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 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 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 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 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: Chemically synthesized RNA molecules (useful for expression of a coding sequence have the general structure 5?-W-X-Y-(coding sequence)-Z-3? wherein W is selected from the group consisting of a 5?-Cap, a free 5?-triphosphate group, a free 5?-disphosphate group, a free 5?-monophosphate group, a free 5?—OH group and chemically modified analogues of said 5?-Cap, said 5?-triphosphate group, said free 5?-disphosphate group or said free 5?-monophosphate group, X is an optional 5?UTR sequence, Y is an optional start codon, and Z is directly linked to the coding sequence and is selected from the group consisting of a free 3?—OH group, a stop codon and a stop codon linked, optionally via a 3?UTR sequence, to a poly(A) tail.

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, auto-immune 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 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 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 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 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 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: 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 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 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 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 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.