The present invention relates to an orally-administered gene carrier and a use thereof, and more specifically, to: an oral gene carrier comprising, at the C-terminus of an immunoglobulin Fc region, a linker formed from cationic arginine and enabling the condensation of an anionic gene; and an oral composition for preventing, ameliorating or treating metabolic diseases, the composition comprising the gene carrier and the GLP-1 gene as active ingredients. The gene carrier, according to the present invention, may be usefully employed as an orally-administered carrier for various genes, and especially, is expected to be usable for preventing, ameliorating or treating metabolic diseases, such as diabetes and obesity, by effectively transferring the GLP-1 gene.

Certain exemplary embodiments are directed to a biologically active composition of matter (and uses thereof) configured for targeted delivery of biotin to mitochondria, the composition comprising a first D-biotin conjugated to a water-soluble, cell-permeable, peptide sequence, wherein the peptide sequence is selected from a polypeptide group with an alternating aromatic-cationic motif.

The present disclosure includes the use of a miRNA inhibitor for treating a tauopathy associated with a decreased level of SIRT1 protein or SIRT1 gene expression, PGC-1α protein and/or PGC-α gene expression, and/or CD36 and/or CD36 gene expression.

Methods are provided for improving the manufacture and use of pharmaceutical compositions comprising histidine-lysine copolymers and nucleic acids, which spontaneously form nanoparticles when mixed. The flow rate of mixing and the ratio of copolymer to siRNA strongly affect nanoparticle properties, including size and homogeneity of particles, resulting in greater efficacy in delivery to target cells. Further, an acidic pH of the siRNA solution, as well as the addition of acetate or phosphate salt to the histidine-lysine copolymer prior to mixing with the siRNA also contribute to lower nanoparticle diameters and more uniform particles (lower PDI).

The invention provides compositions and methods for the cytoplasmic delivery of antibodies and other proteins. Specifically, provided herein are compositions having an anionic polypeptide and a cationic transfection agent for facilitating the cytoplasmic delivery of an antibody or a protein.

The present invention relates to polymeric nanoparticles comprising a cytokine or a nucleic acid encoding for a cytokine, pharmaceutical compositions comprising the same, and methods for treating certain diseases comprising administering these polymeric nanoparticles to a subject in need thereof.

The present invention mainly relates to a polymer for delivery of biologically active materials, a complex and a method of synthesis thereof. The polymer comprises a poly(ethylene imine) and at least one monomer, each monomer comprising a modified sugar moiety, preferably galactose, comprising a sulphur atom or a nitrogen atom and a chemical moiety comprising a terminal epoxide for linking the polyethylene imine to the monomer, wherein the sulphur atom or the nitrogen atom links the modified sugar moiety to the chemical moiety. The biologically active material is preferably a gene, siRNA, mRNA or plasmid DNA. Further disclosed is the medical use of said complex in treating a disease caused by a genetic disorder, for example cancer.

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.

The present invention provides, among other things, methods and compositions of formulating nucleic acid-containing nanoparticles for efficient delivery of payload in vivo such that the method and compositions can be used to generate mRNA vaccines.

The present invention relates to a conjugate for delivery of biomolecules using a cationic macromolecule as a linker between the biomolecule and biomarker targeting moiety, and the process of making the conjugate.