Abstract: The present disclosure relates to treatment of bleeding disorders, in particular, hemophilia A (mild, moderate or severe hemophilia) and von Willebrand disease (VWD), Type 1 or Type 2 or Type 3, using VWF targeting agents such as a PEGylated anti-VWF aptamer, BT200.

Abstract: The present invention provides compounds of Formula (I) which can be used as Syk inhibitors and potently as therapeutic agents against diseases mediated by Syk.

Abstract: The present disclosure relates to agents, compositions targeting to von Willebrand factor (VWF). The VWF targeting agents are synthetic polynucleotides, including VWF binding agents and their reversal agents. The VWF binding agents are VWF binding aptamers that bind to and inhibit the VWF activities. The VWF binding agents can be reversed using reversal agents to reverse the inhibitory effect and thereby restore VWF activities. The disclosure further provides methods for regulating the activities of VWF, thereby modulating VWF mediated platelet functionality, such as thrombosis. the present VWF targeting agents may be used for preventing thrombus formation and treating thrombotic disorders.

Abstract: The present invention provides a compound suitable for use as an acetyl CoA carboxylase (ACC) inhibitor, specifically, a thienopyridine derivative, and use of the compound in the preparation of drugs for treating metabolic disorders, cancers or other proliferative disorders, and nonalcoholic steatohepatitis (NASH).

Abstract: Provided herein are compounds of Formula (I), a stereoisomer, enantiomer or a pharmaceutically acceptable salt thereof; wherein variables are as defined herein; and their pharmaceutical compositions, which are useful as modulators of the activity of Farnesoid X receptors (FXR).

Abstract: The present disclosure provides T7 RNA polymerase variants with enhanced transcriptional activity, and methods of using such variants to produce modified oligonucleotides, such as 2?-modified oligonucleotides. These polymerase variants and methods thereof improve the transcription yield of modified oligonucleotides.

Abstract: The present invention provides VWF protective agents, including aptamers and antibodies for the treatment and or amelioration of complications or disorders arising from aberrant binding or function of VWF.

Abstract: The present invention provides VWF protective agents, including aptamers and antibodies for the treatment and or amelioration of complications or disorders arising from aberrant binding or function of VWF.

Abstract: The present invention provides VWF protective agents, including aptamers and antibodies for the treatment and or amelioration of complications or disorders arising from aberrant binding or function of VWF.

Abstract: Materials and methods are provided for producing aptamer therapeutics having fully modified nucleotide triphosphates incorporated into their sequence.

Abstract: The present invention relates to the primate family of cytokines. Provided herein are polynucleotides encoding such polypeptides, and reagents useful for producing the encoded polypeptides in a host source, and for identifying compounds which bind and modulate the activity of the encoded polypeptides. Also provided are reagents useful for the diagnosis or treatment of inflammatory and/or autoimmune related diseases in primates.

Abstract: The present invention provides stabilized, high affinity nucleic acid ligands to PSMA. Methods for the identification and preparation of novel, stable, high affinity ligands to PSMA using the SELEX™ method with 2?-O-methyl substituted nucleic acids, and cell surface SELEX™ are described herein. Also included are methods and compositions for the treatment and diagnosis of disease characterized by PSMA expression, using the described nucleic acid ligands.

Abstract: Materials and methods are provided for producing aptamer therapeutics having fully modified nucleotide triphosphates incorporated into their sequence.

Abstract: The invention provides screening methods for detecting and identifying compounds that bind to fungal specific target proteins and nucleic acids, as well as compounds which, upon binding or otherwise interacting with the target protein, can inhibit fungal growth, a method of preventing or inhibiting fungal growth in culture, a method of preventing or inhibiting fungal growth in a mammal and a method of studying pathogenic mycetes using such nucleic acid and/or protein sequences. Particularly preferred is the inhibition of the fungus Candida albicans.

Abstract: Disclosed are pharmaceutical compositions comprising aryl and heteroaryl hydrazone, hydrazine and thiosemicarbazone derivatives of formulae I and II, and methods for using the compositions as antifungal agents. The invention also relates to novel aryl and heteroaryl hydrazone, hydrazine and thiosemicarbazone derivatives of formulae I and II, their preparation, to pharmaceutical compositions containing them, and to methods of using them to alleviate fungal infections.