Biontech Se

Johannes Gutenberg-Universität Mainz

Tron—translationale Onkologie an Der Universitátsmedizin Der Johannes Gutenberg—universität Mainz Gemeinnützige Gmbh

Tron-Translationale Onkologie an Der Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz

Biontech Rna Pharmaceuticals Gmbh

Tron—translationale Onkologie an Der Universitatsmedizin Der Johannes Gutenberg-Universitat Mainz Ggmbh

Anne Schlegel

Heinrich Haas

Ugur Sahin

Sebastian Kreiter

Mustafa Diken

Kerstin Walzer

Sebastian Hörner

Annette Vogel

Stephanie Erbar

Thorsten Klamp

Kerstin Reuter

Phillip Heller

Jorge Herrero Moreno

Matthias Barz

Benjamin Weber

Philipp Heller

Sara Nogueira

Jorge Moreno Herrero

Daniel Zucker

The present disclosure relates to RNA particles for delivery of RNA to target tissues after administration, in particular after parenteral administration such as intravenous, intramuscular, subcutaneous or intratumoral administration, and compositions comprising such RNA particles. The RNA particles in one embodiment comprise single-stranded RNA such as mRNA which encodes a peptide or protein of interest, such as a pharmaceutically active peptide or protein. The RNA is taken up by cells of a target tissue and the RNA is translated into the encoded peptide or protein, which may exhibit its physiological activity.

RNA particles comprising polysarcosine

The present invention relates to compositions comprising polyplex formulations for delivery of RNA to a target organ or a target cell after parenteral administration, in particular after intramuscular administration. More precisely, the present invention relates to formulations for administration of RNA such as self-replicating RNA, in particular by intramuscular injection. In more detail, the formulations comprise polyplex particles from single stranded RNA and a polyalkyleneimine.

Formulation for administration of RNA

The present invention relates to compositions comprising polyplex formulations for delivery of RNA to a target organ or a target cell after parenteral administration, in particular after intramuscular administration. More precisely, the present invention relates to formulations for administration of RNA such as self-replicating RNA, in particular by intramuscular injection. In more detail, the formulations comprise polyplex particles from single stranded RNA and a polyalkyleneimine. The RNA may encode a protein of interest, such as a pharmaceutically active protein. Furthermore, the present invention relates to pharmaceutical products, comprising said RNA polyplex formulations for parenteral application to humans or to animals. The present invention relates as well to manufacturing of such pharmaceutical products, comprising, optionally, steps of sterile filtration, freezing and dehydration.

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Düsseldorf, Germany

Anne Schlegel