Inhibitors of 2-oxoglutarate dioxygenase as gamma globin inducers
OwnerPeter Ratcliffeinventor
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Abstract
The present invention provides methods for increasing endogenous globin expression in a subject, specifically γ-globin expression. The invention also provides compounds and medicaments for use in the methods. The methods are particularly useful for increasing fetal hemoglobin production in a subject, and can be used to treat various disorders, e.g., β thalassemia and sickle cell disease.
Claims59
A method for increasing endogenous gamma globin (γ-globin) in a subject, the method comprising administering to the subject an agent which increases expression of the gene encoding γ-globin.
A method for increasing the level of fetal hemoglobin in a subject, the method comprising administering to the subject an agent which increases expression of the gene encoding γ-globin.
A method for treating a disorder associated with abnormal hemoglobin in a subject, the method comprising increasing the level of fetal hemoglobin in the subject.
A method for increasing the proportion of fetal hemoglobin relative to non-fetal hemoglobin produced by a cell or population of cells, the method comprising administering to the cell or population of cells an agent which increases expression of the gene encoding γ-globin.
A medicament comprising an agent which increases expression of the gene encoding γ-globin for use in increasing fetal hemoglobin level in a subject.
A method for increasing endogenous gamma globin (γ-globin) in a subject, the method comprising administering to the subject an agent which increases expression of the gene encoding γ-globin, wherein the agent increases expression of the gene encoding γ-globin by increasing the stability or activity of an alpha subunit of hypoxia inducible factor (HIFα).
A method for increasing the level of fetal hemoglobin in a subject, the method comprising administering to the subject an agent which increases expression of the gene encoding γ-globin, wherein the agent increases expression of the gene encoding γ-globin by increasing the stability or activity of an alpha subunit of hypoxia inducible factor (HIFα).
A method for treating a disorder associated with hemoglobinopathy in a subject, the method comprising administering to the subject an agent which increases the level of fetal hemoglobin in the subject, wherein the agent increases the level of fetal hemoglobin in the subject by increasing the stability or activity of an alpha subunit of hypoxia inducible factor (HIFα).
A method for increasing the proportion of fetal hemoglobin relative to non-fetal hemoglobin produced by a cell or population of cells, the method comprising administering to the cell or population of cells an agent which increases expression of the gene encoding γ-globin, wherein the agent increases expression of the gene encoding γ-globin by increasing the stability or activity of an alpha subunit of hypoxia inducible factor (HIFα).
A medicament comprising an agent which increases expression of the gene encoding γ-globin for use in increasing fetal hemoglobin level in a subject, wherein the agent increases expression of the gene encoding γ-globin by increasing the stability or activity of HIFα.
Dependencies are inferred from the claim text.