Abstract: The invention relates to an aqueous suspension formulation for aerosol formation, said suspension formulation comprising lipid or lipidoid nanoparticles which are suspended in an aqueous vehicle solution, wherein the lipid or lipidoid nanoparticles comprise the following components (a) and (b): (a) a nucleic acid and (b) an ionizable lipid or an ionizable lipidoid; and wherein the aqueous vehicle solution comprises a triblock copolymer which contains one poly(propylene oxide) block and two poly(ethylene oxide) blocks. Moreover, the invention relates to an aerosol obtained from the formulation for aerosol formation.

Abstract: The present disclosure provides a pharmaceutical composition comprising a polyribonucleotide for use in treating a ciliopathy in a subject suffering of a ciliopathy, wherein the polyribonucleotide encodes a functional version of a protein a defect of which is associated with said ciliopathy, and wherein administration of said pharmaceutical composition to the respiratory system of said subject is effected when the subject shows an inflammation of the respiratory system. Further, the present disclosure relates to a method for analyzing the effect of a polyribonucleotide on ciliogenesis, wherein said polyribonucleotide encodes a protein involved in and/or required for ciliogenesis.

Abstract: The invention provides a composition which is suitable for the delivery of RNA, which composition comprises (i) particles contained in a liquid phase, wherein the particles comprise RNA and a lipid composition, and (ii) a salt composition. The lipid composition comprises (i-a) a cholesterol derivative, (i-b) a phosphoglyceride, and (i-c) a pegylated phosphoglyceride. Further provided is a method for preparing the composition.

Abstract: The present invention relates to polymers comprising a characteristic combination of alkylene amine moieties which are useful as vehicles for transfecting a cell with a nucleic acid. The present invention furthermore relates to a composition comprising a nucleic acid and such a polymer, and to a method of transfecting a cell using said composition. Furthermore, the present invention relates to pharmaceutical compositions and uses.

Abstract: Provided is a composition comprising (i) a nano- or microparticle formulation of a therapeutically active agent which is suspended in a liquid phase, and (ii) at least one cryoprotective additive selected from C3-C5 alkanes substituted by one or two hydroxy groups which stabilizes the particle formulation. Further aspects relate to a solid composition which can be obtained by freezing the stabilized composition, and to processes for the preparation of the compositions in accordance with the invention.

Abstract: The invention provides a composition which is suitable for the delivery of RNA, which composition comprises (i) particles contained in a liquid phase, wherein the particles comprise RNA and a lipid composition, and (ii) 1,2 propanediol. The lipid composition comprises (i-a) a cholesterol derivative, (i-b) a phosphoglyceride, and (i-c) a pegylated phosphoglyceride. Further provided are a method for preparing the composition, and a solid composition which is obtainable by freezing the composition wherein particles are contained in a liquid phase.

Abstract: The present invention relates to polymers comprising a characteristic combination of alkylene amine moieties which are useful as vehicles for transfecting a cell with a nucleic acid. The present invention furthermore relates to a composition comprising a nucleic acid and such a polymer, and to a method of transfecting a cell using said composition. Furthermore, the present invention relates to pharmaceutical compositions and uses.

Abstract: The present invention relates to a pharmaceutical composition comprising a polyribonucleotide (RNA) and a cationic agent, wherein said pharmaceutical composition is formulated as a solid dosage form for administration to the gastrointestinal (GI) tract. The present invention furthermore relates to the use of such a pharmaceutical composition for systemic delivery of RNA and to a method for systemic delivery of RNA to a subject comprising the step of administering such a pharmaceutical composition to the GI tract. Furthermore, the present invention relates to a kit.

Abstract: The present invention relates to oligomers, polymers and lipidoids comprising characteristic oligo(alkylene amine) moieties which are useful as vehicles for transfecting a cell with RNA. The present invention furthermore relates to a composition comprising at least a nucleic acid and an oligomer or polymer or a lipidoid comprising such oligo(alkylene amine) moieties and to a method of transfecting a cell using said composition. Furthermore, the present invention relates to pharmaceutical compositions and uses.