Heterocyclic entities that modulate PI3 kinase activity, pharmaceutical compositions containing the heterocyclic entities, and methods of using these chemical entities for treating diseases and conditions associated with PI3 kinase activity are described herein.

The present invention relates to a small activating nucleic acid molecules and uses thereof for treating diseases and conditions, such as thrombocytopenia, related to THPO protein deficiency or insufficiency. As described herein, small activating nucleic acid molecules can be double-stranded or single-stranded RNA molecules targeting the promoter region of the Thpo/THPO gene through an RNA activation mechanism and comprise a first nucleic acid strand and a second nucleic acid strand. The double-stranded RNA molecule targeting the promoter region of the Thpo/THPO gene comprises two nucleic acid strands of 16 to 35 nucleotides in length, wherein one nucleic acid strand has at least 75% homology or complementarity to a target selected from the promoter region of the Thpo/THPO gene. The present invention also relates to pharmaceutical compositions and formulations comprising the small activating nucleic acid molecules and methods for up-regulating the expression of the Thpo/THPO gene in a cell and treating diseases and conditions, related to THPO protein deficiency or insufficiency, by administering small activating nucleic acid molecules, pharmaceutical compositions, and formulations thereof.

The present invention relates to a small activating nucleic acid molecules and uses thereof for treating diseases and conditions, such as thrombocytopenia, related to THPO protein deficiency or insufficiency. As described herein, small activating nucleic acid molecules can be double-stranded or single-stranded RNA molecules targeting the promoter region of the Thpo/THPO gene through an RNA activation mechanism and comprise a first nucleic acid strand and a second nucleic acid strand. The double-stranded RNA molecule targeting the promoter region of the Thpo/THPO gene comprises two nucleic acid strands of 16 to 35 nucleotides in length, wherein one nucleic acid strand has at least 75% homology or complementarity to a target selected from the promoter region of the Thpo/THPO gene. The present invention also relates to pharmaceutical compositions and formulations comprising the small activating nucleic acid molecules and methods for up-regulating the expression of the Thpo/THPO gene in a cell and treating diseases and conditions, related to THPO protein deficiency or insufficiency, by administering small activating nucleic acid molecules, pharmaceutical compositions, and formulations thereof.

Anti-human CD40L antibodies engineered to lack the ability to activate platelets and methods for treating patients having a CD40L-associated disease.

The invention provides adeno-associated virus (AAV) Factor VIII (FVIII)-encoding/expressing vectors and virus, including AAV FVIII vectors with high expression activity and AAV FVIII vectors that express full-length or truncated functional FVIII protein. The invention also relates to methods of making the herein described AAV FVIII vectors, recombinant AAV FVIII virus particles comprising or expressing such vectors, associated pharmaceutical formulations comprising the same and therapeutic uses thereof.

The present invention relates to the field of chromatography. More closely, the invention relates to a chromatographic method for purification of plasmaproteins, such as Factor VIII, von Willebrand factor and Factor IX. The chromatographic method is performed on a matrix comprising an inner porous core and outer porous lid surrounding said core.

Use of a solid calcium compound in the fabrication of a hemostatic agent for reducing bleeding from a surgical site during and/or after a surgical treatment in a patient is provided, wherein the solid calcium compound is selected from the group consisting of calcium phosphate, calcium sulfate, calcium carbonate, calcium oxide, calcium hydroxide, hydroxyapatite, and a combination thereof.

Compositions comprised of: (i) a blend of fibrinogen, thrombin, wherein the blend is in the form of a plurality of particles, and (ii) a hydrophobic dispersant, wherein the composition is in the form of a paste at at-least one temperature in the range of 10° C., to 37° C., are disclosed herein. Methods for preparing a fibrin adhesive sealant in/on an injured tissue are further disclosed.

Compositions comprised of: (i) a blend of fibrinogen, thrombin, wherein the blend is in the form of a plurality of particles, and (ii) a hydrophobic dispersant, wherein the composition is in the form of a paste at at-least one temperature in the range of 10° C., to 37° C., are disclosed herein. Methods for preparing a fibrin adhesive sealant in/on an injured tissue are further disclosed.

The present invention relates to a platelet aggregating agent containing amorphous polyphosphate as an active ingredient, wherein the polyphosphate is a Ca salt of polyphosphate. The platelet aggregating agent acts on damaged gastrointestinal mucosa in inflammatory bowel disease and exerts a platelet aggregating action, thereby allowing remission/improvement of the inflammatory bowel disease.