This invention provides nanoparticles containing protein-polynucleotide complexes and methods of manufacture and methods of their use. These particles, when administered to a subject in need, are capable of delivering these complexes to target cells and target intracellular locations where they can perform a therapeutic function. In some embodiments, this therapeutic function includes gene editing, induction of gene skipping, and regulation of gene expression. The instant nanoparticles are generally formed by designing and synthesizing the polynucleotide to according to its intended function, combining it with a protein selected for its substrate specificity and enzymatic function in a manner to form a polynucleotide-protein complex, encapsulating the complexes by dispersion into a water-insoluble surfactant system, optionally adding a targeting ligand, and stabilizing the nanoparticles by crystallization of the ligand to the surface of the nanoparticles.

The invention provides amphiphilic biocompatible copolymers which have a hydrophilic backbone and pendant hydrophobic groups. The polymers form nanoscale molecular aggregates in aqueous environments, which have hydrophobic interiors within which anticancer drugs may be solubilized. The polymers optionally feature attached antibodies, receptor ligands, and other targeting moieties which mediate adherence of the drug-carrying aggregates to targeted cancer cells.

The present invention is directed to glypican-3-specific peptide reagents, methods for detecting hepatocellular carcinoma cells using the peptide reagents, and methods for targeting hepatocellular carcinoma cells using the peptide reagents.

The problem addressed by the present invention is to develop a pharmaceutical having therapeutic efficacy against epirubicin-resistant tumors. The present invention provides a micelle having an anti-cancer agent disposed inside the core of the micelle formed by an epirubicin-conjugated copolymer.

The subject invention pertains to polyacrylate homopolymers produced from acrylolated drug monomers. The homopolymers can be produced in the form of nanoparticles. The nanoparticles comprising the homopolymers can be produced via a free radical-induced emulsion polymerization of the acrylolated drug monomers to produce an aqueous emulsion of uniformly sized nanoparticles. The homopolymers of the invention containing acrylolated antibiotic monomers can be active against Gram-positive and Gram-negative bacteria, such as Staphylococcus aureus and Escherichia coli. Accordingly, methods are provided of treating a disease, for example, an infection, by administering to a subject the homopolymers, homopolymeric nanoparticles, or emulsions containing homopolymeric nanoparticles of the invention.

The present disclosure provides compositions which shown preferential targeting or delivery of a nucleic acid composition to a particular organ. In some embodiments, the composition comprises a steroid or sterol, an ionizable cationic lipid, a phospholipid, a PEG lipid, and a permanently cationic lipid which may be used to deliver a nucleic acid.

The present invention pertains to novel thiocycloheptyne derivatives of general formula (I): and in particular to thiacycloalkynesulfoimine derivatives and their synthesis. The invention also relates to the use of the novel thiocycloheptyne derivatives in coupling reactions with linkers and drugs. The invention further relates to the use of the novel thiocycloheptynes in bioorthogonal (copper-free) click reactions. The invention further pertains to the use of the novel thiocycloheptyne derivatives in the generation of advanced multifunctional drug delivery systems (drug-loaded) nanoparticles.

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An amphiphilic block copolymer having any one of the formulas S-[B]-H, S-[B]-H(D), D-[B]-H, S-B(D)-H, S-[B]-H-[B]-S, S-[B]-H(D)-[B]-S, D-[B]-H-[B]-S, D-[B]-H-[B]-D, S-B(D)-H-[B]-S or S-B(D)-H-B(D)-S; wherein S is a hydrophilic surface stabilizing group; B is a spacer group; H is a hydrophobic polymer or oligomer; D is a drug molecule; ( ) denotes that the group is bonded directly or indirectly as a side chain or as part of a side chain group to the adjacent group; [ ] denotes that the group is optional; and - denotes that each of the adjacent S, B, H or D are linked directly to one another or indirectly to one another via a linker group.

A drug delivery system delivers a cyclic compound to a target cell or tissue while suppressing the pharmacological activity of the cyclic compound. A complex includes the cyclic compound and a delivery carrier having a shaft portion for carrying the cyclic compound. The shaft portion is complexed by inclusion in the cyclic compound to form a host-guest complex.

The present invention relates to a branched peptide that includes a first peptide chain and a second peptide chain having its C-terminal amino acid covalently linked to a sidechain of an amino acid residue of the first peptide chain, wherein the first peptide chain includes a plurality of aromatic amino acids and, optionally, an aromatic group linked to an amino terminus of the first peptide chain; and the second peptide chain includes a plurality of hydrophilic amino acids and an enzyme cleavage site. Pharmaceutical compositions containing the branched peptide and one or more therapeutic agents in an aqueous medium are disclosed, where the branched peptides form micelle structures in the aqueous medium. Methods of using the pharmaceutical composition to deliver therapeutic agents, and for treating various disease conditions are also described.