The present invention relates to polydixylitol polymer based gene transporter (PdXYP) and a preparation method thereof. Further, the present invention relates to a nucleic acid delivery complex where the nucleic acids for treatment are conjugated to the gene transporter and a pharmaceutical composition for gene therapy including the complex as an active ingredient. In addition, the present invention relates to the gene transporter, gene delivery complex, and gene therapy using the gene transporter and gene delivery complex. It was confirmed that the PdXYP of the present invention has a considerably higher nucleic acid delivery rate than existing gene transporters, has almost no cytotoxicity in the conjugate when conjugated with DNA, also has very high in vivo transfection efficiency, and above all, especially has considerably high transfection efficiency for brain tissues, which has involved difficulty in gene therapy due to the blood brain barrier for a while. Accordingly, the gene transporter of the present invention can not only be used as experimental gene transporters, but can also be broadly used for various tissues in the body based on the nucleic acids for treatment to be conjugated in the field of gene therapy regarding various diseases.

The invention provides non-hormonal vitamin D conjugated to therapeutic RNA compounds that result in the compounds having increased absorption, bioavailability or circulating half-life when compared to non-conjugated forms. The vitamin D targeting groups are coupled to the therapeutic RNA compounds via the third carbon on the vitamin D backbone.

In an embodiment of the invention, a composition for treating a cell population comprises an Affinity Medicant Conjugate (AMC). The medicant moiety can be a toxin including an acylfulvene or a drug moiety. The affinity moiety can be an antibody, a binding protein, a steroid, a lipid, a growth factor, a protein, a peptide or non peptidic. The affinity moiety can be covalently bound to the medicant via a linker. Novel linkers that can be directed to cysteine, arginine or lysine residues based on solution pH allow greater flexibility in preserving and/or generating specific epitopes in the AMC.

The invention relates to a method of treating or diagnosing a disease state mediated by monocytes. The method utilizes a composition comprising a conjugate or complex of the general formula
A.sub.b-X
wherein the group A.sub.b comprises a ligand that binds to monocytes, and when the conjugate is being used for treatment of the disease state, the group X comprises an immunogen, a cytotoxin, or a compound capable of altering monocyte function, and when the conjugate is being used for diagnosing the disease state, the group X comprises an imaging agent.

The present invention aims to provide a pharmaceutical composition for transcolonic absorption capable of delivering a physiologically active substance (in particular, a water-soluble physiologically active substance of high molecular weight) having an intracellular site of action into specific tissue cells with high specificity, noninvasively by a means of administration other than injection. The pharmaceutical composition for transcolonic absorption of the present invention is characterized by comprising at least the following (a) and (b); (a) a physiologically active substance having an intracellular site of action and bound with an introduction substance into lipoprotein, and (b) a compound having an action of enhancing large intestinal mucosal epithelial permeability of the physiologically active substance.

The invention provides methods and compositions for delivering a nucleic acid to a cell or the cytosol of the target cell. The method includes contacting the cell with, 1) a membrane-destabilizing polymer; and 2) a nucleic acid conjugate. The nucleic acid conjugate includes a targeting ligand bound to an optional linker and a nucleic acid.

Therapeutic proteins for inducing apoptosis in cells and method for using such proteins are described. The protein, e.g., a chimeric protein, has a host peptide fused to a more stable BH3 domain peptide. The host peptide may be Calbindin D9k, a N-terminal or C-terminal half-domain of Calmodulin, Parvalbimin (“Pa”), an amino acid sequence variant thereof, or a modified variant thereof. The BH3 domain may be Bim, Bid, Bad, Bik, Bmf, Hrk, Puma, or Noxia. Moreover, various conjugates may be attached to the chimeric protein, including a folate and polyethylene glycol.

The present application relates to an inhibitor of fusion between a viral membrane from an enveloped virus and a cell membrane, where the viral membrane comprises a fusion mediating protein including a C-terminal peptide. The inhibitor comprises the C-terminal peptide of the fusion mediating protein from an enveloped virus and tocopherol or a derivative or pharmaceutically acceptable salt thereof attached to the C-terminal peptide. Also disclosed is a pharmaceutical composition including the inhibitor as well as methods of inhibiting viral fusion, blocking viral spread, and preventing or treating viral infection, with the inhibitor or pharmaceutical composition.

The present invention is directed to novel natural product-derived ratjadone-based compounds useful as payloads (or toxins) in drug-conjugates constructs with cell target binding moieties (CTBM) and payload-linker compounds useful in connection with drug conjugates. The present invention further relates to new ratjadone compositions including the aforementioned payloads, payload-linkers and drug conjugates, and methods for using these payloads, payload-linkers and drug conjugates, to treat pathological conditions including cancer, inflammatory and infectious diseases.

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.