Details of the Drug

General Information of Drug (ID: DMSCGM8)

Drug Name
LentiGlobin
Synonyms Globin gene therapy (LentiPak, sickle cell/beta-thalassemia), Genetix; Globin gene therapy (LentiPak, sickle cell/beta-thalassemia), bluebird
Indication
Disease Entry ICD 11 Status REF
Beta thalassemia 3A50.2 Phase 3 [1]
Sickle-cell disorder 3A51 Phase 1 [2]
Cross-matching ID
TTD ID
D0C9LQ
Repurposed Drugs (RPD) Click to Jump to the Detailed RPD Information of This Drug

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Hemoglobin subunit beta (HBB) TTM6HK1 HBB_HUMAN Modulator [3]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

1 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.
2 Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
3 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.