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Automated GMP-compatible production of universal CAR Tregs for organ-targeted tolerance induction

Title: Automated GMP-compatible production of universal CAR Tregs for organ-targeted tolerance induction
Authors: Kavitha Lakshmi; Alexandra von Jutrzenka-Trzebiatowski; Liliana Loureiro; Karla Elizabeth González Soto; Katja Peter; José Manuel Marín Morales; Samikshya Santosh Nirmala; Nicole Berndt; Claudia Arndt; Yueyuan Hu; Jing-Wun Li; Claudia Peitzsch; Anna Taubenberger; Rebekka Wehner; Marc Schmitz; Kristina Hölig; Hinrich Abken; Ezio Bonifacio; Martin Bornhäuser; Michael Bachmann; Anja Feldmann; Anke Fuchs
Source: Journal of Translational Medicine, Vol 23, Iss 1, Pp 1-22 (2025)
Publisher Information: BMC
Publication Year: 2025
Collection: Directory of Open Access Journals: DOAJ Articles
Subject Terms: Regulatory T cells; Treg cell therapy; Universal adapter CAR; Precision immunotherapy; Good manufacturing practice; Closed-system manufacturing; Medicine
Description: Background Adoptive transfer of regulatory T cells (Tregs) has demonstrated safety, feasibility and early signs of efficacy in promoting immunological tolerance in inflammatory conditions such as graft-versus-host disease (GvHD). Chimeric antigen receptor (CAR)-engineered Tregs offer localized activation and suppression compared to polyclonal Tregs, but their clinical translation is limited by high manufacturing costs, lengthy developing times and fixed single-antigen specificity. To address these limitations, we employed the universal adapter Reverse CAR (RevCAR) system, which harbors a peptide epitope lacking intrinsic antigen specificity but provides flexibility in targeting through the use of an antigen-specific RevCAR Target Module (RevTM). As a proof-of-concept, we used a RevTM targeting carcinoembryonic antigen (CEA), which is highly expressed in the gastrointestinal (GI) tract, as a potential strategy to achieve localized immunosuppression in GI acute GvHD. Methods To support clinical translation, we established an automated, GMP-compatible, clinical-scale manufacturing process. Tregs were magnetically enriched from leukapheresis using the CliniMACS® Plus, followed by high-purity sorting on the MACSQuant® Tyto®. The sorted cells were virally transduced and the RevCAR Tregs were expanded on the CliniMACS Prodigy® to obtain clinically relevant cell numbers. The harvested products were evaluated for phenotype, stability, antigen specificity and suppressive function. Results Across five manufacturing runs, Tregs (CD4+CD25highCD127lowFOXP3+) with a median initial purity of 94% were expanded to achieve a median therapeutic yield of 602 × 106 cells. The final product maintained a high purity (median: 91.9%) and exhibited high RevCAR expression (median: 60% RevCAR+). Mass cytometry analysis revealed that expanded RevCAR Tregs predominantly exhibited a central memory phenotype with high expression of functional and homing markers. Under experimental pro-inflammatory conditions, the cells maintained ...
Document Type: article in journal/newspaper
Language: English
Relation: https://doi.org/10.1186/s12967-025-07431-0; https://doaj.org/toc/1479-5876; https://doaj.org/article/929cd19b5d9e4b7aaa22410e4440d55a
DOI: 10.1186/s12967-025-07431-0
Availability: https://doi.org/10.1186/s12967-025-07431-0; https://doaj.org/article/929cd19b5d9e4b7aaa22410e4440d55a
Accession Number: edsbas.7D451713
Database: BASE