10 results about "Acetylgalactosamine" patented technology

Embodiments of the present application relate to functionalized N-acetylgalactosamine-analogs, methods of making, and uses of the same. In particular, mono or trivalent N-acetylgalactosamine analogs may be prepared by utilizing a wide variety of linkers containing functional groups. These functionalized N-acetylgalactosamine-analogs may be used in the preparation of targeted delivery of oligonucleotide-based therapeutics.

The present disclosure relates to delivery platforms that specifically and efficiently direct multimeric RNAi agent payloads to hepatocytes in a subject, in vivo. The delivery platforms disclosed herein include metabolically stabilized N-Acetylgalactosamine (NAG or GalNAc) targeting ligands conjugated to two or more RNAi agents, to facilitate the delivery of the oligonucleotide-based payloads to cells, including to hepatocytes. Pharmaceutical compositions that include the metabolically stabilized multimeric RNAi agent conjugate delivery platform are also described, as well as methods of use for the treatment of various diseases and disorders where delivery of a therapeutic payload to a hepatocyte is desirable.

Provided herein are compositions and methods for non-parenteral delivery of conjugated oligomeric compounds. In certain embodiments, compositions and methods are provided for oral delivery of conjugated oligomeric compounds. In certain embodiments, the oligomeric compounds are conjugated to one or more N-acetylgalactosamines or N-acetylgalactosamine analogues.

The present disclosure relates to delivery platforms that specifically and efficiently direct stabilized RNAi agent payloads to hepatocytes in a subject, in vivo. The delivery platforms disclosed herein include metabolically stabilized N-Acetylgalactosamine (NAG or GalNAc) targeting ligands conjugated to one or more oligonucleotides through a metabolically stabilized linkage that is more stable than a phosphodiester linkage, to facilitate the delivery of the oligonucleotide-based payloads to cells, including to hepatocytes. Pharmaceutical compositions that include the metabolically stabilized RNAi agent conjugate delivery platform are also described, as well as methods of use for the treatment of various diseases and disorders where delivery of a therapeutic payload to a hepatocyte is desirable.

This invention provides a three-effect valence N This invention relates to acetylgalactosamine cationic liposomes, their preparation methods, and applications, belonging to the field of pharmaceutical technology. The invention prepares trivalent... N Acetylgalactosamine cationic liposome nanoparticles possess advantages such as good structural stability, moderate particle size, narrow particle size distribution, moderate surface charge, and low preparation cost, meeting the requirements for nucleic acid drug delivery carriers. The prepared trivalent... N Acetylgalactosamine cationic liposome nanoparticles can also be used to construct sustained-release drug delivery systems for nucleic acid drugs or as transport carriers for non-nucleic acid drugs.

This invention discloses an antisense oligonucleotide targeting S100A11 and its applications, belonging to the field of biomedicine. Experiments have demonstrated that the antisense oligonucleotide targeting S100A11 provided by this invention effectively and exclusively reduces the expression level of the target protein S100A11, which is of great significance for tumors and liver-related diseases with high S100A11 expression. Subcutaneous injection of an S100A11 ASO linked to N-acetylgalactosamine into the interscapular region effectively and exclusively reduces the expression level of S100A11 in mouse liver tissue; injection of an S100A11 ASO linked to N-acetylgalactosamine into a mouse model of liver cancer significantly reduced total bile acids in the liver and blood of mice, and reduced the number and size of tumors in the liver cancer mice. This invention discovers the value of an ASO targeting S100A11 and reveals its regulation of bile acid-mediated liver cancer, laying a research foundation for the treatment of bile acid-mediated liver cancer.

This disclosure relates to a delivery platform for specifically and efficiently delivering a multimeric RNAi agent payload to target hepatocytes in vivo. The delivery platform disclosed herein comprises a metabolically stable N-acetylgalactosamine (NAG or GalNAc) target ligand conjugated to two or more RNAi agents, and facilitates the delivery of oligonucleotide-based payloads to cells, including hepatocytes. Pharmaceutical compositions comprising the metabolically stable multimeric RNAi agent conjugated delivery platform, as well as methods of use for the treatment of various diseases and disorders in which delivery of a therapeutic payload to hepatocytes is desirable, are also described.

The present invention provides a topical skin preparation composition containing a heparin-like substance that has an excellent effect in improving the skin barrier function. [Solution] A topical skin preparation composition containing a heparin-like substance (A) in which the proportion of disaccharide units consisting of C2-sulfated D-glucuronic acid and C4,C6-disulfated N-acetyl-D-galactosamine is 7 to 22 mol%.

The present disclosure includes methods of treating 05-associated disease or disorder, such as myasthenia gravis, with a combination that includes an antibody or antigen-binding fragment thereof that binds specifically to C5 and a C5 iRNA which is a glycoconjugate that includes a ligand having terminal N-Acetylgalactosamine (GalNAc) residues and / or N-acetylglucosamine (GIcNAc) residues; or with the C5 iRNA monotherapy.