Abstract: The present invention relates to a pharmaceutical composition comprising a modified mRNA that is stabilised by sequence modifications and optimised for translation. The pharmaceutical composition according to the invention is particularly well suited for use as an inoculating agent, as well as a therapeutic agent for tissue regeneration. In addition, a process is described for determining sequence modifications that promote stabilisation and translational efficiency of modified mRNA of the invention.

Abstract: The present application describes an antibody-coding, non-modified or modified RNA and the use thereof for expression of this antibody, for the preparation of a pharmaceutical composition, in particular a passive vaccine, for treatment of tumours and cancer diseases, cardiovascular diseases, infectious diseases, autoimmune diseases, virus diseases and monogenetic diseases, e.g. also in gene therapy. The present invention furthermore describes an in vitro transcription method, in vitro methods for expression of this antibody using the RNA according to the invention and an in vivo method.

Abstract: The present invention relates to a pharmaceutical composition comprising a modified mRNA that is stabilised by sequence modifications and optimised for translation. The pharmaceutical composition according to the invention is particularly well suited for use as an inoculating agent, as well as a therapeutic agent for tissue regeneration. In addition, a process is described for determining sequence modifications that promote stabilisation and translational efficiency of modified mRNA of the invention.

Abstract: The present invention relates to a pharmaceutical composition comprising a modified mRNA that is stabilized by sequence modifications and optimized for translation. The pharmaceutical composition according to the invention is particularly well suited for use as an inoculating agent, as well as a therapeutic agent for tissue regeneration. In addition, a process is described for determining sequence modifications that promote stabilization and translational efficiency of modified mRNA of the invention.

Abstract: The present invention relates to chimeric particles comprising single stranded RNA (ssRNA), double stranded RNA (dsRNA) and at least one cationic agent, a pharmaceutical composition containing said particles and to a method of producing the same. The particles of the present invention are particularly useful as an immunostimulating medicament with a superlative pattern of immunostimulation.

Abstract: The present invention relates to a method for inducing an immune response manifested by type I interferon production in a synergistic manner comprising the sequential administration of danger signals within a certain timeframe and means for practicing the method. The present invention is particularly useful for immunomodulation, immunotherapy and vaccination.

Abstract: A composition including an immunostimulating RNA molecule in a dication containing solution wherein the RNA includes a chemical modification which is toxic to cancer or tumor cells. Pharmaceutical compositions incorporate the immunostimulating RNA with tumor cytotoxicity. Methods for treating cancer and tumors use the solution of the immunostimulating RNA with tumor cytotoxicity.

Abstract: The present invention relates to particles comprising RNA, in which the RNA is associated with a cationic polymer or lipid or with both a cationic polymer and lipid, wherein the RNA comprises a cytotoxic nucleotide or cytotoxic nucleotide analog and/or the RNA is covalently attached to a cytotoxin, pharmaceutical compositions containing said particles and pharmaceutical uses of the particles and pharmaceutical compositions. The particles of the present invention are particularly useful as an immunostimulating medicament capable to block proliferation or induce death in dividing cells such as tumor cells.

Abstract: The present invention relates to a method for producing RNA. In particular, the present invention relates to a method for producing RNA, which is scalable and provides RNA at a high purity. The present invention provides a method for producing RNA under GMP and/or cGMP-compliant conditions. The invention further provides specific processes for use as a quality control in the manufacturing of a template DNA and/or in a method for producing RNA, in particular by in vitro transcription.

Abstract: The invention relates to the use of RNA and an aqueous injection buffer containing a sodium salt, a calcium salt and optionally a potassium salt and optionally lactate, in the preparation of a RNA injection solution for increasing RNA transfer and/or RNA translation into/in a host organism. The invention relates further to a RNA injection solution and to a method for increasing the RNA transfer and/or RNA translation of RNA in vivo and in vitro.

Abstract: The present invention relates to a method for producing RNA. In particular, the present invention relates to a method for producing RNA, which is scalable and provides RNA at a high purity. The present invention provides a method for producing RNA under GMP and/or cGMP-compliant conditions. The invention further provides specific processes for use as a quality control in the manufacturing of a template DNA and/or in a method for producing RNA, in particular by in vitro transcription.

Abstract: A biologically active RNA-alkali metal-dication formulation, a pharmaceutical composition containing the complexes, and methods of producing the same. The formulation is particularly useful to introduce RNA and an attached cargo into cells allowing its biological intracellular activities: e.g. immunostimulation (immunomodulation), RNA interference or gene expression.

Abstract: A biologically active RNA-alkali metal-dication formulation, a pharmaceutical composition containing the complexes, and methods of producing the same. The formulation is particularly useful to introduce RNA and an attached cargo into cells allowing its biological intracellular activities: e.g. immunostimulation (immunomodulation), RNA interference or gene expression.

Abstract: The present invention relates to chimeric particles comprising single stranded RNA (ssRNA), double stranded RNA (dsRNA) and at least one cationic agent, a pharmaceutical composition containing said particles and to a method of producing the same. The particles of the present invention are particularly useful as an immunostimulating medicament with a superlative pattern of immunostimulation.

Abstract: The present invention relates to an immunostimulating agent comprising at least one chemically modified RNA. The invention furthermore relates to a vaccine which comprises at least one antigen in combination with the immunostimulating agent. The immunostimulating agent according to the invention and the vaccine according to the invention are employed in particular against infectious disease or cancer diseases.

Abstract: The present invention relates to particles comprising protamine, RNA and at least one endosome destabilizing agent, to methods of their production and to pharmaceutical compositions or kits containing the particles. It further relates to particles comprising protamine and RNA for use in methods of treatment or prevention of diseases and to kits comprising such particles together with at least one endosome destabilizing agent.

Abstract: The present invention relates to a pharmaceutical composition comprising at least one mRNA comprising at least one coding region for at least one antigen from a tumor, in combination with an aqueous solvent and preferably a cytokine, e.g. GM-CSF, and a process for the preparation of the pharmaceutical composition. The pharmaceutical composition according to the invention is used in particular for therapy and/or prophylaxis against cancer.

Abstract: The present invention relates to stable low-salt aqueous formulations of particles comprising a polycation, preferably protamine, and RNA, which formulations are preferably isotonic, to pharmaceutical compositions or kits comprising such formulations and to their use in medicine. It further relates to methods for preparing the formulations of the invention and to methods for physically stabilizing particles comprising a polycation, preferably protamine, and RNA.

Abstract: The present invention relates to chimeric particles comprising single stranded RNA (ssRNA), double stranded RNA (dsRNA) and at least one cationic agent, a pharmaceutical composition containing said particles and to a method of producing the same. The particles of the present invention are particularly useful as an immunostimulating medicament with a superlative pattern of immunostimulation.

Abstract: The present invention relates to a method for immunostimulation in a mammal which comprises a. administration of at least one mRNA containing a region which codes for at least one antigen of a pathogen or at least one tumour antigen, and b. administration of at least one cytokine, at least one cytokine mRNA, at least one CpG DNA or at least one adjuvant RNA. The invention likewise relates to a product and a kit comprising the mRNA and cytokine or cytokine mRNA or CpG DNA or adjuvant RNA of the invention.