CAR-T疗法是当今非常受关注的免疫治疗之一,也是近十年来免疫医学的重大突破。它是通过对患者自身的免疫细胞T进行基因改造,从而使被改造过的T淋巴细胞能够精准对抗患者体内癌细胞的一种细胞免疫治疗。 CAR-T细胞是经过基因工程改造以表达靶向特定抗原的嵌...
1. Human Gene Therapy Products Incorporating Human Genome Editing, Guidance for Industry. Retrieved January 29, 2024, from https://www.fda.gov/media/156894/download 2. Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products Guidance for Industry. Retrieved January 29,...
值得注意的是,我们建议CAR-T细胞的鉴定,包括一个分析方法来测量转基因的存在(例如,流式细胞术检测CAR表达,PCR检测CAR基因)和一个分析最终产品特定细胞组成的方法(例如,细胞表面标记物),这在GT CMC指南V.B.5.b ii节中也进行了...
CAR-T 细胞产品是人类基因治疗产品,其中 T 细胞特异性经过基因改造,能够识别用于治疗目的所需的靶抗原。该指南草案中,FDA 提供了关于化学、生产和控制(CMC)、药理学和毒理学以及临床研究设计的 CAR-T 细胞具体建议。另外,指南还针对同种异体 CAR-T 细胞产品提出了额外建议。指南还为 CAR-T 细胞产品的分析可比性...
[3] FDA finalizes CAR-T and gene therapy guidance, offers support for accelerated approvals. Retrieved January 29, 2024, from https://endpts.com/fda-finalizes-car-t-and-gene-therapy-guidance-offers-support-for-accelerated-approvals/
as described in section V.B of this guidance. Many antigen recognition domains are derived from murine monoclonal antibodies that may be immunogenic in humans, leading to rejection of the CAR T cells or other safety risks (e.g., anaphylaxis). If approaches to reduce immunogenicity (e.g., “...
FDA于2020年1月发布的行业指南《Chemistry, Manufacturing, and 56 Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications 57 (INDs): Guidance for Industry》(以下简称 “GT CMC指南”)描述了GT产品生产和检测的一般注意事项。
CAR-T细胞的分析测试通常需要复杂的检测方法和开发特定产品的生物检测方法。因此,FDA建议申报者在CAR-T细胞开发的早期阶段就开始进行检测开发,并使用各种检测方法来表征其产品,如图像分析技术、流式细胞术、代谢通量分析等。 另外,申报者还应在制造过程和终产品中控制CAR-T产品的转导效率、表达载体的拷贝数、CAR表达水平...
[2]. FDA Guidance for Industry: Considerations for the Development of Chimeric Antigen Receptor (CAR) T Cell Products. March 2022. [3]. FDA Guidance for Industry: Human Gene Therapy for Rare Diseases. January 2020. [4]. FDA Guidance for Industry: Human Gene Therapy for Retinal Disorders. ...
as described in section V.B of this guidance. Many antigen recognition domains are derived from murine monoclonal antibodies that may be immunogenic in humans, leading to rejection of the CAR T cells or other safety risks (e.g., anaphylaxis). If approaches to reduce immunogenicity (e.g., “...