Compositions and methods for increasing fetal hemoglobin and treating sickle cell disease
AssigneeFulcrum Therapeutics, Inc.
InventorsPeter Rahl, Angela Marie Cacace, Michael Cameron, Akshay Kakumanu
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Abstract
The present invention relates to compositions and methods of increasing levels of fetal hemoglobin (HbF) in cells. The present invention further relates to methods for treating patients suffering from blood cell diseases, including those associated with reduced amounts of functional adult hemoglobin (HbA), such as sickle cell disease and β-thalassemias.
Claims56
A method for increasing expression of a fetal hemoglobin (HbF) in a cell, optionally a eukaryotic cell, comprising contacting a cell with an inhibitor of a target protein or target protein complex that functions to regulate HbF expression, optionally wherein the target protein is Cullin 3 (CUL3) or Speckle-type POZ protein (SPOP).
A pharmaceutical composition for increasing expression of fetal hemoglobin (HbF) in a subject in need thereof, comprising:
- an inhibitor of a target protein or protein complex that functions to regulate HbF expression, and
- a diluent, excipient, and carrier
- wherein the composition is formulated for delivery to a subject in need thereof.
Dependencies are inferred from the claim text.