Optimized GMP-grade production of non-viral Sleeping Beauty-generated CARCIK cells for enhanced fitness and clinical scalability
Optimizing non-viral CARCIK cell production using G-Rex bioreactors and refined timing adjustments has streamlined the workflow, enhanced cell fitness, and resulted in a highly effective therapeutic product with demonstrated in vivo efficacy in mice. These improvements reduced manipulation and contamination risks, while optimizing logistics and space efficiency, facilitating allogeneic CARCIK generation for a current phase I/II clinical trial (NCT05869279) in patients with R/R CD19 + non-Hodgkin Lymphoma (B-cell NHL) and Chronic Lymphocytic Leukemia (CLL), confirming the approach's scalability and clinical potential.
Authors
- Marta Serafini (ORCID: https://orcid.org/0000-0001-8105-6476)
- Elisa Gotti
- Jolanda Sarno (ORCID: https://orcid.org/0000-0003-3488-0248)
- Barbara Vergani (ORCID: https://orcid.org/0000-0002-8648-0660)
- Olga Pedrini (ORCID: https://orcid.org/0000-0002-5460-8367)
- Valentina Colombo (ORCID: https://orcid.org/0000-0002-0364-0463)
- Massimiliano Mazza (ORCID: https://orcid.org/0000-0001-9053-5743)
- Giada Matera (ORCID: https://orcid.org/0000-0001-8383-1974)
- Michele Quaroni
- Stefania Cesana (ORCID: https://orcid.org/0000-0001-5462-3519)
- Josée Golay (ORCID: https://orcid.org/0000-0002-7932-909X)
- Anna Gaimari (ORCID: https://orcid.org/0000-0002-7456-4197)
- Beatrice Landoni (ORCID: https://orcid.org/0000-0002-3815-5011)
- Chiara Capelli (ORCID: https://orcid.org/0000-0002-4454-1541)
- Sarah Tettamanti (ORCID: https://orcid.org/0000-0002-7327-418X)
- Carolina Cuofano
- Martino Introna (ORCID: https://orcid.org/0000-0001-6216-0892)
- Stefania Galimberti (ORCID: https://orcid.org/0000-0003-1504-7242)
- Patricia Borges de Souza (ORCID: https://orcid.org/0000-0001-6862-8302)
- Martine Bocchini (ORCID: https://orcid.org/0000-0001-7534-480X)
- Fabio Nicolini (ORCID: https://orcid.org/0000-0001-6846-5435)
- I. Pisani (ORCID: https://orcid.org/0000-0001-9914-2153)
- Giusi Melita (ORCID: https://orcid.org/0000-0001-9707-357X)
- Giuseppe Dastoli
- Angela Maria Savino (ORCID: https://orcid.org/0000-0002-4639-1161)
- Stefano Crippa (ORCID: https://orcid.org/0000-0002-7370-1508)
- Andrea Biondi (ORCID: https://orcid.org/0000-0002-6757-6173)
- Marcella Tazzari (ORCID: https://orcid.org/0000-0002-8112-1773)
- Alessandro Rambaldi (ORCID: https://orcid.org/0000-0002-3739-7502)
- Benedetta Cabiati
- Benedetta Rambaldi (ORCID: https://orcid.org/0000-0002-5246-9471)
- Giuseppe Gaipa (ORCID: https://orcid.org/0000-0002-1006-5946)
- Daniela Belotti
Institutions
- University of Milan (IT)
- Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori (IT)
- Ospedale Papa Giovanni XXIII (IT)
- Azienda Ospedaliera San Gerardo (IT)
- Ospedale San Giuseppe (IT)
- University of Milano-Bicocca (IT)
Publication Details
- Journal
- Journal of Translational Medicine
- Published
- 2025-05-19
- DOI
- https://doi.org/10.1186/s12967-025-06416-3
- Citations
- 2
- Primary Topic
- CAR-T cell therapy research
- Type
- article
- Field-Weighted Citation Impact
- 2.09
Funders
- Ministero della Salute
- Fondazione Regionale per la Ricerca Biomedica