先进疗法药品（ATMPs）是利用细胞和/或基因的药理学、免疫学或代谢特性，旨在恢复、纠正或修改受体的生物功能的创新临床治疗。ATMPs是异质的药物产品，主要开发为个体化和特定患者的治疗，并代表了罕见、遗传性和神经退行性疾病、血液恶性肿瘤、癌症、自身免疫、炎症和骨科疾病等有高医疗需求的疾病的新机会。进入欧洲联盟（EU）市场，第一种ATMPs于2009年推出，截至目前已批准了24种ATMPs。本综述旨在报道EU授权的基于细胞的疗法的当前证据，包括体细胞疗法、组织工程产品和基于细胞的基因疗法产品，如嵌合抗原受体（CAR）T细胞，重点关注在临床试验和现实世界环境中的疗效和安全性评估。尽管基于细胞的疗法对于治疗选择有限的患者具有重要意义，但其在临床实践中的广泛应用仍存在一些限制，包括适应症有限、生产工艺高度复杂、生产成本高、细胞产品易变性以及与活细胞的内在特性相关的潜在安全问题，包括CAR-T诱导的神经毒性和细胞因子释放综合征（CRS）等严重或危及生命的毒性风险。尽管鼓舞人心的研究结果支持ATMPs的临床应用，但还需要额外的数据、长期随访的比较研究和更广泛的现实世界证据，以提供进一步的疗效和安全性的见解。版权所有©2023 Bellino、La Salvia、Cometa和Botta。

Advanced Therapy Medicinal Products (ATMPs) are innovative clinical treatments exploiting the pharmacological, immunological, or metabolic properties of cells and/or gene(s) with the aim to restore, correct, or modify a biological function in the recipient. ATMPs are heterogeneous medicinal products, developed mainly as individualized and patient-specific treatments, and represent new opportunities for diseases characterized by a high-unmet medical need, including rare, genetic and neurodegenerative disorders, haematological malignancies, cancer, autoimmune, inflammatory and orthopaedic conditions. Into the European Union (EU) market, the first ATMP has been launched in 2009 and, to date, a total of 24 ATMPs have been approved. This review aims at reporting on current evidence of cell-based therapies authorized in the EU, including Somatic Cell Therapies, Tissue Engineering Products, and Cell-based Gene Therapy Products as Chimeric Antigen Receptor (CAR) T-cells, focusing on the evaluation of efficacy and safety in clinical trials and real-world settings. Despite cell-based therapy representing a substantial promise for patients with very limited treatment options, some limitations for its widespread use in the clinical setting remain, including restricted indications, highly complex manufacturing processes, elevated production costs, the lability of cellular products over time, and the potential safety concerns related to the intrinsic characteristics of living cells, including the risk of severe or life-threatening toxicities, such as CAR-T induced neurotoxicity and cytokine release syndrome (CRS). Although encouraging findings support the clinical use of ATMPs, additional data, comparative studies with a long-term follow-up, and wider real-world evidences are needed to provide further insights into their efficacy and safety profiles.Copyright © 2023 Bellino, La Salvia, Cometa and Botta.