参考资料: [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...
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...
The FDA has approved an individual patient IND, allowing for a second dose of a novel FSHR-targeting CAR T-cell therapy for the treatment of a patient with recurrent ovarian cancer that may be demonstrating clinical activity to the initial treatment.1 The CAR T-cell therapy is currently unde...
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,...
细胞与基因治疗(Cellular and Gene Therapy,CGT)产品由于其自身特点与复杂性,相较于小分子化学药或其他生物制品,其生产过程也更复杂。随着FDA批准上市的CAR-T细胞治疗产品数量的增加,越来越多的新药研发公司开展细胞与基因治疗产品的研发。细胞与基因治疗产品由于其自身特点与复杂性,相较于小分子化学药或其他生物制品,...
1. Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry,Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs); dated April 2008 2. Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene ...
在这份最新发布的指南中,FDA明确要求在CAR-T产品生产中使用的载体需要符合GMP要求,并会在上市前检查中对这些载体供应商进行检查。 • Frequently Asked Questions - Cell and Gene Therapy Products; Draft Guidance for Industry 指南题目:细胞和...
2022年3月我国BCMA CAR-T产品西达基奥仑赛(cilta-cel)的生物制品许可申请(BLA)获FDA批准,用于治疗成人复发/难治性多发性骨髓瘤(r/r MM)。国产细胞治疗产品CAR-T成功在美国上市标志着国产新药继PD-1后再次出海成功,也使我国本土企业备受鼓舞,将对细胞疗法的开发和关注度推向了更高。然而,一款全新疗法的细胞治疗...
1. Guidance for FDA Reviewers and Sponsors: Content and Review of Chemistry,Manufacturing, and Control (CMC) Information for Human Somatic Cell Therapy Investigational New Drug Applications (INDs); dated April 2008 2. Guidance for Industry: Preclinical Assessment of Investigational Cellular and Gene Th...
这些CMC风险控制点在FDA于2024年1月正式发布的《嵌合抗原受体(CAR)T细胞治疗的研发考量》【Considerations for the Development of Chimeric Antigen Receptor(CAR) T Cell Products Guidance for Industry】指南中被重点提及。 杜新强调,这是FDA专门针对CAR-T产品的指南,尤其对CMC的要求占有很大篇幅,直接反映出CMC对CAR...