Moderna, Inc., Cambridge, MA 02142, USA.
Bioanalysis. 2024;16(21-22):1141-1154. doi: 10.1080/17576180.2024.2411914. Epub 2024 Oct 18.
Messenger RNA (mRNA)-based therapeutics have emerged as a promising modality for various clinical applications, necessitating robust methods for mRNA quantification. This biodistribution study compares the performance of branched DNA and reverse transcriptase quantitative polymerase chain reaction (RT-qPCR) assays for measuring lipid nanoparticle-encapsulated mRNA. Following intravenous administration of nascent peptide imaging luciferase mRNA (1 mg/kg) to rats, mRNA levels in various tissues and serum were quantified using both assays. Statistical analyses, including Bland-Altman, Deming regression and Passing-Bablok regression, were employed to assess method comparability and reproducibility. The results indicated that mRNA pharmacokinetics measured by branched DNA and RT-qPCR were largely consistent across tissues, with RT-qPCR showing greater reproducibility across multiple laboratories. RT-qPCR also demonstrated a wider dynamic range and higher sensitivity, making it a more versatile option for large-scale studies. Despite some differences in data due to tissue types and timepoints, both methods provided comparable pharmacokinetic profiles for mRNA quantification. This study underscores the importance of selecting an appropriate quantification method based on study requirements and highlights RT-qPCR's adaptability for multisite research, especially for the clinical development of mRNA-based therapeutics.
信使 RNA(mRNA)为基础的疗法已经成为各种临床应用的一种很有前途的方式,这就需要有强大的方法来进行 mRNA 定量。本生物分布研究比较了分枝 DNA 和逆转录定量聚合酶链反应(RT-qPCR)测定法在测量脂质纳米颗粒包封的 mRNA 中的性能。在给大鼠静脉注射新生肽成像荧光素酶 mRNA(1mg/kg)后,使用两种测定法来定量各种组织和血清中的 mRNA 水平。采用 Bland-Altman、Deming 回归和 Passing-Bablok 回归等统计分析来评估方法的可比性和可重复性。结果表明,分枝 DNA 和 RT-qPCR 测定的 mRNA 药代动力学在各组织中基本一致,RT-qPCR 在多个实验室中具有更好的重现性。RT-qPCR 还具有更宽的动态范围和更高的灵敏度,使其成为大规模研究的更通用的选择。尽管由于组织类型和时间点的不同,数据存在一些差异,但两种方法都为 mRNA 定量提供了可比的药代动力学特征。本研究强调了根据研究要求选择适当的定量方法的重要性,并突出了 RT-qPCR 对多地点研究的适应性,特别是对于基于 mRNA 的治疗剂的临床开发。