Patent classifications
A61P7/06
3-(5-oxy)-1-oxoisoindolin-2-yl)piperidine-2,6-dione Derivatives and Uses thereof
The present disclosure relates to compounds of formula (I) and pharmaceutical compositions and their use in reducing Widely Interspaced Zinc Finger Motifs (WIZ) expression levels, or inducing fetal hemoglobin (HbF) expression, and in the treatment of inherited blood disorders (e.g., hemoglobinopathies, e.g., beta-hemoglobinopathies), such as sickle cell disease and beta-thalassemia.
CRISPR/Cas9 complex for introducing a functional polypeptide into cells of blood cell lineage
Provided herein are CRIS-PR/Cas9 complexes and methods of using same.
CRISPR/Cas9 complex for introducing a functional polypeptide into cells of blood cell lineage
Provided herein are CRIS-PR/Cas9 complexes and methods of using same.
Hematopoiesis-enhancing agent
The purpose of the invention is to provide a novel hematopoiesis-promoting agent and a medicament comprising the hematopoiesis-promoting agent as an active ingredient for preventing or treating anemia, in particular refractory anemia. The present invention provides a hematopoiesis-promoting agent comprising an S-adenosylmethionine synthase inhibitor.
Hematopoiesis-enhancing agent
The purpose of the invention is to provide a novel hematopoiesis-promoting agent and a medicament comprising the hematopoiesis-promoting agent as an active ingredient for preventing or treating anemia, in particular refractory anemia. The present invention provides a hematopoiesis-promoting agent comprising an S-adenosylmethionine synthase inhibitor.
BORON CONTAINING SMALL MOLECULES AS ANTI-INFLAMMATORY AGENTS
Methods of treating anti-inflammatory conditions through the use of boron-containing small molecules are disclosed.
PYRROLO[2,3-D]PYRIMIDINYL, PYRROLO[2,3-B]PYRAZINYL AND PYRROLO[2,3-D]PYRIDINYL ACRYLAMIDES
The present invention provides pharmaceutically active pyrrolo[2,3-d]pyrimidinyl and pyrrolo[2,3-d]pyridinyl acrylamides and analogues thereof. Such compounds are useful for inhibiting Janus Kinase (JAK). This invention also is directed to compositions comprising methods for making such compounds, and methods for treating and preventing conditions mediated by JAK.
ANTIGEN BINDING MOLECULES WITH INCREASED FC RECEPTOR BINDING AFFINITY AND EFFECTOR FUNCTION
The present invention relates to antigen binding molecules (ABMs). In particular embodiments, the present invention relates to recombinant monoclonal antibodies, including chimeric, primatized or humanized antibodies specific for human CD20. In addition, the present invention relates to nucleic acid molecules encoding such ABMs, and vectors and host cells comprising such nucleic acid molecules. The invention further relates to methods for producing the ABMs of the invention, and to methods of using these ABMs in treatment of disease. In addition, the present invention relates to ABMs with modified glycosylation having improved therapeutic properties, including antibodies with increased Fc receptor binding and increased effector function.
METHODS AND COMPOSITIONS FOR TREATING ANEMIA USING ACTRIIB LIGAND TRAPS AND MTOR INHIBITORS
Provided herein are methods of treating anemia or for enhancing late stage erythropoiesis in a subject comprising administering to the subject an activin type IIB (ActRIIB) ligand trap and an mTOR inhibitor.
METHODS AND COMPOSITIONS FOR TREATING ANEMIA USING ACTRIIB LIGAND TRAPS AND MTOR INHIBITORS
Provided herein are methods of treating anemia or for enhancing late stage erythropoiesis in a subject comprising administering to the subject an activin type IIB (ActRIIB) ligand trap and an mTOR inhibitor.