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先进治疗药物的管道分析。

A pipeline analysis of advanced therapy medicinal products.

机构信息

NIHR Innovation Observatory at Newcastle University, The Catalyst, 3 Science Square, Newcastle Helix, Newcastle upon Tyne NE4 5TG, UK.

NIHR Innovation Observatory at Newcastle University, The Catalyst, 3 Science Square, Newcastle Helix, Newcastle upon Tyne NE4 5TG, UK.

出版信息

Drug Discov Today. 2023 May;28(5):103549. doi: 10.1016/j.drudis.2023.103549. Epub 2023 Mar 22.

Abstract

Advanced therapy medicinal products (ATMPs) are innovative biological products categorised as either gene, somatic cell, tissue-engineered or combined therapies. As of March 2022, 12 ATMPs are marketed in the EU and UK. We identified 482 unique ATMPs within 616 technology records from the National Institute for Health and Care Research Innovation Observatory database, 4 of which are currently marketed. These 482 ATMPs were identified in 583 clinical trials. Of the 616 records, 57.1% were for gene therapies and 1.6% were for combined therapies. Records covered various indications, including 130 haematological malignancies and 60 genetic disorders. Marketing authorisation intelligence was included in 14% of records.

摘要

先进治疗药物产品(ATMPs)是创新性的生物产品,分为基因、体细胞、组织工程或联合疗法。截至 2022 年 3 月,欧盟和英国市场上有 12 种 ATMP 产品。我们从国家卫生与保健卓越研究所创新观察站数据库中,在 616 个技术记录中发现了 482 种独特的 ATMP,其中 4 种正在市场上销售。这些 ATMP 在 583 项临床试验中被识别出来。在 616 个记录中,57.1%是基因疗法,1.6%是联合疗法。记录涵盖了各种适应症,包括 130 种血液系统恶性肿瘤和 60 种遗传疾病。14%的记录包含上市授权情报。

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