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Müller, J.T.* ; Kromer, A.P.E.* ; Ezaddoustdar, A.* ; Alexopoulos, I.* ; Steinegger, K.M.* ; Porras-Gonzalez, D.L. ; Berninghausen, O.* ; Beckmann, R.* ; Braubach, P.* ; Burgstaller, G. ; Wygrecka, M.* ; Merkel, O.M.*

Nebulization of RNA-loaded micelle-embedded polyplexes as a potential treatment of idiopathic pulmonary fibrosis.

ACS Appl. Mater. Interfaces 17, 11861-11872 (2025)

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Biodegradable poly(β-amino) esters (PBAEs) have been a focus of interest for delivering therapeutic siRNA for several years. While no approved therapies are on the market yet, our study aims to advance PBAE-based treatments for currently "undruggable" diseases. The PBAEs used in this study are based on a recently reported step-growth copolymerization, which results in polymers with a unique balance of lipophilicity and positive charge, thereby showcasing diverse properties. Upon incubation with siRNA, these PBAEs form a unique structure and topology, which we classify as a subtype of classical polyplex, termed "micelle-embedded polyplexes" (mPolyplexes). The impact of different nebulizers on the physicochemical performance of these nanoparticles was investigated, and it was found that various mPolyplexes can be nebulized using vibrating-mesh nebulizers without the loss of gene silencing activity nor a change in physicochemical properties, setting them apart from other nanoparticles such as marketed LNPs. Finally, their therapeutic application was tested ex vivo in human precision-cut lung slices from patients with lung fibrosis. mPolyplexes mediated 52% gene silencing of matrix metalloprotease 7 (MMP7) and a downstream effect on collagen I (Col I) with 33% downregulation as determined via qPCR.

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