From production to bedside: A tiered QC framework for regulatory alignment and analytical comparability in decentralized CGT

从生产到临床:分散式细胞术中监管一致性和分析可比性的分层质量控制框架

阅读:1

Abstract

Decentralized and point-of-care (PoC) manufacturing is reshaping cell and gene therapy (CGT) by bringing production closer to patients. This model offers advantages, including reduced vein-to-vein times, streamlined logistics, and greater flexibility for patient-specific treatments, with the potential to improve affordability and broaden access. However, it introduces new quality control (QC) challenges that are central to ensuring product safety, potency, and consistency. Autologous CGTs, typically produced as single patient lots with limited shelf lives and compressed release timelines, require QC systems capable of delivering reliable results within hours for critical quality attributes (CQA) such as identity, potency, sterility, and safety. In both autologous and allogeneic settings, variability in starting material, shortages of trained personnel, and a lack of standardized assays across sites complicate consistency and hinder data pooling in multicenter trials. Hospital-based facilities also require enhanced infrastructure, harmonized procedures, and digital oversight to sustain network-wide comparability. Emerging solutions emphasize automation, validated rapid-testing platforms, and methods that minimize reliance on highly specialized expertise to accelerate batch release and improve reproducibility across distributed networks. Regulatory agencies are updating frameworks for modular and Point-of-Care (PoC) models, emphasizing flexibility while maintaining rigorous standards. Here, we outline a tiered QC model designed to maintain product comparability and enable timely release. QC has become the defining challenge of decentralized CGT manufacturing. However, implementing robust strategies, assay harmonization, and validated rapid-release methods can ensure uniform product quality across distributed sites, so that every patient, regardless of treatment site, receives safe, effective, and timely therapy.

特别声明

1、本页面内容包含部分的内容是基于公开信息的合理引用;引用内容仅为补充信息,不代表本站立场。

2、若认为本页面引用内容涉及侵权,请及时与本站联系,我们将第一时间处理。

3、其他媒体/个人如需使用本页面原创内容,需注明“来源:[生知库]”并获得授权;使用引用内容的,需自行联系原作者获得许可。

4、投稿及合作请联系:info@biocloudy.com。