用于细胞治疗的临床级晶状病毒载体的长期稳定性
Julie K Jadlowsky1, Rachel Leskowitz1, Stephen McKenna1
1Center for Cellular Immunotherapies, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA 19104, USA.
Molecular therapy. Methods & clinical development
|January 29, 2024
概括
临床级的lentiviral载体在细胞和基因疗法制造中表现出极好的长期稳定性和有效性. 这项研究证实它们的耐用性长达八年,支持研究治疗的延长保质期.
科学领域:
- 生物技术是生物技术.
- 细胞和基因疗法细胞和基因疗法
- 病毒载体制造业 病毒载体制造业
背景情况:
- 病毒载体对于细胞和基因疗法至关重要,临床应用越来越多.
- 现有的数据支持临床级别的隐形病毒载体的安全性和实用性.
- 关于这些载体的长期稳定性的全面研究是有限的,尽管有很大的生产成本和制造约束.
研究的目的:
- 彻底检查临床级别的隐形病毒载体的长期稳定性.
- 评估关键属性,如向量标位,转导效率和长时间存储期间的效力.
- 提供数据支持在ex vivo细胞疗法中使用的lentiviral载体的延长保质期要求.
主要方法:
- 分析了为16项临床试验制造的13个临床级别的隐形病毒载体批量.
- 在制造后至少36个月评估载体稳定性属性.
- 对在-80°C下存储长达8年的载体进行评估.
主要成果:
- 临床级的晶状病毒载体在长时间的储存期间保持了基本的稳定性属性.
- 矢量标位,传导效率和功效在制造后的8年内保持稳健.
- 这些发现强调了这些载体在细胞治疗应用中的可靠性.
结论:
- 良好的GMP等级的lentiviral载体具有显著的长期耐用性和有效性.
- 延长保质期是可行的,这证明了对用于细胞疗法的大规模载体生产的投资是合理的.
- 这些结果支持在ex vivo细胞制造中继续和扩大使用lentiviral载体.
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