是时候让先进疗法更加个性化了——我们如何在欧洲为更有效的ATMP开发创造合适的生态系统?

E. Oldoni, A. Ussi, A. L. Andreu, D. Morrow
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引用次数: 0

摘要

在过去的几年中,先进治疗药物产品(ATMPs),特别是细胞和基因疗法,带来了治疗学领域的显著变革。atmp有可能根据其独特的分子特征为个体患者量身定制,使其成为个性化医疗(PM)策略的关键方面。释放atmp的全部潜力对于它们成为未来的治疗方法至关重要。尽管它们有着巨大的希望,但它们的成功受到严重复杂性的阻碍,正如科学、临床实施和监管领域的各种系统性瓶颈所证明的那样。目前,atmp面临的挑战包括对每位患者体内细胞命运的理解和可预测性有限、技术快速进步引起的监管问题、数据采集标准化不足、临床前开发可重复性有限以及关键利益相关者之间的知识交流不足。解决这些问题对于充分利用atmp在医疗保健中的优势至关重要。欧洲转化医学研究基础设施(EATRIS)正通过一系列关键举措积极提高其在PM领域的能力。这些努力旨在支持ATMP的开发,并专注于为科学界提供新颖和创新的科学工具。最终目标是通过更好地为学术界和工业界翻译ATMP以造福患者,为欧洲更有效的ATMP开发创造合适的生态系统。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
It is time we got more personal with advanced therapies- How do we create the right ecosystem for more effective ATMP development in Europe?
Over the past few years, Advanced Therapy Medicinal Products (ATMPs), especially cell and gene therapies, have brought about a remarkable transformation in the field of therapeutics. ATMPs have the potential to be tailored to individual patients based on their distinct molecular characteristics, making them a crucial aspect of personalized medicine (PM) strategies. Unlocking the full potential of ATMPs is crucial for them to become the treatments of the future. Despite their immense promise, their success is hindered by significant complexity, as evidenced by various systemic bottlenecks in the realms of science, clinical implementation, and regulation. Presently, ATMPs face challenges such as a limited understanding and predictability of in vivo cell fate specific to each patient, regulatory issues caused by rapid technological advancements, inadequate standardization in data acquisition, limited reproducibility during preclinical development, and insufficient knowledge exchange among key stakeholders. Addressing these aspects is essential to fully harness the benefits of ATMPs in healthcare. EATRIS, the European Research Infrastructure for Translational Medicine, is actively enhancing its capabilities in the field of PM through a series of key initiatives. These efforts aim to support also ATMP development and are focused on delivering novel and innovative scientific tools for the scientific community. The final aim is to create the right ecosystem for more effective ATMP development in Europe, by better serving academia and industry in the translation of ATMPs for patient benefit.
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