Target Information
| Target General Information | Top | |||||
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| Target ID |
T31825
(Former ID: TTDI01041)
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| Target Name |
Hemoglobin subunit beta (HBB)
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| Synonyms |
LVVhemorphin7; Hemoglobin beta chain; Betaglobin; Beta-globin
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| Gene Name |
HBB
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| Target Type |
Clinical trial target
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[1] | ||||
| Disease | [+] 1 Target-related Diseases | + | ||||
| 1 | Thalassaemia [ICD-11: 3A50] | |||||
| Function |
Involved in oxygen transport from the lung to the various peripheral tissues.
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| BioChemical Class |
Pore-forming globin
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| UniProt ID | ||||||
| Sequence |
MVHLTPEEKSAVTALWGKVNVDEVGGEALGRLLVVYPWTQRFFESFGDLSTPDAVMGNPK
VKAHGKKVLGAFSDGLAHLDNLKGTFATLSELHCDKLHVDPENFRLLGNVLVCVLAHHFG KEFTPPVQAAYQKVVAGVANALAHKYH Click to Show/Hide
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| 3D Structure | Click to Show 3D Structure of This Target | PDB | ||||
| HIT2.0 ID | T40KDU | |||||
| Drugs and Modes of Action | Top | |||||
|---|---|---|---|---|---|---|
| Clinical Trial Drug(s) | [+] 2 Clinical Trial Drugs | + | ||||
| 1 | LentiGlobin | Drug Info | Phase 3 | Beta thalassemia | [2] | |
| 2 | OTL-300 | Drug Info | Phase 1/2 | Beta thalassemia | [3] | |
| Mode of Action | [+] 1 Modes of Action | + | ||||
| Modulator | [+] 1 Modulator drugs | + | ||||
| 1 | LentiGlobin | Drug Info | [1] | |||
| Target Affiliated Biological Pathways | Top | |||||
|---|---|---|---|---|---|---|
| KEGG Pathway | [+] 2 KEGG Pathways | + | ||||
| 1 | African trypanosomiasis | |||||
| 2 | Malaria | |||||
| Reactome | [+] 4 Reactome Pathways | + | ||||
| 1 | Erythrocytes take up carbon dioxide and release oxygen | |||||
| 2 | Erythrocytes take up oxygen and release carbon dioxide | |||||
| 3 | Scavenging of heme from plasma | |||||
| 4 | Factors involved in megakaryocyte development and platelet production | |||||
| WikiPathways | [+] 7 WikiPathways | + | ||||
| 1 | Binding and Uptake of Ligands by Scavenger Receptors | |||||
| 2 | Uptake of Carbon Dioxide and Release of Oxygen by Erythrocytes | |||||
| 3 | Uptake of Oxygen and Release of Carbon Dioxide by Erythrocytes | |||||
| 4 | Factors involved in megakaryocyte development and platelet production | |||||
| 5 | Folate Metabolism | |||||
| 6 | Vitamin B12 Metabolism | |||||
| 7 | Selenium Micronutrient Network | |||||
| References | Top | |||||
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| REF 1 | Preclinical evaluation of efficacy and safety of an improved lentiviral vector for the treatment of beta-thalassemia and sickle cell disease. Curr Gene Ther. 2015;15(1):64-81. | |||||
| REF 2 | ClinicalTrials.gov (NCT03207009) A Study Evaluating the Efficacy and Safety of the LentiGlobin BB305 Drug Product in Subjects With Transfusion-Dependent beta-Thalassemia. U.S. National Institutes of Health. | |||||
| REF 3 | ClinicalTrials.gov (NCT03275051) Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL). U.S. National Institutes of Health. | |||||
| REF 4 | Clinical pipeline report, company report or official report of Orchard Therapeutics. | |||||
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