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

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Authors: Sagi Nahum, Devanshi Doshi, Cris Kosnik, Emiko Jeffries, Kristine S. Nishida, Vered Caplan, Heiko v. der Leyen
Cited by: 1
References: 48

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.

Fields of Study & Subjects

CAR-T cell therapy research Biomedical Ethics and Regulation Viral Infectious Diseases and Gene Expression in Insects

Publication details

Journal / source
Regenerative Therapy
Publisher
Elsevier BV
Volume
32
Pages
101112–101112
Publication date
21 April 2026
Language
EN