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4CPS-297 Evolution of clinical trials with advanced therapy medicinal products at a tertiary care centre: experience 2022–2025

ejhpharm · 2026-03-18 · canonical JSON source

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Background and Importance Advanced Therapy Medicinal Products (ATMPs) are innovative medicines for human use and are classified into three main categories:Gene therapy: uses recombinant genes created in the laboratory to treat, prevent or diagnose diseases.Somatic–cell therapy: contains manipulated cells or tissues with altered characteristics or new functions, used for treatment, diagnosis or prevention.Tissue–engineered: use modified cells or tissues to repair, regenerate or replace human tissue. Over the past few years, we have witnessed substantial growth in the number of clinical trials (CT) in this field.Aim and Objectives To evaluate the growth and identify emerging trends in the development of ATMPs at our hospital during the last few years.Material and Methods Observational, descriptive, and retrospective study with a comparative analysis of the evolution of CT in ATMPs at a tertiary care hospital between 2022-2025. The variables analysed included the number of CT of ATMPs, type of ATMPs (gene therapy, somatic-cell therapy or tissue-engineered), number of patients per trial and per therapy type during the specified period.Results We participated in 38 CT with ATMPs, 28 of them were conducted between 2022-2025 ( table 1):46.5% Gene therapy: 53.8% RNAm, 38.5% viral vectors and 7.7% others.53.5% Somatic–cell therapy: 60.0% Chimeric Antigen Receptor T–cell (CAR–T), 13.3% tumour–infiltrating lymphocytes (TIL), 13.3% Mesenchymal Stromal Cells (MSC), 6.7% lymphocyte T–citotoxic (LTC) and 6.7% Autologous Engineered Macrophages (AEM).We have not participated in any CT involving tissue–engineered.We treated 55 patients with ATMPs between 2022-2025 (table 2):54.5% Gene therapy: 53.8% RNAm, 38.5% viral vectors and 7.7% others.45.5% Somatic–cell therapy: 63.6% CAR–T, 4.5% TIL, 22.7% MSC, 4.5% LTC and 4.5% AEM.Abstract 4CPS-297 Table 1CT with ATMPs between 2022 and 2025Abstract 4CPS-297 Table 2Patients included in CT with ATMPs between 2022–2025Conclusion and Relevance A substantial increase was observed in both the number of CT and patients treated with ATMPs. mRNA and CAR-T therapies showed the most pronounced growth starting from 2024. In contrast, viral vectors, MSC, and LTC exhibited declining trends. Notably, in 2025, the centre engaged in an increasing number of novel therapies, including TILs and AEMs, reflecting diversification and consolidation of expertise across advanced therapeutic modalities. The slight decline in 2025 is likely due to the pending completion of the final quarter.Conflict of Interest No conflict of interest