US2022071981A1PendingUtilityA1

Compositions and methods for hemoglobin production

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Assignee: CHILDRENS HOSPITAL PHILADELPHIAPriority: Jan 30, 2017Filed: Nov 22, 2021Published: Mar 10, 2022
Est. expiryJan 30, 2037(~10.5 yrs left)· nominal 20-yr term from priority
Inventors:Gerd BlobelJeremy GrevetJunwei ShiChristopher R. Vakoc
C12N 15/1137A61K 48/00C12N 15/90A61K 31/517A61P 7/06A61K 45/06A61K 31/454C12N 2310/20
58
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Claims

Abstract

Methods and compositions for producing fetal hemoglobin and treating a hemoglobinopathy or thalassemia are disclosed.

Claims

exact text as granted — not AI-modified
What is claimed is: 
     
         1 . A method of increasing the level of fetal hemoglobin in a cell or subject, the method comprising administering at least one eukaryotic translation initiation factor 2-alpha kinase 1 (eIF2αK1) inhibitor to the cell or subject. 
     
     
         2 . The method of  claim 1 , wherein the subject has a β-chain hemoglobinopathy. 
     
     
         3 . The method of  claim 1 , wherein the subject has thalassemia. 
     
     
         4 . The method of  claim 1 , wherein the subject has sickle cell anemia. 
     
     
         5 . The method of  claim 1 , wherein the eIF2αK1 inhibitor is a small molecule. 
     
     
         6 . The method of  claim 1 , wherein the eIF2αK1 inhibitor is a kinase domain inhibitor or a heme binding domain inhibitor. 
     
     
         7 . The method of  claim 1 , wherein the cell is an erythroid cell. 
     
     
         8 . The method of  claim 1 , further comprising administering at least one fetal hemoglobin inducer to the cell or subject. 
     
     
         9 . The method of  claim 8 , wherein said fetal hemoglobin inducer is pomalidomide. 
     
     
         10 . The method of  claim 8 , wherein said fetal hemoglobin inducer is a histone methyltransferase (HMT) inhibitor. 
     
     
         11 . The method of  claim 10 , wherein said HMT inhibitor is UNC0638. 
     
     
         12 . A method of treating a hemoglobinopathy in a subject in need thereof, the method comprising administering a composition comprising at least one eukaryotic translation initiation factor 2-alpha kinase 1 (eIF2αK1) inhibitor and a pharmaceutically acceptable carrier to the subject. 
     
     
         13 . The method of  claim 12 , wherein the subject has a β-chain hemoglobinopathy. 
     
     
         14 . The method of  claim 12 , wherein the subject has thalassemia. 
     
     
         15 . The method of  claim 12 , wherein the subject has sickle cell anemia. 
     
     
         16 . The method of  claim 12 , wherein the eIF2αK1 inhibitor is a small molecule. 
     
     
         17 . The method of  claim 12 , wherein the eIF2αK1 inhibitor is a kinase domain inhibitor or a heme binding domain inhibitor. 
     
     
         18 . The method of  claim 12 , further comprising administering at least one fetal hemoglobin inducer to the subject. 
     
     
         19 . The method of  claim 18 , wherein said fetal hemoglobin inducer is pomalidomide. 
     
     
         20 . The method of  claim 18 , wherein said fetal hemoglobin inducer is a histone methyltransferase (HMT) inhibitor. 
     
     
         21 . The method of  claim 20 , wherein said HMT inhibitor is UNC0638. 
     
     
         22 . The method of  claim 12 , wherein the eIF2αK1 inhibitor is contained within a cell administered to the subject.

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