摘要:
The present invention is directed to an inventive polymeric carrier molecule according to generic formula (I) and variations thereof, which allows for efficient transfection of nucleic acids into cells in vivo and in vitro, a polymeric carrier cargo complex formed by a nucleic acid and the inventive polymeric carrier molecule, but also to methods of preparation of this inventive polymeric carrier molecule and of the inventive polymeric carrier cargo complex. The present invention also provides methods of application and use of this inventive polymeric carrier molecule and the inventive polymeric carrier cargo complex as a medicament, for the treatment of various diseases, and in the preparation of a pharmaceutical composition for the treatment of such diseases.
摘要:
The present invention relates to nanoparticles comprising nucleic acids coated with a (biodegradable) polymer for reversible immobilization and/or controlled release of the nucleic acid comprising nanoparticles. Furthermore, the present invention is directed to medical or diagnostic devices, particularly stents and implants coated by a (biodegradable) polymer with the nucleic acid comprising nanoparticles for reversible immobilization and/or controlled release. Furthermore, the present invention is directed to the use of these nanoparticles coated with a (biodegradable) polymer and to the use of medical devices and implants coated by the (biodegradable) polymer with these nucleic acid comprising nanoparticles in the prophylactic or therapeutic treatment of diseases, particularly in the prevention or treatment of restenosis, calicification, foreign body reaction, or inflammation. Additionally, the present invention is directed to a method of preparing these nucleic acid comprising nanoparticles coated with a (biodegradable) polymer and to a method for coating nucleic acid comprising nanoparticles by a (biodegradable) polymer on medical or diagnostic devices.
摘要:
The invention relates to an artificial nucleic acid molecule comprising at least one open reading frame and at least one 3'UTR element comprising a nucleic acid sequence which is derived from the 3'UTR of an albumin gene or from a variant of the 3'UTR of an albumin gene. The invention further relates to the use of such an artificial nucleic acid molecule in gene therapy and/or genetic vaccination. Furthermore, the invention relates to the use of a 3'UTR element comprising a nucleic acid sequence which is derived from the 3'UTR of an albumin gene or from a variant of the 3'UTR of an albumin gene for the stabilization and/or prolongation of protein expression from a nucleic acid sequence comprising such 3'UTR element.
摘要:
The present invention is directed to a pharmaceutical composition including (e.g. for use as an adjuvant) a polymeric carrier cargo complex, comprising as a carrier a polymeric carrier formed by disulfide-crosslinked cationic components; and as a cargo at least one nucleic acid molecule, and at least one antigen that is selected from an antigen from a pathogen associated with infectious disease; an antigen associated with allergy or allergic disease; an antigen associated with autoimmune disease; or an antigen associated with a cancer or tumour disease, or in each case a fragment, variant and/or derivative of said antigen. The pharmaceutical composition allows for efficient induction of an adaptive immune response directed against said antigen. The present invention furthermore provides kits, as well as the use of the pharmaceutical composition or the kit as a vaccine, particularly in the treatment of infectious diseases, allergies, autoimmune diseases and tumour or cancer diseases.
摘要:
The present invention relates to vaccines comprising at least one mRNA encoding at least one antigen for use in the treatment of a disease in an elderly patient preferably exhibiting an age of at least 50 years, more preferably of at least 55 years, 60 years, 65 years, 70 years, or older, wherein the treatment comprises vaccination of the patient and eliciting an immune response in said patient. The present invention is furthermore directed to kits and kits of parts comprising such a vaccine and/or its components and to methods applying such a vaccine or kit.
摘要:
The present invention is directed to a composition or vaccine composition comprising a) an adjuvant component comprising or consisting of at least one immunostimulatory nucleic acid sequence, complexed with a complexing agent; b) an antigen, preferably a protein or peptide antigen and/or a nucleic acid sequence encoding said antigen; and c) a carrier molecule for combined packaging the adjuvant component and the antigen. The present invention is also directed to the first medical use of such a composition or vaccine composition and to the second medical use of such a composition or vaccine composition or components thereof for the treatment of diseases, such as infectious or cancer or tumour diseases as defined herein. The present invention furthermore discloses kits comprising such a composition or vaccine composition.
摘要:
The present invention relates to an immunostimulatory composition comprising a) an adjuvant component, comprising or consisting of at least one (m)RNA, complexed with a cationic or polycationic compound, and b) at least one free mRNA, encoding at least one therapeutically active protein, antigen, allergen and/or antibody, wherein the immunostimulatory composition is capable to elicit or enhance an innate and optionally an adaptive immune response in a mammal. The inventive immunostimulatory composition may be a pharmaceutical composition or a vaccine. The invention furthermore relates to a method of preparation of the inventive immunostimulatory composition. The invention also relates to the use of the inventive immunostimulatory composition or its components (for the preparation of a pharmaceutical composition or a vaccine) for the treatment of various diseases. Finally, the invention relates to kits containing the inventive immunostimulatory composition, its components and/or the pharmaceutical composition or vaccine.
摘要:
Die vorliegende Erfindung betrifft eine pharmazeutische Zusammensetzung, die Blutzellen bzw. hämatopoietische Zellen, bspw. rote Blutzellen (Erythrocyten), Granulocyten, mononucleäre Zellen (PBMCs) und/oder Blutplättchen, in Verbindung mit einem pharmazeutisch verträglichen Träger und/oder Vehikel enthält, wobei die Zellen mit mindestens einer mRNA transfiziert sind, die mindestens einen für mindestens ein Antigen codierenden Bereich umfaßt Weiterhin wird erfindungsgemäß ein Verfahren zur Herstellung der vorgenannten pharmazeutischen Zusammensetzung sowie die Verwendung derartig transfizierter Blutzellen zur Herstellung von Arzneimitteln bzw. pharmazeutischen Zusammensetzungen zur Immunstimulation gegen die von der mRNA codierten Antigene offenbart. Die erfindungsgemäßen Gegenstände dienen insbesondere der Therapie von und/oder Prophylaxe gegen Krebs oder Infektionskrankheiten und sind darüber hinaus in der Gentherapie einsetzbar.
摘要:
Die vorliegende Erfindung betrifft eine pharmazeutische Zusammensetzung, die Blutzellen bzw. hämatopoietische Zellen, bspw. rote Blutzellen (Erythrocyten), Granulocyten, mononucleäre Zellen (PBMCs) und/oder Blutplättchen, in Verbindung mit einem pharmazeutisch verträglichen Träger und/oder Vehikel enthält, wobei die Zellen mit mindestens einer mRNA transfiziert sind, die mindestens einen für mindestens ein Antigen codierenden Bereich umfaßt. Weiterhin wird erfindungsgemäß ein Verfahren zur Herstellung der vorgenannten pharmazeutischen Zusammensetzung sowie die Verwendung derartig transfizierter Blutzellen zur Herstellung von Arzneimitteln bzw. pharmazeutischen Zusammensetzungen zur Immunstimulation gegen die von der mRNA codierten Antigene offenbart. Die erfindungsgemäßen Gegenstände dienen insbesondere der Therapie von und/oder Prophylaxe gegen Krebs oder Infektionskrankheiten und sind darüber hinaus in der Gentherapie einsetzbar.