相关实验视频

Updated: Jul 20, 2025

A GMP-Compliant Procedure for the Generation of Gene-Modified T cells

A GMP-Compliant Procedure for the Generation of Gene-Modified T cells

Published on: October 6, 2023

细胞和基因治疗产品的国际协调

Judith Arcidiacono¹

¹U.S. Food and Drug Administration, Center for Biologics Evaluation and Research, Silver Spring, MD, USA. Judith.Arcidiacono@fda.hhs.gov.

Advances in experimental medicine and biology

|August 1, 2023

概括

国际监管计划旨在通过促进合作和协调要求,改善全球获得细胞和基因疗法的机会. 这些倡议支持低收入和中等收入国家的监管能力建设.

科学领域:

生物技术是生物技术.
监管科学监管科学
药品制造业药品制造业药品制造业药品制造业

背景情况:

细胞和基因疗法等先进疗法的全球可用性有限.
监管监督能力在全球范围内有很大差异,特别是在低收入和中等收入国家.
监管要求的协调对于国际产品访问至关重要.

研究的目的:

提供国际监管计划的概述,以提高细胞和基因疗法产品的可用性.
突出支持监管能力建设和协调的倡议.
讨论促进全球监管合作的具体计划.

主要方法:

审查国际监管计划和倡议.
国际制药监管机构计划 (IPRP) 细胞治疗工作组 (CTWG) 和基因治疗工作组 (GTWG) 的概述.
讨论国际协调会议 (ICH) 和亚太经济合作组织 (APEC) 监管协调指导委员会 (RHSC).
探索"讨论集群"和并行科学咨询 (PSA) 计划.

主要成果:

现有多个国际计划,以促进细胞和基因疗法的监管对话和协调.
像IRPP,ICH和APEC RHSC这样的倡议是监管合作的关键平台.
讨论集群和PSA等有针对性的计划为合作的科学建议提供了具体的途径.

结论:

监管机构之间的国际合作对于增加全球获取细胞和基因疗法的必要.
协调努力和能力建设计划对于这些先进疗法的公平可用性至关重要.
继续参与国际监管计划将支持开发和获得拯救生命的治疗方法.

关键词:

先进的治疗方法先进的治疗方法.先进疗法的药物,先进疗法的药物.细胞疗法是一种细胞疗法.基因治疗是一种基因疗法.监管趋同监管趋同监管趋同监管协调监管协调监管协调监管协调监管协调

更多相关视频

Author Spotlight: Advancements in CAR-T Cell Manufacturing and Gene Therapy Production

Author Spotlight: Advancements in CAR-T Cell Manufacturing and Gene Therapy Production

Published on: August 18, 2023

Clinical Application of Sleeping Beauty and Artificial Antigen Presenting Cells to Genetically Modify T Cells from Peripheral and Umbilical Cord Blood

Clinical Application of Sleeping Beauty and Artificial Antigen Presenting Cells to Genetically Modify T Cells from Peripheral and Umbilical Cord Blood

Published on: February 1, 2013

相关概念视频

Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

Cell Lines

Cell Lines

A cell line is a population of cells grown in vitro that can be subcultured over several generations. Normal cells cease to divide after a certain number of cell divisions, a process known as replicative senescence. This number, called the Hayflick limit, was conceptualized by Leonard Hayflick in 1961 when he observed that fetal cells grown in culture could only divide 40-60 times. This limit is due to the shortening of the telomeres during each round of cell division, preventing cell division...

iPS Cell Differentiation

iPS Cell Differentiation

The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.