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.

The present disclosure relates to double stranded RNA agents targeting the hepatitis B virus (HBV) genome, and methods of using such agents to inhibit expression of one or more HBV genes and methods of treating subjects having an HBV infection or HBV-associated disorder, e.g., chronic hepatitis B infection.

The invention relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting the SCAP gene, as well as methods of inhibiting expression of a SCAP gene and methods of treating subjects having a SCAP-associated disorder, such as nonalcoholic fatty liver disease (NAFLD) or nonalcoholic steatohepatitis (NASH), using such dsRNAi agents and compositions.

The present disclosure relates to the use of inositol-stabilized arginine silicate complexes (“ASI”) with the addition of free inositol (“I”) to form a composition ASI+I for improving cognitive functioning in humans, particularly video game players.

Amphiphilic oligonucleotide conjugates that enhance adjuvant function are disclosed. The conjugates typically include: a lipophilic component, and conjugated thereto (directly or indirectly) an immunomodulating oligonucleotide that, if it were not conjugated to the lipophilic component, would suppress TLR7 and/or TLR8 stimulation. In the presence of albumin, these conjugates significantly enhance adjuvant function, in particular the function of TLR7/8-mediated adjuvants such as an imidazoquinolinamine. The conjugates can be administered, along with an adjuvant compound, to a subject in order to cause and/or enhance an immune response (for instance, to an infectious agent or a cancer antigen) in the subject.

Compounds and compositions that have an asialoglycoprotein receptor (ASGPR) binding ligand bound to an extracellular protein binding ligand for the selective degradation of the target extracellular protein in vivo to treat disorders mediated by the extracellular protein are described.

RNAs, such as miRNA and siRNA, and their use in treating complement-mediated disorders, are described.

Antibody conjugates with camptothecin compounds are described, with methods of use and preparations.

Described herein is a bi-functional compound for removing macrophage migration inhibitory factor (MIF) or immunoglobin G (IgG). Further described herein is a pharmaceutical composition which comprise these bi-functional compounds. Further described herein is a method for treating disease states and/or conditions with the compounds or the composition. The disease states and/or conditions are mediated through MIF/IgG or where MIF/IgG is a contributing factor to the development and perpetuation of diseases and/or conditions, such as autoimmune diseases and cancer, among others.

Compositions and methods of use thereof for delivering nucleic acid cargo into cells are provided. The compositions typically include (a) a 3E10 monoclonal antibody or an antigen binding, cell-penetrating fragment thereof; a monovalent, divalent, or multivalent single chain variable fragment (scFv); or a diabody; or humanized form or variant thereof, and (b) a nucleic acid cargo including, for example, a nucleic acid encoding a polypeptide, a functional nucleic acid, a nucleic acid encoding a functional nucleic acid, or a combination thereof. Elements (a) and (b) are typically non-covalently linked to form a complex.