Table 1 Novel therapeutics in Sickle Cell Disease
From: Erythropoiesis: insights into pathophysiology and treatments in 2017
a. Gene therapy | ||||||
Bluebird Bio | BB305 lentiviral vector (betibeglogene darolentivec) | Anti-sickling β-globin | - Severe SCD - age ≥ 18 years | IV | NCT02140554 | Open - Phase 1/2 |
Children’s Hospital Medical Center, Cincinnati | Gamma globin lentiviral vector | Anti-sickling γ-globin | - Severe SCD - age 18–35 | IV | NCT02186418 | Open - Phase 1/2 |
b. Small molecule targets | ||||||
Boston University | SIRT1 | HbF induction | Early stage of development - pre-clinical | |||
The Cleveland Clinic | Decitabine and tetrahydrouridine | HbF induction | - age ≥ 18 years - HbSS, HbSβ°, HbSβ+, HbSC | Oral | NCT01685515 | Completed - Phase 1 |
Celgene | Pomalidomide | HbF induction | - age 18–60 - HbSS, HbSβ° | Oral | NCT01522547 | Completed - Phase 1 |
Novartis Pharmaceuticals | Panobinostat | HbF induction | - age ≥ 18 yers - HbSS, HbSβ° | Oral | NCT01245179 | Open - Phase 1 |
Dana Farber Cancer Institute | Vorinostat (Zolinza) | HbF induction | - age 18–60 - HbSS, HbSβ° | Oral | NCT01000155 | Discontinued - Phase 1/2 |