Abstract: The present invention relates to (a) an active camptothecin derivative represented by Chemical Formula 1, which is designed to bind to DDX5 protein and E3 ligase; (b) a prodrug thereof, preferably an antibody-drug conjugate (ADC) thereof, which is designed to release the active camptothecin derivative (a) at a target site in vivo; or (c) a complex containing a DDX5 protein-targeting ligand, in which the ability of the active camptothecin derivative (a) or an FL118 compound of Chemical Formula 2 to inhibit type 1 topoisomerase is inactivated through a linker connection. The active camptothecin derivative designed according to the present invention can bind to DDX5 protein in cells to induce cell death through DDX5 protein degradation.

Abstract: The present invention relates to a method for preparing 4?-thio-5-aza-2?-deoxycytidine which exhibits DNMT1 inhibitory activity. The production method according to the present invention can enhance reaction efficiency through efficient process development and can stereoselectively prepare 4?-thio-5-aza-2?-deoxycytidine, and thus is economical and appropriate for mass production.

Abstract: Provided is an antibody-drug conjugate (ADC) having a drug-linker conjugate (A) linked thereto and a preparation method therefor, including a combination of a camptothecin-based drug that degrades DDX5 protein with a DAR of 4 or more and either (i) an acid-sensitive linker or (ii) an enzyme-sensitive linker. The antibody-drug conjugate is designed to increase the therapeutic index of the camptotechin-based drug payload while suppressing the non-selective uptake of the camptothecin-based drug and/or ADC released from apoptotic cells. The ADC is designed and/or synthesized so that two types of drug-linker conjugates including: a drug-linker conjugate (A) composed of a combination of a camptothecin-based drug that degrades DDX5 protein with a DAR of 4 or more and either (i) an acid-sensitive linker or (ii) an enzyme-sensitive linker; and a drug-linker conjugate (B) composed of a combination of a non-camptothecin cytotoxic drug and an enzyme-sensitive linker are each linked to a single antibody.

Abstract: The present invention relates to the combination therapy of 4?-thio-5-aza-2?-deoxycytidine (Aza-T-dCyd), a multi-target inhibitor including a DNMT1 inhibitor, and Venetoclax, an inhibitor of BCL-2, which mediates apoptosis.

Abstract: The present invention relates to (a) an active camptothecin derivative represented by Chemical Formula 1 below, which is designed to bind to the DDX5 protein and an E3 ligase; (b) a prodrug thereof designed to release the active camptothecin derivative (a) at an in vivo target site; or (c) a ligand-containing complex targeting the DDX5 protein, in which an topoisomerase-1 inhibitory ability of the active camptothecin derivative (a) or FL118 compound of Chemical Formula 2 is inactivated through linker conjugation. The active camptothecin derivative designed according to the present invention may bind to the DDX5 protein in cells to induce cell death through DDX5 protein degradation.

Abstract: The present invention relates to pharmaceutical compositions for treating blood cancer, which includes 4?-thio-5-aza-2?-deoxycytidine (Aza-T-dCyd), and methods of treating blood cancer using a composition for oral administration that includes Aza-T-dCyd. The present invention provides a significant blood cancer therapeutic effect without having toxicity in a blood cancer mouse model or a human even with the Aza-T-dCyd dose conventionally known to have toxicity. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

Abstract: Provided herein are a linker compound, a linker-drug conjugate where the linker compound is conjugated to a drug, an antibody-drug conjugate where a drug is conjugated to an antibody or an antigen binding fragment thereof via the linker compound, and a method for treating cancer by administering the antibody-drug conjugate to a subject in need thereof.

Abstract: Ophthalmic therapeutic agents having as pharmaceutically active ingredient a poorly water soluble drug of formula 1 are described. Specifically, an ophthalmic formulation containing an inclusion complex of a poorly soluble drug of formula 1 enclosed in cyclodextrin or a cyclodextrin derivative in an aqueous solution of pH 10 or higher is administered to a patient in need of optic nerve protection.

Abstract: The present disclosure provides crystalline polymorphs of 5-aza-4?-thio-2?-deoxycytidine. The crystalline polymorphs may be formulated in a pharmaceutical composition, optionally in combination with an additional chemotherapeutic agent. The crystalline polymorphs are useful to treat various diseases including blood cancers, such as myelodysplastic syndrome and leukemia. A process for preparing the crystalline polymorphs of 5-aza-4?-thio-2?-deoxycytidine is also disclosed. This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.