

The Simple Western platform provided quantifiable, GMP-compliant data to ensure consistency of each autologous ZEVASKYN product lot, overcoming key challenges in analytical standardization, limited sample availability, and complex assay development. Abeona's final assay design was optimized through rigorous antibody selection, assay optimization, and use of an appropriate reference standard – enabled by Simple Western's automated and high-throughput capabilities..

Simple Western平台提供了可量化的、符合GMP规范的数据，以确保每个自体ZEVASKYN产品批次的一致性，克服了分析标准化、样品可用性有限和复杂检测开发等关键挑战。Abeona的最终检测设计通过严格的抗体选择、检测优化以及使用适当的参考标准进行了优化，这些都得益于Simple Western的自动化和高通量能力。

'ZEVASKYN represents a significant milestone for patients with RDEB and for the field of cell-based gene therapy,' said

‘ZEVASKYN代表了RDEB患者和基于细胞的基因治疗领域的一个重要里程碑，’表示

Will Geist

威尔·盖斯特

, Bio-Techne's President, Protein Sciences Segment. 'We are honored that our Simple Western platform helped Abeona develop and validate critical potency assays that supported FDA approval of this first-in-class therapy.'

，Bio-Techne公司蛋白质科学部门总裁。“我们感到荣幸的是，我们的Simple Western平台帮助Abeona开发并验证了关键的效力测定方法，支持了这一首创疗法获得FDA的批准。”

'Potency method development and validation were critical components in the clinical development and regulatory approval of ZEVASKYN,' said Dr. Ann Durbin, Senior Director of Quality Control at Abeona Therapeutics. 'The Simple Western platform was the best choice for our quality control laboratories due to the reproducibility, time-to-result, and GMP compliance of the platform.

“效力方法的开发和验证是ZEVASKYN临床开发和监管审批的关键组成部分，”Abeona Therapeutics质量控制高级总监Ann Durbin博士表示，“Simple Western平台因其可重复性、结果产出速度以及符合GMP标准，成为我们质量控制实验室的最佳选择。”

We collaborated with Bio-Techne's ProteinSimple to address the challenges of our large molecular weight analyte, Collagen VII, and our requirement to evaluate the protein's tertiary structure under non-denaturing conditions. With the capillary electrophoresis platform, our validated assay efficiently quantifies Collagen VII to support both release testing of autologous ZEVASKYN lots, and the release and stability testing of our viral vector.

我们与Bio-Techne的ProteinSimple合作，解决了我们大分子量分析物——VII型胶原蛋白的挑战，以及在非变性条件下评估该蛋白质三级结构的需求。通过毛细管电泳平台，我们经过验证的检测方法能够高效定量VII型胶原蛋白，以支持自体ZEVASKYN批次的放行测试，以及病毒载体的放行和稳定性测试。

The partnership of scientists at Abeona and at ProteinSimple was instrumental in advancing ZEVASKYN as the first approved cell-based gene therapy for patients with RDEB.'.

Abeona和ProteinSimple的科学家们的合作对推进ZEVASKYN作为首个获批的针对RDEB患者的基于细胞的基因疗法起到了关键作用。

About Bio-Techne

关于Bio-Techne

Bio-Techne Corporation (NASDAQ:

生物科技公司（纳斯达克：

TECH

技术

) is a global life sciences company providing innovative tools and bioactive reagents for the research and clinical diagnostic communities. Bio-Techne products assist scientific investigations into biological processes and the nature and progress of specific diseases. They aid in drug discovery efforts and provide the means for accurate clinical tests and diagnoses.

）是一家全球生命科学公司，为科研和临床诊断界提供创新工具和生物活性试剂。Bio-Techne 产品助力对生物过程以及特定疾病的性质和进展的科学研究。它们帮助药物发现工作，并提供准确的临床测试和诊断手段。

With hundreds of thousands of products in its portfolio, Bio-Techne generated approximately .

由于其产品组合中有数十万个产品，Bio-Techne 的年收入大约为 .

$1.2 billion

12亿美元

in net sales in fiscal 2024 and has approximately 3,100 employees worldwide. For more information on Bio-Techne and its brands, please visit

在2024财年净销售额达到，并在全球拥有约3,100名员工。有关Bio-Techne及其品牌的更多信息，请访问

http://www.bio-techne.com

or follow the Company on social media at

或在社交媒体上关注公司

Facebook

，

领英

，

Twitter

或

YouTube

。

About Bio-Techne Corporation

关于Bio-Techne公司

(NASDAQ:

（纳斯达克：

TECH

技术

)

Contact: David Clair, Vice President, Investor Relations & Corporate Development

联系人：大卫·克莱尔，副总裁，投资者关系与企业发展部

david.clair@bio-techne.com

612-656-4416

About Abeona Therapeutics

关于Abeona Therapeutics

Abeona Therapeutics Inc. is a commercial-stage biopharmaceutical company developing cell and gene therapies for serious diseases. Abeona's ZEVASKYN™ (prademagene zamikeracel) is the first and only autologous cell-based gene therapy for the treatment of wounds in adults and pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB).

Abeona Therapeutics Inc. 是一家处于商业化阶段的生物制药公司，致力于开发用于严重疾病的细胞和基因疗法。Abeona 的 ZEVASKYN™（prademagene zamikeracel）是首个也是唯一一个用于治疗隐性营养不良型大疱性表皮松解症（RDEB）成人和儿童患者的自体细胞基因疗法。

The Company's fully integrated cell and gene therapy cGMP manufacturing facility in .

公司完全集成的细胞和基因治疗cGMP生产设施位于。

Cleveland, Ohio

克利夫兰，俄亥俄州

serves as the manufacturing site for ZEVASKYN commercial production. The Company's development portfolio features adeno-associated virus (AAV)-based gene therapies for ophthalmic diseases with high unmet medical need. Abeona's novel, next-generation AAV capsids are being evaluated to improve tropism profiles for a variety of devastating diseases.

作为ZEVASKYN商业生产的制造基地。该公司的发展组合包括基于腺相关病毒（AAV）的基因疗法，用于治疗具有高度未满足医疗需求的眼科疾病。Abeona的新型下一代AAV衣壳正在被评估，以改善多种严重疾病的向性特征。

For more information, visit .

欲了解更多信息，请访问。

www.abeonatherapeutics.com

。

ZEVASKYN™, Abeona Assist™, Abeona Therapeutics

ZEVASKYN™，Abeona Assist™，Abeona Therapeutics

, and their related logos are trademarks of Abeona Therapeutics Inc.

，及其相关徽标是Abeona Therapeutics公司的商标。

SOURCE Bio-Techne Corporation

来源：Bio-Techne Corporation

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