Stable and inhalable powder formulation of mRNA-LNPs using pH-modified spray-freeze drying

Int J Pharm. 2024 Nov 15:665:124632. doi: 10.1016/j.ijpharm.2024.124632. Epub 2024 Sep 7.

Abstract

A powder formulation for mucosal administration of mRNA-encapsulated lipid nanoparticles (mRNA-LNPs) is expected to be useful for respiratory diseases. Although freeze-drying is widely used to obtain solid formulations of mRNA-LNPs, highly hydrosoluble cryoprotectants, such as sucrose are necessary. However, sucrose is not a suitable excipient for inhalation powders because of its hygroscopic and deliquescence properties. Spray freeze-drying (SFD) is a method to produce inhalable powder formulation. In this study, we prepared inhalable powder formulations of mRNA-LNPs without deliquescence excipients using pH-modified SFD, which strengthens the interaction between mRNA and ionizable lipids of LNPs by acidic pH modifier, leading to retention of the encapsulated structure of mRNA-LNPs even after SFD. Powdered mRNA-LNPs were suitable for inhalation, and mRNA was encapsulated in LNPs after SFD. The mRNA encapsulation efficiency and mRNA transfection efficiency of pH-modified SFD-mediated powdered mRNA-LNPs were higher than those of conventional SFD, although they were significantly lower than those of liquid intact mRNA-LNPs. However, after long-term storage, the powdered formulation of the mRNA-LNPs exhibited higher mRNA transfection efficiency than liquid mRNA-LNP. Powdered mRNA-LNPs also exerted their function in air-liquid interface cultivation and in vivo intratracheal administration. Collectively, the powder formulation of mRNA-LNPs especially prepared by SFD is expected to be applied for dry powder inhalers.

Keywords: Lipid nanoparticles; Powder formulation; Spray freeze drying; mRNA.

MeSH terms

  • Administration, Inhalation
  • Animals
  • Chemistry, Pharmaceutical / methods
  • Drug Compounding / methods
  • Drug Stability
  • Excipients / chemistry
  • Freeze Drying*
  • Humans
  • Hydrogen-Ion Concentration
  • Lipids / chemistry
  • Liposomes
  • Nanoparticles* / chemistry
  • Powders*
  • RNA, Messenger* / administration & dosage
  • Spray Drying
  • Transfection / methods

Substances

  • Powders
  • RNA, Messenger
  • Lipid Nanoparticles
  • Lipids
  • Liposomes
  • Excipients