Provided are structural properties of double-stranded RNA molecules for inducing RNA interference, and more specifically, a novel nucleic acid molecule structure for inducing RNAi with improved stability, and a use thereof. A nucleic acid molecule for inducing RNAi provided herein is capable of significantly improving stability of a double-stranded RNA molecule in vivo while maintaining inhibition efficiency for a target gene, and therefore, as a expandable platform for targeting various genes, the RNA nucleic acid molecule is expected to replace siRNA molecules in the art, and be usefully utilized in the field of diagnosis or treatment of various diseases in research and clinical fields.

A construct, or a pharmaceutically acceptable salt thereof, comprising: (a) a polyethylene glycol-block-poly(L-lysine) polymer moiety, wherein the polyethylene glycol is thiol-functionalized; (b) a cholecystokinin-B (CCK-B) receptor ligand coupled to the polyethylene glycol of the polymer moiety; and (c) a siRNA complexed with the poly(L-lysine) of the polymer moiety, wherein the construct is neutralized.

The present invention relates to conjugate compounds which comprise a peptide moiety (a) which is preferably a hydrophobic modified preS-derived peptide of hepatitis B virus or a respective cyclic peptide, and a NTCP substrate moiety (b), which is preferably a bile acid. The present invention further relates to pharmaceutical compositions comprising at least one conjugate compound. The present invention further relates to medical uses of said conjugate compounds and the pharmaceutical compositions, such as in the diagnosis, prevention and/or treatment of a liver disease or condition, and/or in the inhibition of HBV and/or HDV infection. The present invention further relates to methods of diagnosis, prevention and/or treatment of a said diseases and/or infections.

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 invention relates to a composition in the form of an injectable aqueous solution, wherein the pH is comprised from 6.0 to 8.0, comprising at least: a) amylin, an amylin receptor agonist or an amylin analog; b) a co-polyamino acid bearing carboxylate charges and hydrophobic radicals Hy, characterized in that the composition does not comprise a basal insulin wherein the isoelectric point pI is comprised from 5.8 to 8.5.

It also relates to a composition characterized in that it further comprises a prandial insulin.

Polymer-encapsulated viral vector nanoparticles and methods of using them provide enhanced delivery of genetic material for use in gene therapy and other applications. The nanoparticles include an outer shell containing an oligopeptide-modified poly(beta-amino ester) polymer which encapsulates the vector and allows the vector to transduce cells without the need for pseudotyping or the inclusion of any viral fusion protein, such as VSV-G. The polymer-encapsulated vector nanoparticles have a natural tropism for peripheral blood cells, such as leucocytes, without the need for a targeting moiety, and have an improved safety profile compared to pseudotyped viral vectors.

The invention relates to new cationic polymers conjugated with D-fructose, as a result of which they can selectively interact with specific structure elements on cell surfaces. The problem was that of creating novel, biocompatible, easy-to-produce, D-fructose-conjugated cationic polymers that have a higher selectivity with respect to certain cell types. To solve this problem, the invention proposes cationic polymers with covalently bonded D-fructose of general formula (I) with the following components: a) cationic polymer: macromolecular compounds of n repeat units with one or more positive charges; b) linker: a unit that links the cationic polymer with D-fructose or derivatives of D-fructose by means of any alkyl or aryl group, any alkenyl or alkinyl group, an ether, thioether or amine, an ester, amide or other carboxylic acid derivative, a heterocycle (e.g. triazole or m maleimide), a disulphide, an imine or an imide; c) D-fructose: one or more D-fructoses or D-fructose derivatives in an open-chain, furanoid or pyranoid structure, not glycosidically linked via one of the five possible carbon atoms (1, 3, 4, 5, 6).

A novel anti-tumor agent capable of delivering siRNA or shRNA specifically to pancreatic cancer cells and suppressing tumor growth, invasion, and metastasis of pancreatic cancer is provided. The present invention provides a nucleic acid delivery enhancer for delivering siRNA or shRNA into cells, which consists of a folic acid-cationic oligopeptide complex. The present invention also provides an anti-tumor agent having siRNA or shRNA capable of binding to mRNA or snoRNA expressed in pancreatic cancer cells to inhibit its expression and a folic acid-cationic oligopeptide complex. The siRNA is capable of binding to RNA selected from the group consisting of, for example, SNORA18 snoRNA, NUP85 mRNA, WASF2 mRNA, and SNORA22 snoRNA.

Disclosed are peptides and peptidomimetics that in some embodiments include the amino acid sequence KRGARST or (SEQ ID NO: 1), AKRGARSTA or (SEQ ID NO: 2), or CKRGARSTC (SEQ ID NO: 3). Also disclosed are conjugates and compositions that include the peptides and/or peptidomimetics, methods for directing a moiety to tumor lymphatic vasculature, methods for imaging tumor lymphatic vasculature, methods for reducing or inhibiting tumor metastasis, methods for reducing the number of tumor lymphatic vessels, methods for treating cancer, methods for treating a disease or disorder associated with a gC1q/p32 receptor biological activity, methods for detecting the presence of a gC1q/p32 receptor, methods for detecting interactions between gC1q/p32 receptors and the presently disclosed conjugates and compositions, methods for delivering the presently disclosed conjugates and compositions to gC1q/p32 receptors, methods for assessing gC1q/p32 receptor levels in cells, methods for identifying subjects having diseases associated with gC1q/p32 receptor biological activities, and methods for screening for compounds that interact with gC1q/p32 receptors.

The present disclosure relates to methods and compositions comprising derivatized-chitosan polyplexes reversibly coated with a polyanion-containing block co-polymer for the localized expression of IL-12 in mucosal tissues, preferably in combination with an IFN-1 activator/inducer, for use in cancer immunotherapy.