Advances in Gene Therapy: Lipid Nanoparticles Show Promising Results

Strategic Insights -

Researchers at Sartorius BIA Separations d.o.o. in Ajdovscina, Slovenia, have made significant breakthroughs in the development of gene therapy using lipid nanoparticles (LNPs). According to a new report, LNPs have emerged as the most advanced drug delivery system for gene therapies and vaccines due to their ability to encapsulate a diverse range of payloads, including nucleic acids and proteins.

Key Takeaways:

  • The research focuses on the development of a two-dimensional chromatography (2D) method to analyze crucial parameters of LNP formulations, including encapsulation efficiency, payload integrity, and size distribution.
  • The proposed method employs a switching chromatography system comprising two distinct analytical columns, independent pumps, and two detectors, including an ultraviolet-visible detector (UV-Vis) and a multi-angle light scattering detector (MALS).
  • The results demonstrate that the proposed method provides a comprehensive and robust analytical solution, enabling further development and understanding of lipid-based drug delivery systems.
  • The study highlights the sensitivity, linearity, and precision of the technique, with a limit of detection (LOD) of 15 ng mRNA, limit of quantification (LOQ) of 45 ng mRNA, and relative standard deviation (RSD) of less than 5%.
  • The authors conclude that the validation of the technique confirms its suitability for the analysis of LNP formulations.

The research team has been led by Nejc Pavlin from Sartorius BIA Separations d.o.o. The study has been published in the Journal of Chromatography B-analytical Technologies In the Biomedical and Life Sciences.

Statistics:

  • LOD of 15 ng mRNA
  • LOQ of 45 ng mRNA
  • RSD of less than 5%
  • Number of authors: 7
  • Journal: Journal of Chromatography B-analytical Technologies In the Biomedical and Life Sciences, 2025;1265:124751

Sources:

  • NewsRx. New Gene Therapy Study Results Reported from Nejc Pavlin et al (Analysis of lipid nanoparticles using two-dimensional chromatography: Simultaneous determination of encapsulation efficiency, nucleic acid integrity, and size of LNP formulations). Vaccine Weekly. August 27, 2025; p 123.
  • Nejc Pavlin, Mojca Bavcar, Tristan Kovacic, Tjasa Kascek, Anze Martincic Celjar, Ines Bergoc, Andreja Gramc Livk, and Ales Strancar. Analysis of lipid nanoparticles using two-dimensional chromatography: Simultaneous determination of encapsulation efficiency, nucleic acid integrity, and size of LNP formulations. Journal of Chromatography B-analytical Technologies In the Biomedical and Life Sciences, 2025;1265:124751.