College of Pharmacy, Gachon Institute of Pharmaceutical Science, Gachon University, Incheon, South Korea.
Drug Deliv. 2022 Dec;29(1):2644-2657. doi: 10.1080/10717544.2022.2108523.
Ionizable LNPs are the latest trend in nucleic acid delivery. Microfluidics technology has recently gained interest owing to its rapid mixing, production of nucleic acid-ionizable LNPs, and stability of nucleic acid inside the body. Industrial scale-up, nucleic acid-lipid long-term storage instability, and high production costs prompted scientists to seek alternate solutions to replace microfluidic technology. We proposed a single-pot, organic solvent-free thermocycling technology to efficiently and economically overcome most of the limitations of microfluidic technology. New thermocycling technology needs optimization of process parameters such as sonication duration, cooling-heating cycle, number of thermal cycles, and lipid:aqueous phase ratio to formulate precisely sized particles, effective nucleic acid encapsulation, and better shelf-life stability. Our research led to the formulation of siRNA-ionizable LNPs with particle sizes of 104.2 ± 34.7 nm and PDI 0.111 ± 0.109, with 83.3 ± 4.1% siRNA encapsulation. Thermocycling siRNA-ionizable LNPs had comparable morphological structures with commercialized microfluidics ionizable LNPs imaged by TEM and cryo-TEM. When compared to microfluidics ionizable LNPs, thermocycling siRNA-ionizable LNPs had a longer shelf life at 4°C. Our thermocycling technology showed an effective alternative to microfluidics technology in the production of nucleic acid-ionizable LNPs to meet global demand.
可离子化 LNPs 是核酸递送的最新趋势。微流控技术由于其快速混合、生产核酸-可离子化 LNPs 以及核酸在体内的稳定性而最近受到关注。工业规模扩大、核酸-脂质长期储存不稳定和高生产成本促使科学家寻求替代方案来取代微流控技术。我们提出了一种单锅、无有机溶剂的热循环技术,以高效且经济地克服微流控技术的大多数限制。新的热循环技术需要优化工艺参数,例如超声持续时间、冷却-加热循环、热循环次数和脂质:水相比例,以精确制定粒径、有效封装核酸并提高储存稳定性。我们的研究导致了 siRNA-可离子化 LNPs 的配方,其粒径为 104.2 ± 34.7nm,PDI 为 0.111 ± 0.109,siRNA 包封率为 83.3 ± 4.1%。热循环 siRNA-可离子化 LNPs 的形态结构与 TEM 和 cryo-TEM 成像的商业化微流控可离子化 LNPs 相当。与微流控可离子化 LNPs 相比,热循环 siRNA-可离子化 LNPs 在 4°C 下具有更长的保质期。我们的热循环技术为生产核酸-可离子化 LNPs 提供了一种有效的替代微流控技术的方法,以满足全球需求。