A61K47/554

LEUCINE-RICH REPEAT KINASE 2 (LRRK2) iRNA AGENT COMPOSITIONS AND METHODS OF USE THEREOF

The disclosure relates to double stranded ribonucleic acid (dsRNAi) agents and compositions targeting a leucine-rich repeat kinase 2 (LRRK2) gene, as well as methods of inhibiting expression of a LRRK2 gene and methods of treating subjects having a LRRK2-associated disease or disorder, e.g., Parkinson's disease, using such dsRNAi agents and compositions.

Tripartite Modulators of Endosomal G Protein-Coupled Receptors

The present invention relates to tripartite compounds comprising a modulator moiety for endosomal G protein-coupled receptors like neurokinin-1 receptor, a linker and a lipid anchor suitable for anchoring the tripartite compound into a plasma membrane. The present invention also relates to a prodrug and a pharmaceutical composition comprising the tripartite compound and the use of the tripartite compound for the treatment of a disease or disorder mediated by endosomal G protein-coupled receptors signalling like NK.sub.1R signalling.

ANTI-CANCER NUCLEAR HORMONE RECEPTOR-TARGETING COMPOUNDS

The disclosure relates to anti-cancer compounds derived from nuclear steroid receptor binders, to products containing the same, as well as to methods of their use and preparation.

NANOPARTICLES COMPRISING DRUG DIMERS, AND USE THEREOF
20230120021 · 2023-04-20 ·

The present invention relates to a nanoparticle comprising a drug dimer and a use thereof. The nanoparticle comprising a drug dimer of the present invention can increase the drug content and improve the dispersibility of the drug. In addition, the nanoparticle has increased targeting efficiency. Therefore, an effective pharmacological effect can be obtained by using a drug in a less amount, and thus the nanoparticle has excellent commercial applicability.

Compound for treating clostridium difficile

The invention relates to compounds, compositions and polymers comprising a first component adapted to promote germination of Clostridium difficile (C. diff) and a second component which acts as an antimicrobial agent. Said compounds, compositions and polymers are useful for destroying C. diff where conventional antimicrobial agents are unsuccessful. The compositions can be formulated as coating or materials which actively destroy C. diff which come into contact with it. The germination promotion is induced by bile salts. The invention also relates to the use of such materials as a treatment for C. diff associated diseases and toxic megacolon.

PHOSPHINE-MEDIATED AMINE-AZIDE COUPLING IN IONIC LIQUID FOR BIOCONJUGATION REACTIONS

The use of ionic liquids as a solvent for chemoselective bioconjugation reactions is described. For example, methods of preparing bioconjugates in ionic liquids via a phosphine-mediated azide-amine reaction to form a urea linkage between a biomolecule substrate and a second molecule are described. Methods of preparing bioconjugates with amide or enamine linkages in ionic liquids are also described. The methods can be used to prepare tagged biomolecules, such as dye-tagged proteins, peptides, nucleic acids, or saccharides (e.g., aminosaccharides), for use in various applications; to form biomolecule-polymer conjugates; or to form biomolecule-therapeutic agent conjugates, such as antibody-drug conjugates.

LIPID-PEPTIDE FUSION INHIBITORS AS SARS-COV-2 ANTIVIRALS

Described herein is a composition and method of treating COVID-19 with lipid-peptide fusion antiviral therapy. Also described is a composition and method of treating Ebola with lipid-peptide fusion antiviral therapy.

TARGETED COMPOSITIONS

The invention provides certain nucleic acids (e.g., double stranded siRNA molecules), as well as conjugates that comprise a targeting moiety, a double stranded siRNA, and optional linking groups. Certain embodiments also provide synthetic methods useful for preparing the conjugates. The conjugates are useful to target therapeutic double stranded siRNA to the liver and to treat liver diseases including hepatitis (e.g. hepatitis B and hepatitis D).

COMPOUNDS FOR REGULATING TRAINED IMMUNITY, AND THEIR METHODS OF USE

Provided herein are compounds of Formula (I), as well as compositions comprising a compound of Formula (I) and uses thereof.

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SEQUENTIAL TARGETING IN CROSSLINKING NANO-THERANOSTICS FOR TREATING BRAIN TUMORS
20230076792 · 2023-03-09 ·

The present invention provides a compound of Formula (I) as defined herein. The present invention also provides a nanoparticle comprising a plurality of the conjugates of the present invention, and methods of using the nanoparticles for drug delivery, treating a disease, and methods of imaging.